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1、Impact Innovation Predictability AccessCENTER FOR DRUG EVALUATION AND RESEARCHADVANCING HEALTH THROUGH INNOVATION:NEW DRUG THERAPYAPPROVALS 2021January 2022www.fda.govAdvancing Health Through Innovation2New Drug Therapy Approvals 2021Table of ContentsDirectors Message.5Executive Summary.6CDERs Novel
2、 Drug Approvals of 2021.10 First-in-Class Drugs.11Drugs for Rare Diseases.13Other Novel Drug Approvals.15Innovation:Use of Expedited Development and Review Pathways.16Fast Track.16Breakthrough Therapy.17Priority Review.17Accelerated Approval.18Overall Use of Expedited Development and Review Methods.
3、18Predictability:Meeting PDUFA Goals.19Access:First Cycle Approvals and First in U.S.Approvals.20New Uses of Approved Drugs.23Approved Drugs Expanded for New Pediatric Populations.25Biosimilar and Interchangeable Biosimilar Approvals.28Other Non-Novel Approvals.30Conclusion.33Appendix A:CDERs Novel
4、Approvals of 2021(in alphabetical order).34Appendix B:Novel Drug Designation.363New Drug Therapy Approvals 20214Advancing Health Through Innovation5New Drug Therapy Approvals 2021Directors MessageWelcome to FDAs Center for Drug Evaluation and Researchs(CDER)annual report,Advancing Health Through Inn
5、ovation:New Drug Therapy Approvals 2021,representing our 11th consecutive year of reporting CDERs notable human drug approvals.This report illustrates our role in bringing innovative drug therapies that are safe and effective to patients in need.Throughout the year,the COVID-19 pandemic continued to
6、 present significant challenges to our entire staff.In spite of these hardships,we have approved many therapies that will advance health for the American public.As in the past,this years new drug therapies span many areas of medicine and disease areas.This is our fifth consecutive year of publishing
7、 an expanded version of the report with an overview of non-novel notable approvals for instance,approvals in which a previously approved drug will be used to treat a different disease or a new patient population,such as children.Our report showcases the new therapies we evaluated for safety and effi
8、cacy,as well as important regulatory tools we used to expedite the review and approval of these applications.We approved the large majority of these therapies by or before their goal dates as defined by congressionally authorized agreements with industry(referred to as user fee programs).Most drugs
9、were also approved in the U.S.before any other country.This report also highlights the years biosimilar product approvals.Of particular note,CDER approved the first two interchangeable biosimilar products in 2021,which are biological products that meet additional requirements and may be substituted
10、for the reference product at the pharmacy without the intervention of a prescriber,similar to how generic drugs are substituted for brand name drugs.More biosimilar and interchangeable biosimilar products mean greater competition that may lead to increased access to therapies and potentially lower c
11、osts to patients.Our work in reviewing and approving new therapies includes thorough safety examinations.New drugs must not only meet our rigorous premarket safety standards,but also undergo postmarket safety surveillance.We will discuss our comprehensive safety activities in a different report.This
12、 report summarizes CDERs 2021 approvals and highlights examples of innovative treatments.FDAs Center for Biologics Evaluation and Research(CBER)also approves important therapies to advance and protect public health.Perhaps most significantly in 2021,CBER approved a COVID-19 vaccine,a milestone in ou
13、r continued fight against this pandemic.For more information about CBER actions,please visit CBERs webpage for 2021 Biological Approvals.In closing,we hope the following overview of new drug therapy approvals will promote greater understanding of the CDER mission to improve treatment options for pat
14、ients.Patrizia Cavazzoni,M.D.,Director,Center for Drug Evaluation and Research6Advancing Health Through Innovation6Executive Summary In 2021,CDER approved many different drug therapies,helping patients have a better quality of life,reducing disease symptoms or severity,and in many instances,protecti
15、ng patients against life-threatening illnesses.Heart,Blood,Kidney,and Endocrine Diseases In 2021,CDER took important steps in advancing treatment options for patients with diabetes.CDER approved several diabetes medications,including one interchangeable biosimilar insulin product and one biosimilar
16、insulin product,which can provide patients with additional safe,high-quality,and potentially more cost-effective options.CDER also approved a treatment for pediatric patients ages 10 and older with type 2 diabetes and a therapy for severe hypoglycemia,or low blood sugar,in patients aged six years an
17、d older with diabetes.We approved a therapy to reduce the risk of serious complications in adults with chronic kidney disease associated with type 2 diabetes.In addition,we approved a treatment to reduce the risk of serious outcomes in adults with chronic kidney disease at risk of chronic kidney dis
18、ease progression.For patients with obesity and overweight,CDER approved a new add-on therapy for chronic weight management in certain adults the first drug approved for this indication since 2014 and a tool that may help combat our nations obesity epidemic.A treatment was also approved to reduce the
19、 frequency of chemotherapy-induced bone marrow suppression in adults with types of small cell lung cancer.Drug approvals for rare diseases in this area include:1)several add-on therapies to treat certain patient populations with homozygous familial hypercholesterolemia,a life-threatening condition c
20、haracterized by severely high cholesterol levels;2)a treatment See page 10 for a complete list of these therapies.7New Drug Therapy Approvals 2021for paroxysmal nocturnal hemoglobinuria,a rare and serious blood disease;3)the first treatment to increase height in people with achondroplasia,the most c
21、ommon type of dwarfism;4)a treatment for polycythemia vera,a rare chronic disease involving circulatory risks caused from the overproduction of red blood cells;and 5)a treatment for children aged 4-11 years with sickle cell disease,an inherited disorder characterized by dysfunctional red blood cells
22、.Autoimmune,Inflammatory,and Lung Diseases 2021 was an important year for treatments for inflammatory bowel disease(IBD),or disorders that involve digestive tract inflammation.For example,CDER approved a therapy for adults with moderately to severely active ulcerative colitis,a type of IBD in which
23、the lining of the colon becomes inflamed and develops tiny open sores,or ulcers.CDER also approved a therapy for pediatric patients with moderately to severely active ulcerative colitis the first therapy in this biologics class for this patient population that can be administered at home rather than
24、 at an infusion center.On the biosimilars front,CDER approved an interchangeable biosimilar and a biosimilar to treat ulcerative colitis in adults,Crohns disease(another type of IBD that affects the lining of the digestive tract)and other inflammatory diseases,including different types of arthritis.
25、In 2021,CDER also approved a treatment for systemic lupus erythematosus(SLE).SLE is the most common form of lupus,a disease in which the immune system attacks the bodys own tissues,causing inflammation and tissue damage.In addition,CDER approved a therapy for adults with active lupus nephritis,a ser
26、ious kidney disease associated with lupus.CDER approved a drug to treat pruritus(itchiness)that can occur in patients with chronic kidney disease.CDER also approved two new therapies to treat moderate-to-severe atopic dermatitis(eczema)that has not responded to other therapies.Approvals of note to t
27、reat rare diseases in this area include:1)a therapy for children aged 6-11 years with the most common mutation for cystic fibrosis,a progressive disorder that causes severe lung damage and limits the ability to breathe;2)a therapy to prevent organ rejection in patients receiving lung transplants;3)a
28、 treatment for systemic sclerosis-associated interstitial lung disease,a multi-organ autoimmune disorder characterized by inflammation,vascular injury and tissue scarring;4)a medication to treat anti-neutrophil cytoplasmic antibody-associated vasculitis,a group of diseases characterized by destructi
29、on and inflammation of small vessels;and 5)drugs to treat pruritus associated with rare liver disorders,often occurring in children.2021 was an important year for treatments for inflammatory bowel disease.8Advancing Health Through InnovationInfectious Diseases In 2021,CDER continued to help advance
30、the treatment and prevention of HIV-1 infection,the most common type of HIV.Early in the year,we approved the first extended-release injectable drug that functions as a complete regimen,administered monthly,to treat HIV-1 infection in adults.We also approved a combination drug product,administered a
31、s a single tablet and taken once a day,to treat HIV-1 infection in children aged two years or older.By years end,CDER approved the first extended-release,injectable,antiviral drug administered every two months,to prevent HIV-1 infection in adults and adolescents.Also in the antiviral area,CDER appro
32、ved a drug to treat human smallpox disease in adult and pediatric patients,including newborn babies.Although the World Health Organization has declared smallpox eradicated,there have been longstanding concerns that smallpox virus could be used as a bioweapon.This approval is therefore an important m
33、edical countermeasure.CDER also approved the first antiviral drug to treat adults and adolescents with post-transplant cytomegalovirus(CMV)infection or disease that does not respond to other antiviral drugs.This approval is an important advancement in treating CMV infection or disease,where there ha
34、s been an unmet medical need for patients receiving transplants.For patients with rare infectious diseases,CDER approved the first all-oral treatment for sleeping sickness(Human African trypanosomiasis),a disease caused by parasites transmitted by infected tsetse flies and endemic in sub-Saharan Afr
35、ica.Other sleeping sickness therapies include oral components taken together with non-oral drugs.Neurological and Psychiatric Disorders To help address the opioid crisis,we approved two therapies to reverse an opioid overdose in an emergency situation.Using the accelerated approval pathway,CDER appr
36、oved a new drug to treat Alzheimers disease.This drug is the first new treatment approved for Alzheimers disease since 2003 and the first therapy that targets the fundamental disease pathophysiology.CDER also approved two drugs that help reduce the frequency of migraine attacks in patients with epis
37、odic migraines.Approvals of note for rare neurological disorders include:1)a treatment for certain patients with Duchenne muscular dystrophy,a genetic disorder occurring primarily in young boys that leads to progressive muscle degeneration and weakness;and 2)a treatment for myasthenia gravis,a chron
38、ic neuromuscular disease.2021 marked an important year for new HIV-1 treatments and preventive therapies.9New Drug Therapy Approvals 2021Cancers2021 marked the 50th anniversary of the National Cancer Act,landmark legislation committed to fighting cancer.In this anniversary year,CDER approved many th
39、erapies for different cancers.Four new drugs were approved for non-small cell lung cancer,including one non-small cell lung cancer type previously thought to be resistant to treatment.CDER also approved a therapy for some types of basal cell carcinoma,the most common form of skin cancer,for certain
40、patient populations.The first approved immunotherapy to be used as a first-line treatment for esophageal(esophagus-related)cancer,gastric(stomach)cancer,and gastroesophageal junction(GEJ)adenocarcinoma was an important cancer approval for 2021.CDER approved two other therapies for certain patients w
41、ith HER2-positive gastric cancer and GEJ adenocarcinoma;one of these therapies was also approved for esophageal cancer,advanced kidney cancer and as an adjuvant treatment for kidney cancer.With regard to breast cancer,CDER approved a therapy for early-stage,triple negative breast cancer,or cancer th
42、at does not respond to hormone therapies or medications that target HER2 protein receptors.Other CDER approvals for rare cancers include:1)two treatments for adults with certain kinds of cholangiocarcinoma,a group of aggressive cancers that start in the bile duct;2)a therapy for adults to treat cert
43、ain tumors that are associated with von Hippel-Lindau disease,an inherited disorder characterized by tumors and cysts;3)a therapy to be used together with other treatments for light chain amyloidosis,a condition that interferes with normal organ functions due to abnormal protein build-up;and 4)the f
44、irst FDA-approved treatment for locally advanced unresectable or metastatic perivascular epithelioid cell tumor(PEComa),a group of rare tumors that form in the soft tissues of the stomach,intestines,lungs and other body parts.(Note that FDAs Oncology Center of Excellence will produce its 2021 annual
45、 report to feature important cancer therapies approved in 2021 by CDER,CBER,and FDAs Center for Devices and Radiological Health CDRH.)Other Advances in Drug Therapies In other advances of 2021,CDER approved 1)a drug for late-onset Pompe disease,a rare disease that causes muscle weakness and prematur
46、e death;and 2)a medication to treat neurogenic detrusor overactivity,a bladder dysfunction,in children.CDERs drug approvals of 2021 will help advance patient care in many new areas.In 2021,CDER approved four new therapies for non-small cell lung cancer.10Advancing Health Through InnovationCDERs Drug
47、 Approvals of 2021 In 2021,CDER approved 50 new drugs,either as new molecular entities(NMEs)under New Drug Applications(NDAs),or as new therapeutic biologics under Biologics License Applications(BLAs).The active ingredient or ingredients in a new drug have never before been approved in the U.S.The n
48、ovel drugs CDER approved in 2021 are collectively notable for their potential positive impact and unique contributions to patient care.CDERs novel drug approvals for 2021 are listed alphabetically below*See Appendix B for a summary chart of designations for CDERs novel approvals.*This information is
49、 accurate as of December 31,2021.In rare instances FDA may need to change a drugs NME designation or the status of its application as a novel BLA.For instance,new information may become available that could lead to a reconsideration of the original designation or status.If FDA makes these types of c
50、hanges,the agency intends to communicate the nature of,and the reason for,any revisions as appropriate.*Product approved with no trade nameTrade NameActive Ingredient(s)Adbrytralokinumab-ldrmAduhelmaducanumab-avwaAmondys 45casimersenAzstarysserdexmethylphenidate and dexmethylphenidateBesremiropegint
51、erferon alfa-2b-njftBrexafemmeibrexafungerp BylvayodevixibatCabenuvacabotegravir and rilpivirine (co-packaged)CoselatrilacicilibCytaluxpafolacianine EmpavelipegcetacoplanEvkeezaevinacumab-dgnbExkivitymobocertinibfexinidazole*FotivdativozanibJemperlidostarlimab-gxlyKerendiafinerenoneKorsuvadifelikefa
52、linLeqvioinclisiranLivmarlimaralixibatLivtencity maribavir LumakrassotorasibLupkynisvoclosporinLybalviolanzapine and samidorphanNextstellisdrospirenone and estetrol Trade NameActive Ingredient(s)Nexviazymeavalglucosidase alfa-ngptNulibryfosdenopterinPepaxtomelphalan flufenamidePonvoryponesimodPylari
53、fypiflufolastat F 18 QelbreeviloxazineQuliptaatogepantRezurockbelumosudilRybrevantamivantamab-vmjwRylazeasparaginase erwinia chrysanthemi(recombinant)-rywnSaphneloanifrolumab-fniaScemblixasciminibSkytrofalonapegsomatropin-tcgdTavneosavacopanTepmetkotepotinibTezspiretezepelumab-ekkoTivdaktisotumab ve
54、dotin-tftvTruseltiqinfigratinibUkoniqumbralisibVerquvovericiguatVoxzogovosoritideVyvgartefgartigimod alfa-fcabWeliregbelzutifanZegaloguedasiglucagonZynlontaloncastuximab tesirine-lpyl11New Drug Therapy Approvals 2021First-in-Class Drugs CDER identified 27 of the 50 novel drugs approved in 2021(54%)a
55、s first-in-class.These drugs have mechanisms of action different from those of existing therapies.Novel drugs approved in 2021 that CDER identified as first-in-class were:Adbry,Aduhelm,Besremi,Brexafemme,Bylvay,Cosela,Cytalux,Empaveli,Evkeeza,Kerendia,Korsuva,Leqvio,Livtencity,Lumakras,Lupkynis,Nuli
56、bry,Rezurock,Rybrevant,Saphnelo,Tavneos,Tezspire,Tivdak,Verquvo,Voxzogo,Vyvgart,Welireg,ZynlontaExamples of notable First-in-Class novel approvals for 2021 include:Adbry(tralokinumab-ldrm)injection see also Opzelura on page 32 to treat moderate-to-severe atopic dermatitis(eczema)in adults whose dise
57、ase cannot be adequately controlled by therapies applied directly on the skin.Aduhelm(aducanumab-avwa)injection to treat Alzheimers disease.This drug is the first therapy for Alzheimers that targets the fundamental disease pathophysiology.CDER identified 27 of the 50(54%)novel drugs approved in 2021
58、 as first-in-class.54%First-in-Class Drugs CDERs Annual Novel Drug Approvals:20122021The 10-year graph below shows that from 2012 through 2021,CDER has averaged 43 novel drug approvals per year.Quantity605550 454035302520145465948Calendar Year2253502012 2013 2014 2015 2016 2017 2018 2019
59、2020 202112Advancing Health Through Innovation Brexafemme(ibrexafungerp)tablets to treat vulvovaginal candidiasis,a type of yeast infection.This is the first approved drug in a new antifungal class in more than 20 years.Cosela(trilaciclib)injection to reduce the frequency of chemotherapy-induced bon
60、e marrow suppression in certain adults treated for extensive-stage small cell lung cancer.Evkeeza(evinacumab-dgnb)see also Praluent on page 24 and Repatha on page 26 injection as an add-on treatment for patients aged 12 years and older with homozygous familial hypercholesterolemia,a genetic conditio
61、n that causes severely high cholesterol.Leqvio(inclisiran)see Repatha on page 26 injection to treat heterozygous familial hypercholesterolemia,a ge-netic condition that causes severely high cholesterol,and clinical atherosclerotic cardiovascular disease,which involves cholesterol buildup in the arte
62、ries.Livtencity(maribavir)capsules to treat adults with post-transplant cytomegalovirus(CMV)infection.CMV is a common virus that doesnt normally cause any symptoms.However,people with weakened immune systems,including patients receiving transplants,can develop severe and life-threatening complicatio
63、ns from CMV.Lupkynis(voclosporin)capsules to treat adults with active lupus nephritis,a serious kidney disease and complication of lupus,a disease in which the immune system attacks the bodys own tissues.Kerendia(finerenone)tablets to reduce the risk of several serious complications in adults with c
64、hronic kidney disease associated with type 2 diabetes.Korsuva(difelikefalin)injection to treat moderate-to-severe pruritus(itching)associated with chronic kidney disease in adults undergoing hemodialysis.Rezurock(belumosudil)tablets to treat patients with chronic graft-versus-host disease after fail
65、ing at least two previous systemic(throughout the body)therapies.Rybrevant(amivantamab-vmjw)injection to treat adults with certain forms of non-small cell lung cancer whose tumors have specific mutations.Rybrevant is the first FDA-approved treatment for this subset of non-small cell lung cancer and
66、was approved through the accelerated approval pathway.13New Drug Therapy Approvals 2021 Saphnelo(anifrolumab-fnia)injection to treat systemic lupus erythematosus(SLE).SLE is the most common type of lupus,a disease in which the immune system attacks the bodys own tissues.Tezspire(tezepelumab)injectio
67、n as an add-on treatment for patients aged 12 and older with severe asthma.Welireg(belzutifan)tablets for adults who require treatment for certain tumors associated with von Hippel-Lindau disease.These tumors include renal cell carcinoma,central nervous system hemangioblastomas,or pancreatic neuroen
68、docrine tumors that do not require immediate surgery.Drugs in brackets refer to other treatments for the same or similar disease.Drugs for Orphan DiseasesIn 2021,26 of CDERs 50 novel drug approvals(52%)were approved to treat rare or“orphan”diseases(diseases that affect than 200,000 people in the U.S
69、.).Patients with rare diseases often have fewer or no drugs available to treat their conditions.Novel drugs approved in 2021 with the orphan drug designation were:Amondys 45,Besremi,Bylvay,Cytalux,Empaveli,Evkeeza,Exkivity,fexinidazole*,Livmarli,Livtencity,Lumakras,Nexviazyme,Nulibry,Pepaxto,Rezuroc
70、k,Rylaze,Scemblix,Skytrofa,Tavneos,Tepmetko,Truseltiq,Ukoniq,Voxzogo,Vyvgart,Welireg,Zynlonta*approved without a trade nameNotable examples of novel approvals of 2021 for orphan diseases include:Besremi(ropeginterferon alfa-2b-njft)injection to treat adults with polycythemia vera,a blood disease tha
71、t causes the overproduction of red blood cells.The excess cells thicken the blood,slowing blood flow and increasing the chance of blood clots.Bylvay(odevixibat)capsules and oral pellets to treat pruritus(itching)in progressive familial intrahepatic cholestasis(PFIC),a disorder that causes progressiv
72、e liver disease and typically leads to liver failure.This is the first approved treatment for pruritus in patients with PFIC.26(52%)of CDERs 50 novel drug approvals were for rare or orphan diseases.52%Orphan Drug Approvals14Advancing Health Through Innovation Cytalux(pafolacianine)injection to help
73、identify ovarian cancer lesions during surgery.Ovarian cancer is hard to detect early,making it difficult to successfully treat.Empaveli(pegcetacoplan)injection to treat adults with paroxysmal nocturnal hemoglobinuria,a rare,life-threatening blood disease.fexinidazole(no trade name)tablets to treat
74、sleeping sickness(Human African trypanosomiasis),a life-threatening disease caused by parasites transmitted by infected tsetse flies that is endemic in many sub-Saharan African countries.Fexinidazole is the first all-oral treatment for sleeping sickness.(Other treatments include oral components take
75、n together with non-oral drugs.)Livmarli(maralixibat)oral solution to treat cholestatic(liver-related)pruritus in Alagille syndrome,an inherited condition in which bile builds up in the liver.Lumakras(sotorasib)tablets to treat adults with a specific type of non-small cell lung cancer that is locall
76、y advanced or metastatic.Adults must have received a prior systemic(throughout the body)therapy.The mutation(KRAS G12C)involved in this cancer was previously considered resistant to drug therapy.Nexviazyme(avalglucosidase alfa-ngpt)infusion to treat patients one year and older with late-onset Pompe
77、disease,a disease characterized by an enzyme deficiency that causes muscle weakness and premature death from respiratory or heart failure.Nulibry(fosdenopterin)injection to reduce the risk of death due to molybdenum cofactor deficiency type A,an inherited disorder that typically presents in the firs
78、t few days of life,causing hard-to-control seizures,brain injury,and death.Nulibry is the first approved therapy for this disease.Rylaze(asparaginase erwinia chrysanthemi(recombinant)-rywn)injection as a component of a chemotherapy regimen to treat acute lymphoblastic leukemia,the most common type o
79、f childhood cancer,and lymphoblastic lymphoma in patients who are allergic to the E.coli-derived asparaginase products used most commonly for treatment.The only other FDA-approved drug for patients with allergic reactions has been in global shortage for years.Skytrofa(lonapegsomatropin-tcgd)injectio
80、n to treat pediatric patients with growth hormone deficiency.Patients can take Skytrofa weekly;other approved human 15New Drug Therapy Approvals 2021growth hormone formulations for this indication must be administered daily.Tavneos(avacopan)capsules to treat anti-neutrophil cytoplasmic antibody-asso
81、ciated vasculitis,a group of diseases characterized by destruction and inflammation of small vessels.The diseases can affect organs and cause skin lesions.Other Novel Drug Approvals In addition to the first-in-class and orphan-designated drugs,the 2021 novel drug field also includes these notable ap
82、provals:Cabenuva(cabotegravir and rilpivirin)see also Vocabria on page 32 and Apretude on page 30 injectable formulation as the first monthly complete regimen to treat HIV.Jemperli(dostarlimab)injection to treat a specific genetic form of recurrent or advanced endometrial cancer,or cancer that begin
83、s in the uterus,that has progressed despite other specific treatments.Jemperli was approved under the accelerated approval pathway.Nextstellis(drospirenone and estetrol)oral contraceptive tablets to prevent pregnancy.Qelbree(viloxazine)capsules to treat attention deficit hyperactivity disorder(ADHD)
84、in patients aged six to 18.Qelbree is one of a few approved nonstimulant medications for ADHD.Qulipta(atogepant)see also Nurtec ODT on page 24 tablets to help reduce the frequency of migraine attacks in patients with episodic migraines.Verquvo(vericiguat)tablets to reduce the risk of cardiovascular
85、death and heart failure hospitalization in certain high-risk patients.Zegalogue(dasiglucagon)injection to treat severe hypoglycemia,or low blood sugar,in patients aged six years and older with diabetes.Drugs in brackets refer to other treatments for the same or similar disease.In 2021,CDER approved
86、a therapy to treat patients with recurrent or advanced endometrial cancer.16Advancing Health Through InnovationInnovation:Use of Expedited Development and Review PathwaysCDER used diverse regulatory pathways to enhance and expedite drug product review in 2021.These pathways facilitate increased flex
87、ibility,efficiency,and interactions between CDER staff and drug developers.These pathways also allow shorter review times to speed the availability of new therapies to patients with serious conditions,especially when there are no satisfactory alternative therapies,while preserving appropriate standa
88、rds for safety and effectiveness.Fast TrackCDER designated 18 of the 50 novel drugs(36%)in 2021 as Fast Track status.Fast Track speeds new drug development and review by increasing the level of communication between FDA and drug developers and by enabling CDER to review portions of a drug applicatio
89、n on a rolling basis.Drugs designated with Fast Track status were:Aduhelm,Amondys 45,Brexafemme,Bylvay,Cabenuva,Cytalux,Empaveli,Exkivity,Kerendia,Lumakras,Lupkynis,Nexviazyme,Rylaze,Saphnelo,Scemblix,Truseltiq,Verquvo,VyvgartCDER designated 18 of the 50 novel drugs(36%)as Fast Track.36%Fast Track16
90、17Innovation:Use of Expedited Development and Review PathwaysBreakthrough TherapyCDER designated 14 of the 50 novel drugs(28%)in 2021 as Breakthrough Therapies.A Breakthrough Therapy designation includes all the Fast Track program features and offers more intensive FDA guidance on efficiencies for d
91、rug development.Drugs designated with Breakthrough therapy status were:Cosela,Evkeeza,Exkivity,Jemperli,Korsuva,Livmarli,Livtencity,Lumakras,Nexviazyme,Nulibry,Rezurock,Rybrevant,Scemblix,Ukoniq Priority ReviewIn 2021,34 of the 50 novel drugs approved(68%)were designated Priority Review.A drug recei
92、ves a Priority Review if CDER determines that the drug could potentially provide a significant advance in medical care.This means CDER aims to take action on a drug application within six months of filing(compared to 10 months under standard review).In some instances,sponsors may redeem a priority r
93、eview voucher under CDERs Priority Review Voucher program.Such drugs are not included in the list below.Drugs designated Priority Review were:Aduhelm,Amondys 45,Brexafemme,Bylvay,Cabenuva,Cosela,Cytalux,Empaveli,Evkeeza,Exkivity,fexinidazole*,Jemperli,Kerendia,Korsuva,Livmarli,Livtencity,Lumakras,Lu
94、pkynis,Nexviazyme,Nulibry,Pepaxto,Pylarify,Rezurock,Rybrevant,Scemblix,Tepmetko,Tezspire,Tivdak,Truseltiq,Ukoniq,Verquvo,Voxzogo,Welireg,Zynlonta*approved with no trade name CDER identified 34 of the 50(68%)drugs approved in 2021 as priority review.68%Priority ReviewCDER identified 14 of the 50(28%)
95、novel drugs of 2021 as Breakthrough Therapies.28%Breakthrough Therapy18Advancing Health Through InnovationAccelerated ApprovalCDER approved 14 of the 50 novel drugs(28%)in 2021 under Accelerat-ed Approval.This program aims to bring drugs that can provide import-ant treatment advances sooner to marke
96、t than with traditional approv-als.It provides FDA more flexibility in what endpoints can be used to approve a drug that offers a benefit over current treatments for a serious or life-threatening illness.These accelerated approval endpoints may include those that are reasonably likely to predict cli
97、nical benefit,which may enable the drug to show benefits over a shorter treatment duration(whereas longer-term demonstration of benefit is needed for traditional approval).Subsequent confirmatory trials must be conducted to support traditional approval.The Novel drugs approved in 2021 via Accelerate
98、d Approval were:Aduhelm,Amondys 45,Exkivity,Jemperli,Lumakras,Pepaxto,Rybrevant,Scemblix,Tepmetko,Tivdak,Truseltiq,Ukoniq,Voxzogo,ZynlontaOverall Use of Expedited Development and Review MethodsThirty-seven of the 50 novel drug approvals of 2021(74%)used one or more expedited programs,specifically Fa
99、st Track Designation,Breakthrough Therapy Designation,Priority Review,and/or Accelerated Approval.Novel drugs approved in 2021 that used at least one expedited program were:Aduhelm,Amondys 45,Brexafemme,Bylvay,Cabenuva,Cosela,Cytalux,Empaveli,Evkeeza,Exkivity,fexinidazole*,Jemperli,Kerendia,Korsuva,
100、Livmarli,Livtencity,Lumakras,Lupkynis,Nexviazyme,Nulibry,Pepaxto,Pylarify,Rezurock,Rybrevant,Rylaze,Saphnelo,Scemblix,Tepmetko,Tezspire,Tivdak,Truseltiq,Ukoniq,Verquvo,Voxzogo,Vyvgart,Welireg,Zynlonta*approved with no trade name CDER identified 14 of the 50(28%)novel drugs as Accelerated Approvals.2
101、8%Accelerated ApprovalsCDER used at least one expedited program to speed approval for 74%of all novel drugs approved in 2021.19Innovation:Use of Expedited Development and Review PathwaysPredictability:Meeting PDUFA GoalsUnder the Prescription Drug User Fee Act(PDUFA),industry are assessed user fees.
102、With PDUFA,applications are reviewed targeting specific timeframes.Throughout 2021,CDER met or exceeded the PDUFA goal date for taking action on 98%(49 of 50)of the novel drugs approved.Novel drugs approved in 2021 on or before their PDUFA goal dates were:Adbry,Aduhelm,Amondys 45,Azstarys,Besremi,Br
103、exafemme,Bylvay,Cabenuva,Cosela,Cytalux,Empaveli,Evkeeza,Exkivity,fexinidazole*,Fotivda,Kerendia,Korsuva,Leqvio,Livmarli,Livtencity,Lumakras,Lupkynis,Lybalvi,Nextstellis,Nexviazyme,Nulibry,Pepaxto,Ponvory,Pylarify,Qelbree,Qulipta,Rezurock,Rybrevant,Rylaze,Saphnelo,Scemblix,Skytrofa,Tavneos,Tepmetko,
104、Tezspire,Tivdak,Truseltiq,Ukoniq,Verquvo,Voxzogo,Vyvgart,Welireg,Zegalogue,Zynlonta*approved with no trade nameCDER met its PDUFA goal date for 98%of the novel drugs approved in 2021.In 2021,49(98%)of 50 novel drugs were approved on or before their PDUFA goal date.98%Meeting PDUFA Goals1920Advancing
105、 Health Through InnovationAccess:First Cycle Approval and First in U.S.ApprovalsFirst Cycle Approvals CDER approved 43 of the 50 novel drugs of 2021(86%)on the“first cycle”of review.This high percentage reflects the extent to which CDER staff provide clarity to drug developers on the necessary study
106、 design elements and other data needed in the drug application to support a full and comprehensive drug assessment.Novel drugs approved in 2021 on the first cycle were:Aduhelm,Amondys 45,Azstarys,Brexafemme,Bylvay,Cosela,Cytalux,Empaveli,Evkeeza,Exkivity,Fotivda,Jemperli,Kerendia,Korsuva,Livmarli,Li
107、vtencity,Lumakras,Lupkynis,Nextstellis,Nexviazyme,Nulibry,Pepaxto,Ponvory,Pylarify,Qulipta,Rezurock,Rybrevant,Rylaze,Saphnelo,Scemblix,Skytrofa,Tavneos,Tepmetko,Tezspire,Tivdak,Truseltiq,Ukoniq,Verquvo,Voxzogo,Vyvgart,Welireg,Zegalogue,Zynlonta The high percentage of first cycle approvals reflects t
108、he extent to which CDER staff provide clarity to drug developers.20CDER approved 43 of the 50 novel drugs of 2021(86%)on the first cycle.First Cycle Approvals86%21Access:First Cycle Approval and Approvals Compared to Other Countries38 of the 50 novel drugs(76%)approved in 2021 were first approved in
109、 the U.S.76%First in the U.S.76%of the novel drugs approved in 2021 were approved in the U.S.before any other country.Approval in the U.S.Before Other CountriesThirty-eight of the 50 novel drugs approved in 2021(76%)were approved in the U.S.before any other country.Novel drugs of 2021 approved first
110、 in the United States were:Aduhelm,Amondys 45,Azstarys,Brexafemme,Cosela,Cytalux,Empaveli,Evkeeza,Exkivity,Jemperli,Kerendia,Korsuva,Livmarli,Livtencity,Lumakras,Lupkynis,Lybalvi,Nexviazyme,Nulibry,Pepaxto,Ponvory,Pylarify,Qulipta,Rezurock,Rybrevant,Rylaze,Saphnelo,Scemblix,Skytrofa,Tezspire,Tivdak,
111、Truseltiq,Ukoniq,Verquvo,Vyvgart,Welireg,Zegalogue,Zynlonta22Advancing Health Through Innovation2021s Novel Drug Approvals Expedited Review Programs 28%Cosela Evkeeza Exkivity Jemperli Korsuva Livmarli Livtencity Lumakras Nexviazyme Nulibry Rezurock Rybrevant Scemblix Ukoniq Breakthrough Therapy(14
112、of 50)68%Aduhelm Amondys 45 Brexafemme Bylvay Cabenuva Cosela Cytalux Empaveli Evkeeza Exkivity fexinidazole*Jemperli Kerendia Korsuva Livmarli Livtencity Lumakras Lupkynis Nexviazyme Nulibry Pepaxto Pylarify Rezurock Rybrevant Scemblix Tepmetko Tezspire Tivdak Truseltiq Ukoniq Verquvo Voxzogo Welir
113、eg ZynlontaPriority Review(34 of 50)Aduhelm Amondys 45 Brexafemme Bylvay Cabenuva Cosela Cytalux Empaveli Evkeeza Exkivity fexinidazole*Jemperli Kerendia Korsuva Livmarli Livtencity Lumakras Lupkynis Nexviazyme Nulibry Pepaxto Pylarify Rezurock Rybrevant Rylaze Saphnelo Scemblix Tepmetko Tezspire Ti
114、vdak Truseltiq Ukoniq Verquvo Voxzogo Vyvgart Welireg ZynlontaUsed One or More Expedited Program(37 of 50)74%Aduhelm Amondys 45 Brexafemme Bylvay Cabenuva Cytalux Empaveli Exkivity Kerendia Lumakras Lupkynis Nexviazyme Rylaze Saphnelo Scemblix Truseltiq Verquvo VyvgartFast Track(18 of 50)36%Aduhelm
115、Amondys 45 Exkivity Jemperli Lumakras Pepaxto Rybrevant Scemblix Tepmetko Tivdak Truseltiq Ukoniq Voxzogo ZynlontaAccelerated Approval(14 of 50)28%Other Key MeasuresTotal Novel Drugs Approved in 2021100%90%(48 of 48)00Approved First in U.S.First Cycle ApprovalMet PDUFAGoal DateOrphan Drug
116、First-In-Class98%54%52%86%76%(49 of 50)(27 of 50)(26 of 50)(38 of 50)(43 of 50)23New Uses of CDER-Approved Drugs Actemra(tocilizumab)injection.Originally approved to treat rheumatoid arthritis,Actemra was approved in 2021 to slow the rate of decline in pulmonary function in adults with systemic scle
117、rosis-associated interstitial lung disease,an autoimmune disorder characterized by inflammation,vascular injury,and tissue scarring.Ayvakit(avapritinib)tablets were originally approved to treat advanced or inoperable gastrointestinal stromal tumors in patients with a certain mutation.In 2021,CDER ap
118、proved Ayvakit to treat adults with advanced systemic mastocytosis,a group of rare disorders in which too many white blood cells known as“mast”cells build up in the body.Cabometyx(cabozantinib)tablets,which were originally approved in 2016,were approved in 2021 for patients undergoing treatment for
119、advanced renal(kidney)cell carcinoma and for patients aged 12 years and older with locally advanced or metastatic differentiated thyroid cancer that has progressed after prior therapy.Carbaglu(carglumic acid)tablets were originally approved as an add-on or maintenance therapy to treat patients with
120、high blood levels of ammonia.In 2021,Carbaglu became the first approved treatment for hyperammonemia due to propionic acidemia(PA)or methylmalonic acidemia(MMA).PA and MMA are rare inherited metabolic disorders.Darzalex Faspro(daratumumab and hyaluronidase-fihj),injection,originally approved in 2020
121、,was approved in 2021 to help treat patients newly diagnosed with light chain amyloidosis,a condition that interferes with normal organ functions due to abnormal protein build-up.Enhertu(fam-trastuzumab deruxtecan-nxki)injection,which was first approved in 2019,was approved in 2021 for adults with l
122、ocally advanced or metastatic HER2-positive gastric(stomach)or gastroesophageal junction(lower part of the esophagus that connects to the stomach)adenocarcinoma who have received a certain prior therapy.After CDER approves a new treatment,a drug sponsor may generate new data about the approved produ
123、ct that suggests an additional use.The drug sponsor may then submit an application to modify or expand the use of an approved drug based on this new data.The products below are some notable approvals of 2021 for new uses or indications of an approved drug:New Uses of Approved Drugs 24Advancing Healt
124、h Through Innovation Farxiga(dapagliflozin)oral tablets were originally approved to help improve glycemic control in adults with type 2 diabetes.In 2021,CDER approved Farxiga to reduce the risk of kidney function decline,kidney failure,cardiovascular death,and hospitalization for heart failure in ad
125、ults with chronic kidney disease who are at risk of disease progression.Keytruda(pembrolizumab)injection,which was first approved in 2014,was approved for new or expanded indications in 2021,either to be used alone or in combination with other therapies.These indications include:certain types of eso
126、phageal or gastroesophageal junction carcinoma;some kinds of gastric or gastroesophageal junction adenocarcinoma;locally advanced cutaneous squamous cell carcinoma(a type of skin cancer)that is not curable by surgery or radiation;advanced endometrial(lining of the uterus)cancer;early-stage,triple ne
127、gative breast cancer;advanced kidney cancer;some stages of melanoma;and certain types of cervical cancer.Myrbetriq(mirabergon)extended-release tablets,approved to treat certain patients with overactive bladder,was approved in 2021 to treat neurogenic detrusor overactivity(a neurological impairment o
128、f bladder function)in patients aged three years and older.Nurtec ODT(rimegepant)see also Qulipta on page 15 orally disintegrating tablets,originally approved to treat acute migraine,received approval in 2021 to help reduce the frequency of migraine attacks in patients with episodic migraines.Opdivo(
129、nivolumab)injection,which was first approved in 2014,was approved in 2021 as a first-line treatment for gastric cancer,esophageal cancer in certain patients,and as an adjuvant treatment for patients with high-risk urothelial carcinoma,a type of bladder cancer.Praluent(alirocumab)see also Evkeeza on
130、page 12 and Repatha on page 26 injection,originally approved in 2015,was approved in 2021 to treat homozygous familial hyperfamilial hypercholesterolemia,a genetic condition that causes severely high cholesterol.Prograf(tacrolimus)capsules were previously approved to prevent organ rejection in patie
131、nts receiving liver,kidney,and heart transplants.In 2021,Prograf was approved as the first immunosuppressant drug to prevent organ rejection in patients receiving lung transplants.25New Uses of CDER-Approved Drugs Solosec(secnidazole)oral granules were originally approved to treat a bacterial type o
132、f vaginal inflammation.In 2021,CDER approved the drug to treat trichomoniasis,a common sexually transmitted disease.Tecentriq(atezolizumab)injection,which was originally approved for certain patients with locally advanced or metastatic urothelial carcinoma,was approved in 2021 as an adjuvant treatme
133、nt following surgical removal and platinum-based chemotherapy in patients with stage II to IIIa non-small cell lung cancer with specific tumor characteristics.Xalkori(crizotinib)capsules,which were first approved in 2011 for patients with certain types of non-small cell lung cancer,were approved in
134、2021 to treat systemic anaplastic large cell lymphoma that is ALK-positive in pediatric patients aged one year and older,and certain young adults who have had a history of the disease.“ALK-positive”refers to a certain genetic constitution.Zeposia(ozanimod)see also Humira on page 26 capsules were app
135、roved in 2020 to treat relapsing forms of multiple sclerosis.In 2021,CDER approved Zeposia to treat moderately to severely active ulcerative colitis in adults.Drugs in brackets refer to other treatments for the same or similar disease.Approved Drugs Expanded for New Pediatric Populations The Pediatr
136、ic Research Equity Act(PREA),which gives FDA the authority to require pediatric studies under certain circumstances,has been largely responsible for the inclusion of pediatric information in the labeling for many drugs.Upon drug approval,FDA may require pediatric studies of that drug under PREA.In r
137、esponse to that requirement,sponsors may submit new data to support the safe and effective use of the drug in the pediatric population studied.Sponsors submit this data in an application to expand the patient population.Biktarvy(bictegravir,emtricitabine,and tenofovir alafenamide)tablets were origin
138、ally approved in 2018 to treat certain patients with HIV.In 2021,CDER extended the patient population down to ages two years and older.Bydureon and Bydureon BCise(exenatide extended-release)injection was approved in 2012 to help improve blood sugar levels in adults with type 2 diabetes.In 2021,CDER
139、extended the indication to include pediatric patients aged 10 years and older.26Advancing Health Through Innovation Epclusa(sofosbuvir/velpatasvir),see also Mavyret,below tablets and oral pellets,were originally approved by CDER to treat certain adults with chronic hepatitis C virus(HCV)genotypes 1,
140、2,3,4,5,and 6.In 2021,CDER approved Epclusa to treat children aged three years and older with any of the six HCV genotypes without cirrhosis(liver disease)or with mild cirrhosis.HCV causes liver inflammation that can lead to diminished liver function or liver failure.Humira(adalimumab)see also Zepos
141、ia on page 25 injection,first approved in 2002,is now approved for indications including rheumatoid arthritis,Crohns disease,ulcerative colitis,and psoriasis.In 2021,Humira was approved to treat moderately to severely active ulcerative colitis in pediatric patients aged five years and older.This is
142、the first pediatric treatment for moderately to severely active ulcerative colitis that can be administered at home rather than at an infusion center.Mavyret(glecaprevir and pibrentasvir)see also Epclusa,above tablets were first approved in 2017 to treat patients older than age 12 with certain types
143、 of HCV.In 2021,CDER extended the age group down to three years for the same indications.Mavyret is another hepatitis C treatment regimen available to treat all six genotypes of HCV.Rapivab(peramivir)injection was originally approved in 2014 to treat adults with acute uncomplicated influenza(flu).In
144、 2021,CDER extended the indication to include patients six months and older.Repatha(evolocumab)see Evkeeza and Leqvio on page 12 and Praluent on page 24 injection,previously approved for adults,was approved in 2021 to help treat patients aged 10 years and older with heterozygous familial hypercholes
145、terolemia or homozygous familial hypercholesterolemia,which are life-threatening conditions characterized by severely high cholesterol.Oxbryta(voxelotor)tablets,originally approved in 2019 under the accelerated approval pathway to treat patients aged 12 years and older with sickle cell disease,a blo
146、od disorder that disrupts oxygen transport.In 2021,CDER expanded approved use of the drug to afflicted children as young as four years.Pradaxa(dabigatran etexilate)capsules were originally approved in 2010 to reduce the risk of stroke and blood vessel blockage in adults with a certain type of irregu
147、lar 27New Uses of CDER-Approved Drugsheartbeat.In 2021,CDER expanded Pradaxa use to prevent and treat blood clots in certain specified patients as young as eight years old.Pradaxa is the first FDA-approved oral blood thinning medication for children.Trikafta(elecaftor/lexacaftor/ivacaftor),co-packag
148、ed for oral use,was originally approved in 2019 to treat patients aged 12 and older with the most common genetically defined form of cystic fibrosis.In 2021,CDER extended the patient population to include children as young as six years old.Xarelto(rivaroxaban)tablets were originally approved in 2011
149、 to reduce the risk of stroke and systemic embolism in adults with nonvalvular atrial fibrillation.In 2021,Xarelto was approved to treat pediatric patients from birth onward at risk of recurrent venous thromboembolism(VTE),or blood clots that form in the veins.The 2021 expanded approval also extends
150、 to certain patients with congenital heart disease as young as two years old at risk of thrombosis(blood clots).Drugs in brackets refer to other treatments for the same or similar disease.CDER approved the first oral blood thinning medication for children in 2021.28Advancing Health Through Innovatio
151、nBiosimilar and Interchangable Biosimilar Approvals The biosimilar pathway is an abbreviated approval pathway for biological products that are highly similar to and have no clinically meaningfully differences from an FDA-approved biological reference product.This abbreviated approval pathway was est
152、ablished to provide more treatment options,increase patient access,and potentially reduce the cost of important and complex therapies for serious conditions through competition.In 2021,CDER approved four new biosimilar products.Of particular note,CDER approved the first two interchangeable biosimila
153、r products,which may be substituted for the reference product at the pharmacy without the intervention of a prescriber,subject to state law,similar to how generic drugs are substituted for brand name drugs.Semglee(insulin glargine-yfgn)injection and Rezvoglar(insulin glargine-aglr)injection were app
154、roved as an interchangeable biosimilar and biosimlar,respectively,to Lantus(insulin glargine)to improve glycemic control in adults and pediatric patients with type 1 diabetes and 2829Additional Approvalsin adults with type 2 diabetes.Semglee and Rezvoglar are long-acting insulins that have no peak a
155、nd start to work in about three to four hours.Byooviz(ranibizumab-nuna)injection was approved as the first biosimilar to Lucentis(ranibizumab)to treat several eye diseases and conditions,including neovascular(wet)age-related macular degeneration,a leading cause of vision loss and blindness for Ameri
156、cans aged 65 years and older.Byooviz was also approved to treat macular edema(fluid build-up)following retinal vein occlusion(vein blockage in the retina)and myopic choroidal neovascularization,a vision-threatening complication of myopia(nearsightedness).Yusimry(adalimumab-aqvh)injection was approve
157、d as a biosimilar to Humira(adalimumab)to treat patients with rheumatoid arthritis,juvenile idiopathic arthritis,psoriatic arthritis,ankylosing spondylitis,Crohns disease,ulcerative colitis in adults and plaque psoriasis.In 2021,CDER also approved a change to one biosimilar product:A biosimilar to H
158、umira,Cyltezo(adalimumab-adbm)originally approved in 2017,was approved in 2021 as an interchangeable biosimilar to treat a variety of conditions,including ulcerative colitis in adults,Crohns disease,and different types of arthritis.Cyltezo is the first FDA-approved interchangeable monoclonal antibod
159、y.CDER has approved a total of 33 biosimilar products for 11 different reference products since 2015.This includes at least one biosimilar for each of these top selling biological products in the U.S.CDER has now approved seven biosimilars to Humira;five biosimilars to Herceptin;four biosimilars to
160、Remicade and Neulasta;three biosimilars to Rituxan;two biosimilars to Avastin,Neupogen,Lantus and Enbrel;and one biosimilar to Epogen and Lucentis.There are now also two approved interchangeable biosimilars.Multiple biosimilar products for an approved reference product can enhance competition,which
161、may lead to reduced costs for both patients and our health care system.Biosimilars expand treatment options and bring competition to the U.S.marketplace.30Advancing Health Through Innovation30Other Non-Novel Approvals New formulations of approved drugs can offer significant advances in therapy.Simil
162、arly,new dosage forms(such as from a capsule to a chewable tablet for those unable to swallow)can improve patient health by helping to increase adherence,making sure patients take the proper dose,and improving quality of life for patients who must use the medication on a prolonged basis.Below are no
163、table new formulations,new dosage forms,as well as other notable non-novel drug approvals of 2021.Apretude(cabotegravir)see also Cabenuva on page 15 and Vocabria on page 32 injection.Cabotegravir(the active ingredient)was approved and marketed as Cabenuva in early 2021 to treat HIV as a long-acting
164、therapy that patients receive once a month.In December 2021,Apretude(with the same active ingredient)was approved as the first long-acting antiviral to prevent HIV that patients receive once every two months.Approved BiosimilarsNumber of Biosimilars012345678LucentisLantusEpogenEnbrelNeupogenAvastinR
165、ituxanNeulastaRemicadeHerceptinHumiraReference Products31Additional Approvals Astepro(azelastine hydrochloride)nasal spray was previously approved as a prescription drug to treat seasonal and perennial allergic rhinitis,commonly known as allergies.In 2021,CDER approved Astepro for nonprescription us
166、e through a process called a partial prescription to nonprescription switch.This is a partial switch because the lower strength,which includes the perennial allergy indication for children six months to 11 years and the seasonal allergy indication for children aged two years and older,will remain pr
167、escription-based.Kloxxado(naloxone hydrochloride)see also Zimhi on page 32 nasal spray to treat opioid overdose was approved in 2021.Fabrazyme(agalsidase beta)injection was approved under accelerated approval in 2003 to treat Fabry disease,a rare inherited disorder of glycosphingolipid(fat)metabolis
168、m.In 2021,CDER converted Fabrazyme from accelerated approval to traditional approval.Fyarro(sirolimus protein-bound particles for injectable suspension)(albumin-bound),a new formulation of sirolimus,was approved to treat locally advanced unresectable or metastatic perivascular epithelioid cell tumor
169、(PEComa).Lastacaft(alcaftadine)ophthalmic solution was initially approved in 2010 to prevent itching associated with allergic conjunctivitis,or eye inflammation due to pollen.In 2021,CDER approved Lastacaft for nonprescription use through a process called a full prescription to nonprescription switc
170、h.Midazolam injection was originally approved in 1985 as a sedation medication.In 2021,CDER approved midazolam in sodium chloride injection as a sedation therapy as a component of anesthesia or during treatment in a critical care situation.It is also available as a concentration that can be administ
171、ered via infusion without additional dilution.CDER approved the conversion of two prescription drugs to nonprescription drugs in 2021.FDA converted a drug for Fabry disease from an accelerated approval to a traditional approval.32Advancing Health Through Innovation Opzelura(ruxolitinib)see also Adbr
172、y on page 11 tablets were originally approved in 2011 under the brand name Jakafi to treat patients with intermediate or high-risk myelofibrosis.In 2021,CDER approved it as a cream to treat mild-to-moderate atopic dermatitis(eczema)in patients aged 12 and older who do not respond to,or who cannot ta
173、ke,certain other topical therapies.Vocabria(cabotegravir)tablets see also Cabenuva on page 15 and Apretude on page 30 were approved in 2021 as an oral“lead-in”to assess the tolerability of the HIV treatment Cabenuva and as an oral therapy for patients who will miss a planned injection of Cabenuva.Vo
174、cabria was approved to be used in combination with Edurant,another HIV medication.Wegovy(semaglutide)injection was approved in a new formulation and dosage in 2021 for chronic weight management in adults with obesity or overweight with at least one weight-related condition,in addition to a reduced c
175、alorie diet and increased physical activity.Semaglutide was originally approved in 2017 and is marketed as Ozempic injection and Rybelsus tablets to improve blood sugar levels in adults with type 2 diabetes.Zimhi(naloxone hydrochloride)see also Kloxxado on page 31 injection to treat opioid overdose
176、was approved as a single-shot injection in 2021.Zynrelef(bupivacaine and meloxicam)extended-release solution,a combination of two approved drugs,was approved in 2021 to relieve pain for up to 72 hours in the postsurgical setting for certain procedures.Zynrelef delivers a fixed-dose combination of th
177、e local anesthetic bupivacaine and a low dose of the nonsteroidal anti-inflammatory drug meloxicam.Drugs in brackets refer to other treatments for the same or similar disease.33New Drug Therapy Approvals 2021ConclusionReviewing drug applications and deciding to approve therapies is a well-coordinate
178、d and cross-disciplinary collaboration among our physicians,safety evaluators,chemists,biologists,biostatisticians,nurses,pharmacists,pharmacologists,epidemiologists,regulatory and policy experts.These professionals worked as a well-integrated team to bring safe and effective drug therapies to the A
179、merican public as efficiently as possible in 2021 which we hope will help patients in need for years to come.On a broader level,the work in bringing new drugs to market is something that goes beyond CDER and FDA.CDER collaborates with stakeholders across the medical community,including academia,indu
180、stry,patients and their caregivers,patient advocacy groups,and state and other federal agencies,to help ensure patients have safe and effective therapies for a wide range of medical conditions.We listen to their concerns and strive to see their perspectives.We know it is important to understand the
181、needs of our key constituencies and take actions that will benefit as many Americans as possible.This report provides valuable examples of the ways CDER approves a wide range of drugs to enhance patient health.3334Advancing Health Through InnovationAppendix A:CDERs Novel Approvals of 2021(in alphabe
182、tical order)For information about vaccines,allergenic products,blood and blood products,cellular and gene therapy products go to 2021 Biological License Application Approvals.Approval dateTrade nameActive ingredient(s)Summary of FDA-approved use on approval date (see DrugsFDA for complete indication
183、)Dosage form12/27/2021Adbry tralokinumab-ldrmTo treat moderate-to-severe atopic dermatitisInjection6/7/2021Aduhelmaducanumab-avwaTo treat Alzheimers diseaseInjection2/25/2021Amondys 45casimersenTo treat Duchenne muscular dystrophyInjection3/2/2021Azstarysserdexmethylphenidate and dexmethylphenidateT
184、o treat attention deficit hyperactivity disorderCapsule11/12/2021Besremiropeginterferon alfa-2b-njftTo treat polycythemia veraInjection6/1/2021Brexafemmeibrexafungerp To treat vulvovaginal candidiasis Tablet7/20/2021BylvayodevixibatTo treat pruritus in progressive familial intrahepatic cholestasisCa
185、psule and Oral Pellet1/21/2021Cabenuvacabotegravir and rilpivirine(co-packaged)To treat HIVInjection2/12/2021CoselatrilaciclibTo mitigate chemotherapy-induced myelosuppression in small cell lung cancerInjection11/29/2021CytaluxpafolacianineTo use for ovarian cancer imagingInjection5/14/2021Empavelip
186、egcetacoplanTo treat paroxysmal nocturnal hemoglobinuriaInjection2/11/2021Evkeezaevinacumab-dgnbTo treat homozygous familial hypercholesterolemiaInjection9/15/2021ExkivitymobocertinibTo treat types of locally advanced or metastatic non-small cell lung cancerCapsule7/16/2021fexinidazoleTo treat human
187、 African trypanosomiasisTablet3/10/2021FotivdativozanibTo treat renal cell carcinomaCapsule4/22/2021Jemperlidostarlimab-gxlyTo treat endometrial cancerInjection7/9/2021KerendiafinerenoneTo reduce the risk of kidney and heart complications in chronic kidney dis-ease associated with type 2 diabetesTab
188、let8/23/2021KorsuvadifelikefalinTo treat moderate-to-severe pruritus in chronic kidney diseaseInjection12/22/2021LeqvioinclisiranTo treat heterozygous familial hypercholesterolemia or clinical ASCVDInjection9/29/2021LivmarlimaralixibatTo treat cholestatic pruritus in Alagille syndromeOral Solution11
189、/23/2021Livtencity maribavir To treat cytomegalovirus infection Tablet5/28/2021LumakrassotorasibTo treat types of non-small cell lung cancer Tablet1/22/2021LupkynisvoclosporinTo treat lupus nephritisCapsule5/28/2021Lybalviolanzapine and samidor-phanTo treat schizophrenia and aspects of bipolar I dis
190、order Tablet35New Drug Therapy Approvals 2021Summary of FDA-approved use on approval date(See DrugsFDA for complete indication)Approval dateTrade nameActive ingredient(s)Summary of FDA-approved use on approval date (see DrugsFDA for complete indication)Dosage form4/15/2021Nextstellisdrospirenone and
191、 estetrol To prevent pregnancyTablet8/6/2021Nexviazymeavalglucosidase alfa-ngptTo treat late-onset Pompe diseaseInjection2/26/2021NulibryfosdenopterinTo reduce the risk of mortality in molybdenum cofactor deficiency type AInjection2/26/2021Pepaxtomelphalan flufenamideTo treat relapsed or refractory
192、multiple myelomaInjection3/18/2021PonvoryponesimodTo treat relapsing forms of multiple sclerosisTablet5/26/2021Pylarifypiflufolastat F 18 To identify prostate-specific membrane antigen-positive lesions in prostate cancerInjection4/2/2021QelbreeviloxazineTo treat attention deficit hyperactivity disor
193、derCapsule9/28/2021QuliptaatogepantTo prevent episodic migrainesTablet7/16/2021RezurockbelumosudilTo treat chronic graft-versus-host disease after failure of at least two prior lines of systemic therapyTablet5/21/2021Rybrevantamivantamab-vmjwTo treat types of non-small cell lung cancerInjection6/30/
194、2021Rylazeasparaginase erwinia chrysanthemi(recombi-nant)-rywnTo treat acute lymphoblastic leukemia and lymphoblastic lymphoma in patients who are allergic to E.coli-derived asparaginase products,as a com-ponent of chemotherapyInjection7/30/2021Saphneloanifrolumab-fniaTo treat moderate-to severe sys
195、temic lupus erythematousus along with standard therapyInjection10/29/2021ScemblixasciminibTo treat Philadelphia chromosome-positive chronic myeloid leukemiaTablet8/25/2021Skytrofalonapegsomatropin-tcgdTo treat short stature due to inadequate secretion of endogenous growth hormoneInjection10/7/2021Ta
196、vneosavacopanTo treat severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis in combination with standard therapy,including glucocorticoidsCapsule2/3/2021TepmetkotepotinibTo treat types of non-small cell lung cancerTablet12/27/21Tezspiretezepelumab-ekkoTo treat severe asthma as
197、 an add-on therapyInjection9/20/2021Tivdaktisotumab vedotin-tftvTo treat recurrent or metastatic cervical cancer with disease progression on or after chemotherapyInjection5/28/2021TruseltiqinfigratinibTo treat cholangiocarcinomaCapsule2/5/2021UkoniqumbralisibTo treat marginal zone lymphoma and folli
198、cular lymphomaTablet1/19/2021VerquvovericiguatTo mitigate the risk of cardiovascular death and hospitalization for chronic heart failureTablet11/19/2021VoxzogovosoritideTo improve growth in children five years of age and older with achondropla-sia and open epiphysesInjection12/17/2021Vyvgartefgartig
199、imod alfa-fcabTo treat generalized myasthenia gravisInjection8/13/2021WeliregbelzutifanTo treat von Hippel-Lindau disease Tablet3/22/2021ZegaloguedasiglucagonTo treat severe hypoglycemiaInjection4/23/2021Zynlontaloncastuximab tesirine-lpylTo treat types of relapsed or refractory large B-cell lymphom
200、aInjection36Advancing Health Through InnovationProprietary NameAdbryAduhelmAmondys 45AzstarysBesremiBrexafemmeBylvayCabenuvaCoselaCytaluxEmpaveliEvkeezaExkivityfexinidazoleFotivdaJemperliKerendiaKorsuvaLeqvioLivmarliLivtencityLumakrasLupkynisLybalviNextstellisNexviazymeAppendix B:Novel Drug Designat
201、ion Drugs listed in alphabetical orderFirst-in-ClassOrphanFast TrackBreakthrough TherapyPriority ReviewAccelerated ApprovalPDUFA Goal MetFirst in the United StatesFirst Cycle Approval37New Drug Therapy Approvals 2021Proprietary NameNulibryPepaxtoPonvoryPylarifyQelbreeQuliptaRezurockRybrevantRylazeSa
202、phneloScemblixSkytrofaTavneosTepmetkoTezspireTivdakTruseltiqUkoniqVerquvoVoxzogoVyvgartWeliregZegalogueZynlonta First-in-ClassOrphanFast TrackBreakthrough TherapyPriority ReviewAccelerated ApprovalPDUFA Goal MetFirst in the United StatesFirst Cycle ApprovalAppendix B(continued):Novel Drug Designation Drugs listed in alphabetical orderU.S.Food and Drug Administration10903 New Hampshire Ave.Silver Spring,MD 20993www.fda.gov