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1、Advancing Health Through Innovation:New Drug Therapy Approvals 2021 IMPACT INNOVATION PREDICTABILITY ACCESS FDAs Center for Drug Evaluation and Research January 2022 Table of Contents Directors Message.1 Executive Summary.2 CDERs Novel Drug Approvals of 2021.5 First-in-Class Drugs.7 Drugs for Rare D
2、iseases.8 Other Novel Drug Approvals.9 Innovation:Use of Expedited Development and Review Pathways.11 Fast Track.11 Breakthrough Therapy.11 Priority Review.11 Accelerated Approval.12 Overall Use of Expedited Development and Review Methods.12 Predictability:Meeting PDUFA Goals.13 Access:First Cycle A
3、pprovals and First in U.S.Approvals.13 New Uses of Approved Drugs.14 Approved Drugs Expanded for New Pediatric Populations.15 Biosimilar and Interchangeable Biosimilar Approvals.17 Other Non-Novel Approvals.19 Conclusion.20 Appendix A:CDERs Novel Approvals of 2021 .21 Appendix B:Novel Drug Designati
4、on.23 1 Directors Message Welcome to FDAs Center for Drug Evaluation and Researchs(CDER)annual report,Advancing Health Through Innovation:New Drug Therapy Approvals,representing our 11th consecutive year of reporting CDERs notable drug approvals.This report illustrates our role in bringing innovativ
5、e drug therapies that are safe and effective to patients in need.Throughout 2021,the COVID-19 pandemic continued to 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,the 2021
6、new drug therapies span many areas of medicine and disease areas.This is our fifth consecutive year of publishing 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
7、a new patient population,such as children.Our report showcases the new therapies we evaluated for safety and efficacy,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 date
8、s as defined by congressionally authorized agreements with industry(referred to as user fee programs).Most drugs 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 bi
9、osimilar products in 2021,which are biological products that meet additional requirements and may be substituted 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 bi
10、osimilar products mean greater competition that may lead to increased access to therapies and potentially lower costs 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 unde
11、rgo postmarket safety surveillance.We will discuss our comprehensive safety activities in a different report.This report summarizes CDERs 2021 approvals and highlights examples of innovative treatments.FDAs Center for Biologics Evaluation and Research(CBER)also approves important therapies to advanc
12、e and protect public health.Perhaps most significantly in 2021,CBER approved a COVID-19 vaccine,a milestone in our continued fight against this pandemic.For more information about CBER actions,please visit CBERs webpage for 2021 Biological Approvals https:/www.fda.gov/vaccines-blood-biologics/develo
13、pment-approval-process-cber/2021-biological-license-application-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 patients.Patrizia Cavazzoni,M.D.,Director,Center for Drug Evaluati
14、on and Research Patrizia Cavazzoni,M.D.2 Executive 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,protecting patients against life-threatening illnesses.See pages 5-6 for a complet
15、e list of these therapies.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 insulin product,which
16、 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 and older with diabetes
17、.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 disease progression.For
18、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 frequency of chemoth
19、erapy-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 characterized by sever
20、ely high cholesterol levels;2)a treatment for paroxysmal nocturnal hemoglobinuria,a rare and serious blood disease;3)the first treatment to increase height in people with achondroplasia,the most common type of dwarfism;4)a treatment for polycythemia vera,a rare chronic disease involving circulatory
21、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.Autoimmune,Inflammatory,and Lung Diseases 2021 was an important year for treatments for inflammatory bo
22、wel 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 the lining of the colon becomes inflamed and develops tiny open sores,or ulcers.CDER also approved a the
23、rapy 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 at an infusion center.On the biosimilars front,CDER approved an interchangeable biosimilar and a biosim
24、ilar 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.2021 was an important year for treatments for inflammatory bowel disease.3 In 2021,CDER also approved a
25、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 serious kidney disease associate
26、d with lupus.CDER approved a drug to treat pruritus(itchiness)that can occur in patients with chronic kidney disease.CDER also approved a new therapy to treat moderate-to-severe atopic dermatitis(eczema)that has not responded to other therapies.Approvals of note to treat rare diseases in this area i
27、nclude: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 treatment for systemic sclerosis
28、-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 destruction and inflammation of small vess
29、els;and 5)drugs to treat pruritus associated with rare liver disorders,often occurring in children.Infectious Diseases In 2021,CDER continued to help advance the treatment and prevention of HIV-1 infection,the most common type of HIV.Early in the year,we approved the first injectable,complete regime
30、n to treat HIV-1 infection in adults that is administered once a month.We also approved a combination drug product,administered as 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 injectable antiviral drug to preve
31、nt HIV-1 infection in adults and adolescents that is administered once every two months.Also in the antiviral area,CDER approved a drug to treat human smallpox disease in adult and pediatric patients,including newborn babies.Although the World Health Organization has declared smallpox eradicated,the
32、re have been longstanding concerns that smallpox virus could be used as a bioweapon.This approval is therefore an important medical countermeasure.CDER also approved the first antiviral drug to treat adults and adolescents with post-transplant cytomegalovirus(CMV)infection or disease that does not r
33、espond to other antiviral drugs.This approval is an important advancement in treating CMV infection or disease,where there has 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(Hum
34、an African trypanosomiasis),a disease caused by parasites transmitted by infected tsetse flies and endemic in sub-Saharan Africa.Other sleeping sickness therapies include oral components taken together with non-oral drugs.Neurological and Psychiatric Disorders To help address the opioid crisis,we ap
35、proved two therapies to reverse an opioid overdose in an emergency situation.Using the accelerated approval pathway,CDER approved a new drug to treat Alzheimers disease.This drug is the first new treatment approved for Alzheimers since 2003 and the 2021 marked an important year for new HIV-1 treatme
36、nts and preventive therapies.4 first therapy that targets the fundamental disease pathophysiology.CDER also approved two drugs that help reduce the frequency of migraine attacks in patients with episodic migraines.Approvals of note for rare neurological disorders include:1)a treatment for certain pa
37、tients 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 chronic neuromuscular disease.Cancers 2021 marked the 50th anniversary of the National Cancer Act https:/w
38、ww.cancer.gov/about-nci/overview/history/national-cancer-act-1971,landmark legislation committed to fighting cancer.In this anniversary year,CDER approved many therapies for different cancers.Four new drugs were approved for non-small cell lung cancer,including one non-small cell lung cancer type pr
39、eviously 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 patient populations.The first approved immunotherapy to be used as a first-line treatment for esophageal(esophagus-related)cancer,gastric(
40、stomach)cancer,and gastroesophageal junction(GEJ)adenocarcinoma was an important cancer approval for 2021.CDER approved two other therapies for certain patients with HER2-positive gastric cancer and GEJ adenocarcinoma;one of these therapies was also approved for esophageal cancer,advanced kidney can
41、cer 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 that does not respond to hormone therapies or medications that target HER2 protein receptors.Other CDER approvals for rare cancers include:1
42、)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 certain tumors that are associated with von Hippel-Lindau disease,an inherited disorder characterized by tumors and cysts;3)a therapy to be us
43、ed 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 first FDA-approved treatment for locally advanced unresectable or metastatic perivascular epithelioid cell tumor(PEComa),a group of rare tu
44、mors 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 report to feature important cancer therapies approved in 2021 by CDER,CBER,and FDAs Center for Devices and Radiological Health CDRH.)Othe
45、r 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 premature death;and 2)a medication to treat neurogenic detrusor overactivity,a bladder dysfunction,in children.CDERs drug approvals of 2021 will h
46、elp advance patient care in many new areas.In 2021,CDER approved 4 new therapies for non-small cell lung cancer.5 CDERs Novel Drug 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 Biolog
47、ics License Applications(BLAs).The active ingredient or ingredients in a new drug have never before been approved in the U.S.The novel 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
48、 are listed alphabetically below by trade name.*Trade Name Active Ingredient(s)Adbry tralokinumab-ldrm Aduhelm aducanumab-avwa Amondys 45 casimersen Azstarys serdexmethylphenidate and dexmethylphenidate Besremi ropeginterferon alfa-2b-njft Brexafemme ibrexafungerp Bylvay odevixibat Cabenuva cabotegr
49、avir and rilpivirine(co-packaged)Cosela trilacicilib Cytalux pafolacianine Empaveli pegcetacoplan Evkeeza evinacumab-dgnb Exkivity mobocertinib fexinidazole*Fotivda tivozanib Jemperli dostarlimab-gxly Kerendia finerenone Korsuva difelikefalin Leqvio inclisiran Livmarli maralixibat Livtencity maribav
50、ir Lumakras sotorasib Lupkynis voclosporin Lybalvi olanzapine and samidorphan Nextstellis drospirenone and estetrol Nexviazyme avalglucosidase alfa-ngpt Nulibry fosdenopterin Pepaxto melphalan flufenamide Ponvory ponesimod Pylarify piflufolastat F 18 See Appendix B for a summary chart of designation