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1、ADVANCING HEALTH THROUGH INNOVATION:NEW DRUG THERAPY APPROVALS 2020 January 2021 www.fda.govIMPACT INNOVATION PREDICTABILITY ACCESSFDAs Center for Drug Evaluation and Research2Advancing Health Through Innovation 32020 New Drug Therapy ApprovalsTable of ContentsDirectors Message.42020:Another Strong

2、Year for Innovation and Advances .6CDERs Drug Therapy Approvals of 2020.10Novel Drugs .11CDERs Novel Drug Approvals of 2020.11CDERs Annual Novel Drug Approvals:2011-2020.12Innovation:Frequent Use of Expedited Development and Review Pathways.20Access:First Cycle Approval and Approvals Compared to Oth

3、er Countries.24New and Expanded Uses of Already FDA-Approved Drugs .26New Uses .26New Populations.32Additional Approvals .34Biosimilars.34New Formulations and Other Notable Approvals .35New Dosage Forms.36Conclusion.38Patrizia Cavazzoni,M.D.Acting Director,Center for Drug Evaluation and Research4Adv

4、ancing Health Through Innovation Directors MessageWelcome to the FDAs Center for Drug Evaluation and Researchs(CDERs)annual report,Advancing Health Through Innovation:New Drug Therapy Approvals,representing our tenth consecutive year of reporting our Centers notable new drug approvals,and our role i

5、n bringing innovative new drug therapies that are safe and effective to patients in need.Throughout the year,the COVID-19 pandemic presented unprecedented challenges to our entire staff.Despite these hardships,we have approved many new therapies that will advance health for the American public.This

6、includes the first FDA-approved drug for the treatment of patients with COVID-19.As with previous years,this years notable new drug therapies include a variety of novel drugs-those never before approved or marketed in the United States.Novel drugs often represent important new therapies for advancin

7、g patient care.This is now our fourth consecutive year of pub-lishing an expanded version of the report that goes beyond a discussion of novel approvals and includes an overview of additional notable approvals-for instance,new approvals for uses of already FDA-approved drugs.You will find,as in past

8、 years,that many important advances in drug therapy approved in 2020 use an already FDA-approved drug to treat a new disease beyond that for which it was originally approved or to treat a new population of patients,such as children.Our report emphasizes some of the many innovative ways in which we w

9、ere able to evaluate safety and efficacy for these new therapies,as well as key regulatory tools we used to enhance our efficiency and expedite the review and approval of applications.This report also summarizes the years biosimilar approvals.Biosimilars have great potential for both patients and th

10、e entire health care system.As patents and exclusivity protections for biologics expire in the United States,we can expect many more biosimilars to be submitted for approval.More biosimilar products on the market means greater competition that may lead to increased access to therapies and lower cost

11、s to patients.The decisions we made on these approvals were generally completed by or before their goal dates as defined by Congressionally approved agreements with industry(referred to as user fee programs).Most were approved in the United States before any other country in the world.52020 New Drug

12、 Therapy ApprovalsOur work to review and approve new therapies is meaningful to the extent that we can also ensure their safety.All newly approved products were required to meet our rigorous premarket safety standards-and they will all be part of a strong postmarket safety surveillance system that m

13、onitors how they perform after they are more widely used by larger patient populations.We will summarize our safety activities in a different report.Keep in mind that this report focuses on CDER approvals.FDAs Center for Biologics Evaluation and Research(CBER)also approves many import-ant therapies

14、to advance and protect the health of the American public.For more information,please visit CBERs web page for 2020 Biological Approvals.We trust this report will continue to promote greater under-standing of the many ways CDER works to support innovation and improve treatments for patients.6Advancin

15、g Health Through Innovation 2020:Another Strong Year for Innovation and Advances In 2020,despite severe challenges from the COVID-19 pandemic,the FDAs Center for Drug Evaluation and Research(CDER)approved many new drug therapies to help a wide range of patients suffering from many different medical

16、conditions to gain new hope for improved quality of life,and in some cases,improved chances of surviving life-threatening illnesses.Many of these new therapies were approved for patients with rare diseases,which,as shown below,span a broad spectrum of disease categories.Infectious Disease In 2020,CD

17、ER approved the first medication in the U.S.for the treatment of patients with COVID-19(hospitalized adults and adolescents).For patients living with HIV-1,CDER approved the first new drug in a new class of drugs to treat adults with HIV-1 who have tried multiple HIV-1 medications and whose infectio

18、n cannot be successfully treated with other therapies because of resistance,intolerance,or safety considerations.We also approved a new tablet for oral suspension formulation of an already 72020 New Drug Therapy ApprovalsFDA-approved drug,given in combination with other HIV-1 drugs,that will help en

19、sure earlier treatment for many pedi-atric patients-an important part of HIV therapy.We also approved the first FDA-approved flu prevention medication that is taken as a single dose in pill form when a person is exposed to a household contact.This past year also saw CDER expand approval of a chronic

20、 hepatitis C drug to include treatment for a larger population of patients than those treated under the original approval.CDER also approved two new treatments for patients with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia.Approvals of note for patients with ra

21、re infections in the U.S.include,1)a new drug for the treatment of patients with severe malaria,2)two new drugs approved for the treatment of patients infected with Ebola virus,and,3)a new therapy for Chagas disease,a rare parasitic disease that can cause congestive heart failure:the first FDA-appro

22、ved therapy to treat pediatric patients younger than two years of age.NeurologyFor patients with Parkinsons disease,we approved a new therapy to use during“off”episodes when their regular medi-cation is not working.Additionally,CDER approved a new therapy for certain patients suffering from migraine

23、 headaches.CDER also approved two notable new therapies to help patients with forms of multiple sclerosis.Additionally,CDER approved a new therapy to start and maintain sedation in adults undergoing short(less than 30 minute)medical procedures.Approvals of note for patients with rare neurological di

24、seases include,1)the first orally-administered drug to treat patients with spinal muscular atrophy,a disease that diminishes physical strength by affecting nerve cells in the spinal cord,taking away the ability to walk,eat,or breathe,2)a new drug for certain patients with Duchenne muscular dystrophy

25、,a genetic disorder,usually occurring in young boys,in which alterations of a protein called dystrophin leads to progressive muscle degeneration and weakness,3)a new therapy to treat patients with seizures associated with tuberous sclerosis complex,a rare,multi-system genetic disease that causes non

26、-cancerous tumors to grow in the brain and on other vital organs such as the kidneys,heart,eyes,lungs,and skin,4)a new therapy to help prevent seizures in certain pediatric patients with the seizure disorder,Dravet syndrome,and,5)two new drugs to treat patients with neuromyelitis optica spectrum dis

27、order,a rare disease of the central nervous system that primarily attacks the optic nerves and spinal cord leading to blindness and paralysis.Autoimmune,Inflammatory,and Lung Diseases In 2020,CDER approved two new therapies for the treatment of polyarticular juvenile idiopathic arthritis,a type of j

28、uvenile arthritis(arthritis in children)that causes inflammation in five or more joints within the first six months of the disease.CDER also approved a new therapy for pediatric patients with ulcerative colitis,a chronic disease of the large intestine,in which the lining of the colon becomes inflame

29、d and develops open sores,or ulcers.Additionally,CDER approved a new therapy to treat patients with chronic fibrosing interstitial lung diseases(ILD)with a progressive phenotype(trait)-the first FDA-approved treatment for this group of fibrosing lung diseases that worsen over time.Notable approvals

30、for patients with rare diseases include,1)the first FDA-approved therapy to treat the rare disease lupus nephritis,a serious and sometimes fatal complication of lupus caused by inflammation and dysfunction of the kidneys,2)a new approval for the treatment of active Stills disease,including adult-ons

31、et Stills disease(AOSD),a rare type of inflammatory arthritis that affects the entire body.This is the first FDA-approved therapy for AOSD,3)a new drug to treat patients with hereditary angioedema a rare disorder characterized by recurrent episodes of severe swelling(angioedema),most commonly in the

32、 limbs,face,intestinal tract,and airway,and,4)two new treatments for patients with a condition called,deficiency of interleukin-1 receptor antagonist,a very rare genetic inflammatory condition resembling an infection throughout the body or a bone infection that happens in newborns during the first d

33、ays of life.8Advancing Health Through Innovation Heart,Blood,Kidney,and Endocrine Diseases CDER approved a new therapy to help raise excessively low levels of phos-phate in the blood(induced by a specific type of tumor)in certain patients with osteomalacia(softening of the bones).We also approved a

34、new therapy to reduce the risk of cardiovascular death and hospitalization for adult patients with a type of heart failure.Additionally,CDER approved two different new types of already FDA-approved insulins to improve gly-cemic control in certain adults and children with diabetes,which may help expa

35、nd patient access and market competition for insulin products in the United States.CDER also approved a new drug for adults with growth hormone deficiency,the first of its kind that can be injected weekly,rather than daily.Approvals of note for patients with rare diseases include,1)a new therapy to

36、treat patients with hypereosinophilic syndrome,a group of rare blood disorders that occur when the blood contains too many eosinophils(white blood cells that normally play an important role in the immune system),which eventually damage organs,2)a new drug for the treatment of obesity and the control

37、 of hunger associated with pro-opiomelanocortin defi-ciency,a rare disorder that causes severe obesity that begins at an early age,3)a new drug for the treatment of certain patients with Cushings disease,a condition in which the body makes too much cortisol,a hormone related to stress response,and,4

38、)a new drug for the treatment of adult and pediatric patients with primary hyperoxaluria type 1,the most common and severe type among various forms of this condition.It is a rare disorder that mainly affects the kidneys,which results from buildup of a substance called oxalate,which,absent this disea

39、se,is normally filtered by the kidneys and excreted in the urine.CancersCDER approved many new therapies in 2020 for a wide array of cancers,particularly lung cancers,for which we approved two notable new treat-ments for patients with small cell lung cancer and nine to treat various forms of non-sma

40、ll cell lung cancer,two of which were also approved to treat patients with certain types of thyroid cancer.We also approved four new notable therapies to treat patients with various forms of breast cancer.For patients with other forms of cancer CDER approved,1)two new treatments for certain patients

41、 with urothelial cancer,which is a cancer that begins in cells called urothelial cells in the urinary system,2)a new first-line treatment for patients with a type of colorectal cancer that has spread to other parts of the body or for which surgery is not an option,3)two new therapies for the treatme

42、nt of certain patients with forms of prostate cancer,and,4)a new approval for the treatment of certain patients with bladder cancer who do not respond favorably to first-line treatment.CDERs drug therapy approvals of 2020 will help advance patient care in many new areas.92020 New Drug Therapy Approv

43、alsCDER also approved a variety of new therapies to treat patients with rare cancers.These include 1)three notable new treatments for certain patients with multiple myeloma,a rare cancer that forms in white blood cells called plasma cells,which fight infections,2)a new drug for the treatment of pati

44、ents with high-risk refractory or relapsed neuroblastoma,a very rare type of cancerous tumor,often found in the adrenal glands,that almost always affects children,3)a new drug in tablet form approved for treating patients with the rare blood cancers,myelodysplastic syndromes and chronic myelomonocyt

45、ic leukemia,many of whom will benefit from taking self-administered tablets instead of having to go the health care facility for injections of other treatments,4)two new drugs to treat certain patients with gastrointestinal stromal tumor,a rare type of cancer that starts in specialized nerve cells l

46、ocated in the walls of the digestive system,5)a new drug for the treatment of certain patients who have epithelioid sarcoma,a rare,slow-growing type of cancer that often begins in the soft tissue under the skin of a finger,hand,forearm,lower leg,or foot,6)a new drug for treating certain patients wit

47、h a specific type of metastatic cholan-giocarcinoma(bile duct cancer),7)a new drug for the treatment of certain pediatric patients,two years of age and older,with neurofibromatosis type 1,a genetic disorder of the nervous system causing tumors to grow on nerves-the first drug approved by the FDA to

48、treat this debilitating,progressive,and often disfiguring rare disease that typically begins early in life,and,8)a new use for two already FDA-approved drugs to be used in combination to treat patients with mesothelioma,a type of cancer caused by inhaling asbestos fibers.Other Advances and Other Rar

49、e Diseases In other advances of 2020,CDER approved a new combination of already FDA-approved drugs for the management of heavy menstrual bleeding associated with fibroids(a kind of tumor typically non-cancerous)in premenopausal women.Additionally,2020 saw the first FDA-approved therapy for certain p

50、ediatric patients with neurogenic detrusor over-activity,a bladder dysfunction frequently observed in patients with multiple sclerosis and spinal cord injury.Other notable approvals for patients with rare diseases include,1)a new drug to treat certain patients with Hutchinson-Gilford Progeria Syndro