FDA重磅發布2018年年度新藥報告,59款新藥創歷史新高2017年46款,2016年22款 2015年45款

2019-01-11    來源:    作者:  瀏覽次數:418


The beginning of a new year is a good opportunity to look back over the past year and see how all the valuable work done at FDA's Center for Drug Evaluation and Research (CDER) results in bringing important new drug therapies to patients in need. It is particularly clear that our work is about people—and advancing patient care and public health.

In 2018, we approved many new drugs never before marketed in the United States, known as "novel" drugs, along with a wide variety of approvals of drugs already on the market to put to new and innovative uses. Many of these new approvals will have a significant impact on improving—and indeed, saving—countless patients' lives.

This past year was important for meeting the critical needs of people suffering from rare diseases who will benefit from these advances. Among many new orphan therapies, in 2018 CDER approved the first drug to treat patients with a rare, inherited form of rickets, a condition that leads to impaired bone growth and development. Also, CDER approved the first orally-administered drug to treat Fabry disease, a rare and serious disorder that can cause many adverse symptoms, including pain and burning in the hands and feet, and damage to the kidneys and heart. We also approved a new drug to treat patients with phenylketonuria, a rare dietary condition in which patients are born with an inability to break down protein-containing foods and certain sweeteners, and which can lead to brain and nerve damage.

Many of CDER's other 2018 approvals will provide hope, relief, and enhanced quality of life for patients in need:

  • CDER approved the first drug ever to treat smallpox, the first of a new class of drugs to treat patients with HIV-1 infection who have failed other therapies, and a new single dose treatment for influenza (flu).

  • CDER approved a new drug to treat seizures in patients with the rare and severe forms of epilepsy Lennox-Gastaut syndrome and Dravet syndrome, and a second drug to treat seizures in patients with Dravet syndrome. Previously there had been no FDA-approved therapy for Dravet syndrome. We approved three new drugs—all in a new drug class—to treat patients with migraines. Additionally, CDER approved the first therapy to treat multiple sclerosis in children.

  • We approved new advances for certain patients with breast cancer, prostate cancer, lung cancer, and thyroid cancer. We also approved a variety of new drug therapies to treat blood cancers and other blood disorders such as acute myeloid leukemia (a type of blood cancer), and classical Hodgkin lymphoma (a type of cancer of a part of the immune system called the lymphatic system); and thrombocytopenia (a deficiency of platelets in the blood) in patients with chronic liver disease scheduled to undergo a medical or dental procedure.

  • We also approved seven new biosimilars, which will further help to create competition, increase patient access, and potentially reduce the cost of important biological drug therapies.

Of course, even the best drug therapies are of no use to patients if they are stuck in the approval process. I want to recognize the importance of the efficiencies with which we brought these new therapies to approval.

  • Under the Prescription Drug User Fee Act (PDUFA), drug developers are assessed user fees that provide FDA with the additional resources needed to maintain an efficient and effective review process. PDUFA includes goal dates for application review agreed to with the pharmaceutical industry and approved by Congress. In 2018, CDER met its PDUFA goal dates for 100 percent of the novel drugs approved (59 of 59).

  • We use four specific regulatory tools to ensure prompt and efficient expedited review for approval decisions: Fast Track, Breakthrough, Accelerated Approval, and Priority Review. Each serves a purpose in helping to speed a drug’s development or review. In 2018, 73 percent of CDER’s novel drug approvals (43 of 59) were designated in one or more of these expedited review categories. These innovative approval methods can bring a therapy to patients months or even years sooner than if their application went through the standard review process.

  • In 2018, CDER approved 95 percent of its novel drug approvals (56 of 59) on the first cycle. A "cycle" is the time from when CDER accepts an application for a new drug until we decide about whether or not to approve it. A typical cycle generally takes somewhere between six months to a year, but sometimes things can go wrong. For instance, miscommunication, omissions of important data, and other issues in these complex applications, can lengthen the time it takes to complete a review and may also result in the need for a second cycle, further delaying access to the drug for patients in need. From 2011 through 2017, CDER approved 250 novel drugs, of which 205 (82 percent) were approved on the first cycle. Our consistently high first cycle approval rate reflects CDER’s commitment to working closely with applicants as they design their studies and build their application data. It also reflects our efficiency in getting new therapies to patients as quickly as possible.

  • Although regulatory processes differ widely between FDA and those of regulatory agencies in other countries, 42 of the 59 novel drugs approved in 2018 (71 percent) were approved in the United States before receiving approval in any other country.

More details about CDER’s new drug therapy approvals—including many specific examples of notable new approvals for the year—are available in our annual New Drug Therapy Approvals report.

I am proud to be part of a team of dedicated professionals who work tirelessly to find innovative, efficient, and, most importantly, safe ways to bring new therapies to patients as quickly as possible.

Tomorrow I will share my thoughts on CDER’s drug safety efforts for 2018.



2019年1月8日,美國食品藥品監督管理局(FDA)發布了年度新藥報告,《2018 New Drug Therapy Approvals》,披露了2018年批準新藥59款,為歷史新高:1/3 是 First-in-Class 的原研藥,24%突破性療法,70%+ 加速審批渠道!



FDA下屬藥品評價與研究中心 (CDER)負責新藥審批,包含新藥申請(NDAs)下的新分子實體(NME),以及作生物制劑許可申請(BLAs)下的新治療生物制劑。

CDER 2018年的所有新藥批準 Source: FDA




CDER 最近10年的新藥批準走勢 |  Source: FDA



#一類新藥(First-in-Class): 32%

CDER將2018年批準的59種新藥中的19種確定為一類藥物,這是該藥物對美國人健康產生強烈積極影響的一個指標。 這些藥物通常具有與現有療法不同的作用機制。 

2018年度新藥,一類創新藥32% |  Source: FDA

FDA于2018年批準的新型藥物被認定為一流的藥物:Aimovig,Crysvita,Elzonris,Galafold,Gamifant,Lucemyra,Lutathera,Onpattro,Orilissa,Oxervate,Palynziq,Poteligeo,Tavalisse,Tegsedi,Tibsovo,Tpoxx,Trogarzo,Vitrakvi Xofluza。...



2018年度新藥,整體使用快速發展和審查方法的占73%|  Source: FDA


2018年59種新藥批準中有43份(73%)被指定為快速通道,突破,優先審查和/或加速批準的一個或多個加急類別,包括FAST Track (41%),Breakthrough Therapy (24%), Accelerated Approval(7%)。

各快速審批渠道一覽:FAST Track (41%),Breakthrough Therapy (24%), Accelerated Approval(7%)|  Source: FDA

使用至少一種加急批準方法在2018年批準的新型藥物是:Aemcolo,Ajovy,Biktarvy,Copiktra,Crysvita,Daurismo,Diacomit,Doptelet,Elzonris,Epidiolex,Erleada,Firdapse,Galafold,Gamifant,Krintafel,Libtayo,Lorbrena,Lucemyra, Lumoxiti,Lutathera,moxidectin,Mulpleta,Nuzyra,Omegaven,Onpattro,Orilissa,Oxervate,Palynziq,Poteligeo,Revcovi,Symdeko,Takhzyro,Talzenna,Tegsedi,Tibsovo,Tpoxx,Trogarzo,Vitrakvi,Vizimpro,Xerava,Xofluza,Xospata和Zemdri。


2018年,CDER的59種新藥中有34種(58%)用以治療罕見病或“孤兒”疾病(患者數量20萬或更少)。 患有罕見疾病的患者,通常很少或沒有可用于治療其病癥的藥物。 

2018年度新藥,與罕見病相關藥品占比:58% |  Source: FDA

2018年以孤兒藥物名稱批準的新型藥物是:Asparlas,Braftovi,Copiktra,Crysvita,Daurismo,Diacomit,Elzonris,Epidiolex,Firdapse,Galafold,Gamifant,Krintafel,Lorbrena,Lumoxiti,Lutathera,Mektovi,moxidectin,Omegaven,Onpattro,Oxervate,Palynziq,Poteligeo,Revcovi,Symdeko,Takhzyro,Tavalisse, Tegsedi,Tibsovo,Tpoxx,Trogarzo,Ultomiris,Vitrakvi,Vizimpro和Xospata。...



2018年度新藥,美國優先批準的藥品占比:71% |  Source: FDA

2018年首次在美國批準的新型藥物是:Aemcolo,Aimovig,Ajovy,Akynzeo,Annovera,Asparlas,Biktarvy,Braftovi,Copiktra,Daurismo,Doptelet,Elzonris,Emgality,Epidiolex,Erleada,Gamifant,Ilumya,Krintafel,Libtayo, Lumoxiti,Mektovi,moxidectin,Nuzyra,Onpattro,Orilissa,Palynziq,Pifeltro,Revcovi,Seysara,Symdeko,Takhzyro,Talzenna,Tavalisse,Tibsovo,Tpoxx,Trogarzo,Ultomiris,Vitrakvi,Vizimpro,Xerava,Yupelri和Zemdri。





雖然CDER在2018年批準了59種新藥,比去年任何一年都多,但CDER一如既往地關注批準的醫療價值,而不是數字。 批準了的許多已獲FDA批準的藥物的還有適應證拓展的新用途,新配方和新劑型,這些藥物將有助于促進患者護理....這些都可以增加患者獲得治療的機會并降低患者的成本。 


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