川普要求FDA加快新藥核准 藥界CEO不樂見 !

NBI生技躍14月高！川普讚孤兒藥 藥價政策不致太苛？ 2017/03/02 10:03 MoneyDJ新聞 2017-03-02 10:03:59 記者 郭妍希 報導 美國藥品定價過高、新藥核准程序冗長繁瑣，一直為人詬病。美國總統川普(Donald Trump)決心改革，他週二(2月28日)在首場國會演說中，聲稱要立即壓制遭到人為炒高的藥價，另外並讚揚罕見及孤兒疾病藥廠Amicus Therapeutics執行長John Crowley對研發新藥的努力、宣示要簡化新藥上市流程，消息傳來帶動美國生技類股跳漲至一年多新高。最能代表生技業表現的美國那斯達克生技類股指數(NASDAQ Biotechnology Index，簡稱 NBI)1日勁揚1.35%、收3,149.73點，突破去(2016)年9月的波段高點，創下去年1月8日收盤高。根據白宮發布的演講稿，川普在演說中強調，健保遭不必要的成本拖累、費用居高不下，美國應推動改革，讓病患、醫師不用再因過高的保險費用傷透腦筋，另外也應努力壓低被人為炒高的藥價，且即刻執行。週二正好是國際罕見疾病日(Rare Disease Day)，川普借題發揮，指出Crowley為罹患罕見病的女兒Megan創立了藥廠Amicus，致力尋找治療解藥、最終救了女兒一命，實在令人激賞。然而，美國食品藥品管理局(FDA)的核准程序繁瑣又冗長，導致許多新藥(包括拯救Megan性命的藥品)無法及時送到病患手中，假如美國能簡化整個核發程序，那麼類似Megan的奇蹟將能重現在更多人身上。 barron`s.com報導，Leerink分析師Geoffrey Porges 1日指出，雖然川普對Crowley大加讚揚，並不代表他在為罕見病藥物背書，但這仍意味著川普的藥價政策應該不會太過嚴苛，以免阻撓藥物創新。Porges表示，很難想像新政府會推動可能衝擊藥物研發的藥品訂價政策，而川普要求FDA加快新藥的核准程序，對藥界來說雖是利多，但這樣一來也會降低進入門檻，使得業界的競爭加劇。Porges說，整體來看，新政府應該是傾向減少藥界法規、以市場機制處理藥價議題，而不是直接透過法令去規管。這樣的立場，再加上國會的支持，意味著新藥研發商的前景相對看俏。另一方面，Crowley 1日在接受CNBC專訪時則表示，為了擴大Amicus的產品線，該公司一定要跟川普政府密切合作。他說，川普對藥界和主管機關，將有建設性的影響。編者按：本文僅供參考之用，並不構成要約、招攬或邀請、誘使、任何不論種類或形式之申述或訂立任何建議及推薦，讀者務請運用個人獨立思考能力，自行作出投資決定，如因相關建議招致損失，概與《精實財經媒體》、編者及作者無涉。

Amicus CEO John Crowley, Not His Daughter Megan, Might Compel Trump to Make Big Changes at FDA  by Adam Feuerstein  Mar 1, 2017 8:12 AM EST  Absent specific policy details, Trump's speech leaves investors guessing about his real plans for the FDA and the drug approval process. He has not yet named an FDA commissioner. In his speech to Congress Tuesday night, President Donald Trump called the FDA drug approval process "slow and burdensome."  To support his argument, Trump pointed to Megan Crowley, a 20-year-old Pompe disease patient. Crowley was in the gallery of the House chamber listening to Trump's speech. 

Here's what Trump said:  An incredible young woman is with us this evening who should serve as an inspiration to us all. Today is Rare Disease day, and joining us in the gallery is a Rare Disease Survivor, Megan Crowley. Megan was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past 5. On receiving this news, Megan's dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan's life. Today she is 20 years old -- and a sophomore at Notre Dame. Megan's story is about the unbounded power of a father's love for a daughter. But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan's life, from reaching those in need. If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan. In fact, our children will grow up in a Nation of miracles. Trump has previously expressed a desire to deregulate the FDA and lower the standards by which drugs are approved in the U.S. His comments about Megan Crowley seem to fit that theme.   But you could also argue Megan Crowley is an example of an FDA system that works well to quickly address the needs of patients suffering from rare diseases. Crowley's life was saved by Myozyme, an enzyme replacement therapy developed Genzyme, now a part of Sanofi (SNY). Megan's father, John Crowley, formed a small company to develop Pompe disease drugs that was acquired by Genzyme and contributed to the successful development of Myozyme.  Myozyme was studied in two, uncontrolled clinical trials that enrolled a total of 39 Pompe disease patients. Genzyme submitted the Myozyme clinical data package to the FDA in July 2005. The FDA reviewed the drug's clinical data and approved it in April 2006 -- nine months later. 

That hardly seems slow or burdensome.  The FDA has not been as kind to Amicus Therapeutics (FOLD) , the small orphan drug company run by John Crowley, Megan's father. John Crowley started Amicus after helping to develop Myozyme.  Last November, the FDA refused Amicus' request to submit an accelerated approval filing for Galafold, a new therapy to treat Fabry disease. The FDA told Amicus that an additional clinical study of Galafold would be required before the drug could be submitted and reviewed. Amicus estimates collecting the new data will take two years. In contrast, European drug regulators approved Galafold in May 2016 using the same clinical data FDA deemed insufficient. Amicus is selling Galafold to Fabry patients in Europe but can't do the same in the U.S.  Critics of the current FDA approval process could point legitimately to John Crowley and Amicus -- more than his daughter Megan -- to support their argument for less regulation.  FDA supporters will counter that the agency's decision to turn away Galafold for more data did not harm Fabry patients in the U.S. or deny them treatment because Fabrazyme, an enzyme replacement therapy from Sanofi, is approved here and used to treat Fabry patients.  If Trump's FDA rhetoric becomes reality, Amicus might benefit. Will the rest of the biotech industry? Lowering approval standards at FDA is not something most biotech and drug industry executives want to see happen.