FDA Approves Expanded Use For Sarepta's Rare Muscular Dystrophy Gene Therapy
FDA Approves Expanded Use For Sarepta's Rare Muscular Dystrophy Gene Therapy
Thursday, the FDA approved labeled indication for Sarepta Therapeutics Inc's (NASDAQ:SRPT) Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.
美國食品藥品監督管理局已批准Sarepta Therapeutics Inc.(納斯達克SRPT)的Elevidys(delandistrogene moxeparvovec-rok1)標籤適應症，包括至少4歲的Duchenne肌肉萎縮(DMD)個體，並且已經確認這些個體的基因具有突變。DMD確認其功能益處，FDA爲可行走患者(能夠行走的患者)批准了傳統的批准，並且爲不能行走的患者(不能行走的患者)加速批准。
Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients (patients who can walk) and accelerated approval for non-ambulatory patients (patients who cannot walk).
Duchenne肌肉萎縮患者的不可逆性損傷使得該藥物的價格非常高。不可行走的Duchenne患者的繼續獲批可能取決於在確認性試驗中的臨床受益驗證。ELEVIDYS禁忌於DMD基因的外顯子8和/或外顯子9存在任何缺失的患者。
Related: Sarepta Downgraded As FDA Decision Date Approaches, Shares Up 50% Over Last Six Months.
相關新聞: 吉利德的 Trodelvy 在普通膀胱癌最常見類型的後期研究中未能達到主要目標。Sarepta再次獲批，FDA決策日期臨近，股價在過去六個月中上漲了50%，但升級至中性。
Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
Duchenne不可行走患者的繼續獲批可能取決於在確認性試驗中的臨床受益驗證。ELEVIDYS禁忌於DMD基因的外顯子8和/或外顯子9存在任何缺失的患者。
In June last year, the FDA granted accelerated approval to Sarepta Therapeutics's one-time gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD) in ambulatory pediatric patients aged 4 through 5 with a confirmed mutation in the DMD gene.
去年6月，FDA爲Sarepta Therapeutics的一次性基因療法Elevidys在DMD基因的確認突變的4-5歲的行走患者中獲得了加速批准。
Consistent with the accelerated approval pathway, Sarepta will conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of Elevidys in non-ambulatory patients with Duchenne muscular dystrophy.
與加速批准途徑一致，Sarepta將進行並提交一項隨機、對照試驗的結果，以驗證和確認Elevidys在Duchenne肌肉萎縮的非行走患者中的臨床受益。
ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement.
ENVISION (研究SRP-9001-303)是一個全球、隨機、雙盲、安慰劑對照的3期研究，研究ELEVIDYS在Duchenne的不行走和年長的行走個體中的作用，該研究正在進行中，旨在作爲後期市場需求的一部分。
Sarepta is working with Roche Holdings AG (OTC:RHHBY) as part of a collaboration agreement signed in 2019.
Sarepta與羅氏控股（OTC:RHHBY）在2019年簽署了一項合作協議。
Sarepta is responsible for regulatory approval, manufacturing, and commercialization of Elevidys in the U.S.
Sarepta負責在美國獲得法規批准，製造和商業化Elevidys。
Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world.
羅氏負責在全球範圍內獲得法規批准，並將Elevidys爲患者帶到全球範圍內。
Last week, Pfizer Inc (NYSE:PFE) disclosed disappointing results from its Phase 3 CIFFREO study, evaluating the investigational gene therapy for Duchenne muscular dystrophy (DMD) in young boys.
上週，輝瑞公司（紐交所:PFE）公佈了其CIFFREO研究的令人失望的結果，該研究評估了用於治療年輕男孩的Duchenne肌肉萎縮的研究性基因療法。
The study did not achieve its primary endpoint of improving motor function in patients aged 4 to 7 years.
該研究未能實現改善4至7歲患者運動功能的主要終點。
Also Read: Second Death – Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial.
還閱讀：第二例死亡——輝瑞公司報告年幼男孩在肌肉廢棄失調試驗治療一年後死亡。
CNN reports that Elevidys costs around $3.2 million per patient, making it the second most expensive drug globally, after the $3.5 million hemophilia treatment Hemgenix.
CNN報道稱，Elevidys每個患者約需320萬美元，使其成爲全球第二昂貴的藥物，僅次於350萬美元的血友病治療藥物Hemgenix。
Price Action: SRPT shares are up 37.6% at $169.98 during the premarket session at last check Friday.
截至上週五最後一次檢查時，SRPT股價在盤前交易階段上漲了37.6%，至169.98美元。
Photo via Shutterstock
圖片來自shutterstock。