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.
周四，美国食品药品管理局批准了Sarepta Therapeutics Inc（纳斯达克股票代码：SRPT）的Elevidys（delandistrogene moxeparvovec-rokl）的标记适应症，将已证实存在突变的杜兴氏肌肉萎缩症（DMD）患者包括在内 DMD 至少 4 岁的基因。
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).
美国食品药品管理局对非卧床患者（能行走的患者）给予了传统的批准，对非卧床患者（不能行走的患者）给予了加速批准，这证实了其功能优势。
Related: Sarepta Downgraded As FDA Decision Date Approaches, Shares Up 50% Over Last Six Months.
相关： 随着美国食品药品管理局决策日期的临近，Sarepta下调了评级，股价在过去六个月中上涨了50％。
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.
非流动性杜兴氏病患者的持续批准可能取决于确认性试验中对临床益处的验证。ELEVIDYS 禁用于在 DMD 基因中存在任何外显子 8 和/或 9 外显子缺失的患者。
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，美国食品药品管理局加速批准了Sarepta Therapeutics的一次性基因疗法Elevidys用于杜兴氏肌肉萎缩症（DMD），该疗法适用于经证实DMD基因突变的4至5岁非卧床儿科患者。
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.
根据加速批准途径，Sarepta将进行并提交一项随机对照试验的结果，以验证和确认Elevidys对非活动性杜兴氏肌营养不良症患者的临床益处。
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）是一项针对ELEVIDYS的全球性、随机、双盲、安慰剂对照的3期研究，正在进行中，旨在作为这种上市后要求，该研究针对非卧床和老年卧床患者。
Sarepta is working with Roche Holdings AG (OTC:RHHBY) as part of a collaboration agreement signed in 2019.
作为2019年签署的合作协议的一部分，萨雷普塔正在与罗氏控股股份公司（场外交易代码：RHHBY）合作。
Sarepta is responsible for regulatory approval, manufacturing, and commercialization of Elevidys in the U.S.
萨雷普塔负责美国Elevidys的监管审批、生产和商业化。
Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world.
罗氏负责监管部门的批准，并将Elevidys带给世界其他地区的患者。
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.
上周，辉瑞公司（纽约证券交易所代码：PFE）披露了其三期CIFFREO研究的令人失望的结果，该研究评估了针对小男孩的杜兴氏肌肉萎缩症（DMD）的研究性基因疗法。
The study did not achieve its primary endpoint of improving motor function in patients aged 4 to 7 years.
该研究没有达到改善4至7岁患者运动功能的主要终点。
Also Read: Second Death – Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial.
另请阅读： 第二次死亡—辉瑞报告说，在肌肉萎缩症试验中，经过一年基因疗法治疗后，小男孩死亡。
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.
美国有线电视新闻网报道说，Elevidys每位患者的费用约为320万美元，是全球第二昂贵的药物，仅次于350万美元的血友病治疗Hemgenix。
Price Action: SRPT shares are up 37.6% at $169.98 during the premarket session at last check Friday.
价格走势：周五最后一次盘前交易中，SRPT股价上涨37.6％，至169.98美元。
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