Roche's NDA for SMA Drug Risdiplam Gets Priority Review

美国FDA授予罗氏risdiplam优先审评资格

2019-11-26 10:49:08 YAHOO!FINANCE

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Roche Holding AG RHHBY announced that the FDA has accepted the new drug application for its investigational agent risdiplam. The company is seeking approval of the candidate for the treatment of patients with spinal muscular atrophy . Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN-2) splicing modifier for SMA. With the FDA granting a priority review to the NDA, a decision from the regulatory body is expected on May 24, 2020. If approved, risdiplam will become the first at-home administered medicine for people with SMA. We note that a Priority Review designation is granted to drugs with potential to provide significant improvements in the safety and effectiveness of the treatment plus prevention or diagnosis of a serious disease. The NDA was based on 12-month data from the pivotal FIREFISH and SUNFISH studies, which evaluated risdiplam across a broad patient population suffering type 1, 2 or 3 SMA. In November, Roche announced positive data from the part 2 of the SUNFISH study on risdiplam. The study met its primary endpoint of change from baseline in the MFM-32 scale after one-year regimen with risdiplam compared to placebo, as data showed statistically significant improvements in the overall study population with type 2 or 3 SMA. Moreover, no treatment-related safety findings causing study withdrawal have been seen in any risdiplam study to date. Shares of Roche have rallied 22.5% so far this year compared with the industry’s increase of 5.1%. We remind investors that Roche leads the clinical development of risdiplam in collaboration with the SMA Foundation and PTC Therapeutics PTCT. Risdiplam is also being evaluated in a broad clinical program for SMA in patients ranging from newborns to 60 year-olds. It includes those who are previously treated with SMA-targeting therapies. Additionally, the company has two ongoing studies, namely JEWELFISH and RAINBOWFISH. Successful development of risdiplam will boost Roche’s growth prospects. However, competition is stiff in the SMA market from the likes of Biogen’s BIIB Spinraza and Novartis’ NVS gene therapy Zolgensma. Zacks Rank Roche currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here. Today's Best Stocks from Zacks Would you like to see the updated picks from our best market-beating strategies? From 2017 through Q3 2019, while the S&P 500 gained +39.6%, five of our strategies returned +51.8%, +57.5%, +96.9%, +119.0% and even +158.9%.This outperformance has not just been a recent phenomenon. From 2000 – Q3 2019, while the S&P averaged +5.6% per year, our top strategies averaged up to +54.1% per year. See their latest picks free >> Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Roche Holding AG (RHHBY) : Free Stock Analysis Report Novartis AG (NVS) : Free Stock Analysis Report Biogen Inc. (BIIB) : Free Stock Analysis Report PTC Therapeutics, Inc. (PTCT) : Free Stock Analysis Report To read this article on Zacks.com click here. Zacks Investment Research
罗氏控股( Roche Holding AG ) RHHBY 宣布, FDA 已接受其研究用药 risdilam 的新药申请。公司正在寻求脊柱肌肉萎缩症患者治疗候选人的批准。 Rishdilam 是一种口服液体存活运动神经元-2( SMN-2)剪接调节剂。 随着 FDA 对 NDA 给予优先审评,预计监管机构将于2020年5月24日做出决定。如果获得批准,瑞西他林将成为第一个在家给 SMA 患者服用的药物。 我们注意到,优先审评的指定是给予有可能显著提高治疗的安全性和有效性以及预防或诊断严重疾病的药物。 NDA 是基于关键 FIREFISH 和 SUNFISH 研究的12个月数据,该研究评估了1、2或3型 SMA 患者的利西平。 11月,罗氏(Roche)公布了来自 SUNFISH 关于利西平的研究的第2部分的积极数据。研究满足其主要终点变化从基线的 MFM-32规模后一年养生法与利西平相比,安慰剂,因为数据显示,统计显着改善整体研究人口与2型或3型 SMA 。 此外,迄今为止,在任何利西他林研究中都没有发现导致研究撤回的与治疗有关的安全发现。 今年迄今,罗氏(Roche)股价已上涨22.5%,而该行业的涨幅为5.1%。 我们提醒投资者,罗氏(Roche)与 SMA 基金会和 PTC 治疗学 PTCT 合作,引领利司他林的临床开发。 Rishdilam 也在一项针对从新生儿到60岁儿童的 SMA 患者的广泛临床项目中进行评估。它包括那些以前接受 SMA 靶向治疗的患者。此外,该公司有两个正在进行的研究,即 JEWLFISH 和 RAINBOWFISH 。 瑞地平的成功开发将促进罗氏(Roche)的增长前景。然而,来自百健(Biogen)公司的 BIIB Spiraza 和诺华(Novartis)公司的 NVS 基因疗法 Zolgensma 等公司的 SMA 市场竞争激烈。 扎克斯排名 罗氏(Roche)目前拥有 Zacks 排名第二(购买)。你可以看到今天的 Zacks #1排名( Strong Buy )股票的完整列表。 今天最好的股票来自 Zacks 你希望看到我们最好的市场策略的最新选择吗?2017年至2019年第三季度,标准普尔500指数上涨+39.6%,其中五个策略的回报率分别为+51.8%、+57.5%、+96.9%、+119.0%甚至+158.9%。这种超越不仅仅是最近的现象。从2000年到2019年第三季度,标准普尔平均每年+5.6%,我们的最高策略平均每年+54.1%。 查看他们最新的选择免费>> 想要 Zacks 投资研究公司的最新建议吗?今天,你可以下载7个最佳股票的未来30天。点击获取此免费报告罗氏(Roche)控股公司( RHHBY ):免费股票分析报告诺华(Novartis)公司( NVS ):免费股票分析报告百健(Biogen)公司( BIIB ):免费股票分析报告 PTC 疗法公司( PTCT ):免费股票分析报告阅读本文关于 Zacks 。com 点击这里。Zacks 投资研究

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