Vertex's Kalydeco Gets EU Approval to Treat CF in Infants

福泰制药治疗6至12个月囊性纤维化患儿药物Kalydco获欧盟委员会批准

2019-12-12 09:42:06 YAHOO!FINANCE

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Vertex Pharmaceuticals Inc. VRTX announced that the European Commission has granted marketing authorization to its cystic fibrosis drug Kalydeco for expanded use in infants aged from six to less than 12 months with at least one of specified nine mutations in the CFTR gene. Following this nod, CF patients as young as six months in Europe can now have access to Kalydeco to treat the underlying cause of CF. Kalydeco was approved by the FDA to address the same patient population in April 2019. The approval was based on data from an ongoing open-label phase III study, ARRIVAL, which evaluated children aged less than 24 months with a CFTR gating mutation. The safety profile of the study was similar to the observations in earlier phase III studies of older children and adult CF patients, which demonstrated improvements in sweat chloride, indicating a secondary endpoint. Notably, in Europe, Kalydeco was until now approved to treat CF in children aged 12 months and older with at least one of specified nine mutations in the CFTR gene. The drug is also approved for CF patients aged 18 years and above with R117H mutation in the CFTR gene. Shares of Vertex have surged 32.9% so far this year compared with the industry’s increase of 6.1%. We would like to remind investors that Kalydeco along with Vertex’s two other CF medicines, namely Orkambi and Symdeko (known as Symkevi in Europe) are collectively approved for treating a good number of CF patients in North America, Europe and Australia. Total CF product revenues in the first nine months of 2019 were $2.7 billion, reflecting a rise of approximately 23% year over year. Meanwhile, in October 2019, Vertex gained FDA approval for its fourth medicine Trikafta to treat CF. The drug is a triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor). With this approval, the company can address a significantly larger CF patient population — almost 90% patients — ahead. With the nod to Trikafta, approximately 45,000 patients worldwide are now eligible to be treated with one of Vertex’s four CF medicines. Several companies like AbbVie ABBV, Eloxx Pharmaceuticals, ProQR Therapeutics PRQR and Proteostasis Therapeutics are developing medicines to treat CF. Even though Vertex enjoys a strong position in this market, stiff competition due to entry of additional products would erode its revenues. Zacks Rank & Other Key Pick Vertex currently carries a Zacks Rank #2 (Buy). Another top-ranked stock in the biotech sector is Anika Therapeutics Inc. ANIK, sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here. Anika’s earnings estimates have moved 16% north for 2019 and 17.4% for 2020 over the past 60 days. The stock has soared 64.9% so far this year. 5 Stocks Set to Double Each was hand-picked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2020. Each comes from a different sector and has unique qualities and catalysts that could fuel exceptional growth.Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor. Today, See These 5 Potential Home Runs >> 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 AbbVie Inc. (ABBV) : Free Stock Analysis Report Anika Therapeutics Inc. (ANIK) : Free Stock Analysis Report ProQR Therapeutics N.V. (PRQR) : Free Stock Analysis Report Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report To read this article on Zacks.com click here. Zacks Investment Research
福泰(Vertex) Pharmaceuticals Inc . VRTX 宣布,欧盟委员会已向其囊性纤维化药物 Kalydco 授予上市许可,允许其在 CFTR 基因中至少有9个指定突变之一的6至12个月以下婴儿中扩大使用。 在这种情况下,在欧洲六个月大的 CF 患者现在可以使用 Kalydeco 治疗 CF 的根本原因。 Kalydco 在2019年4月被 FDA 批准用于治疗相同的患者群体。 这一批准是基于正在进行的开放标签 III 期研究的数据, ARRIVAL 对24个月以下儿童进行 CFTR 基因突变的评估。 这项研究的安全性概况类似于早期 III 期研究中对老年儿童和成人 CF 患者的观察,这些研究显示了氯化汗的改善,显示了次要终点。 值得注意的是,在欧洲, Kalydeco 直到现在才被批准用 CFTR 基因的至少九种突变之一治疗12个月以上的儿童的 CF 。该药物还被批准用于18岁及以上 CFTR 基因 R117H 突变的 CF 患者。 今年迄今为止,福泰(Vertex)的股价已飙升32.9%,而该行业的涨幅为6.1%。 我们想提醒投资者, Kalydeco 和福泰(Vertex)的另外两种 CF 药品,即 Orkambi 和 Symdeko (欧洲称为 Symkevi )被共同批准用于治疗北美、欧洲和澳大利亚的大量 CF 患者。2019年前9个月 CF 产品总收入为27亿美元,同比增长约23%。 同时,在2019年10月,福泰(Vertex)获得 FDA 批准其第四种药物 Trikafta 治疗 CF 。该药物是一个三重组合养生法( elexacaptor / tezacaptor / ivacaptor 和 ivator )。通过这项批准,该公司可以提前解决一个更大的 CF 患者群体——几乎90%的患者。通过与 Trikafta 的合作,目前全球约有45000名患者有资格接受福泰(Vertex)四种 CF 药物之一的治疗。 像艾伯维(AbbVie) ABBV 、 Eloxx Pharmaceuticals 、 ProQR Therapeutics PRQR 和 Protostabsis Therapeutics 等公司正在开发治疗 CF 的药物。尽管福泰(Vertex)在这个市场上占有很强的地位,但由于新产品的进入,激烈的竞争将侵蚀其收入。 Zacks 排名和其他关键选择 福泰(Vertex)目前拥有 Zacks 排名第二(购买)。另一家在生物技术领域排名最靠前的股票是 Anika Therapeutics Inc . ANIK ,运动 Zacks 排名第一( Strong Buy )。你可以在这里看到今天 Zacks 1号股票的完整列表。 安妮卡的收益预期在过去的60天里,2019年和2020年分别增长了16%和17.4%。该股今年迄今已飙升64.9%。 5只股票将翻倍 每只股票都是由 Zacks 专家亲手挑选出来的,他们是在2020年获得+100%或以上收益的头号热门股票。每一个都来自不同的部门,具有独特的品质和催化剂,可以促进异常增长。本报告中的大多数股票都是在华尔街的雷达下飞行的,这提供了一个很好的机会进入地面。 今天,请参阅这5个潜在的首页运行>> 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艾伯维(AbbVie) Inc .( ABBV ):免费股票分析报告 Anika Therapeutics Inc .( ANIK ):免费股票分析报告 ProQR Therapeutics N.V .( PRQR ):免费股票分析报告福泰(Vertex) Pharmaceuticals Incorporated ( VRTX ):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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