Alnylam's Amvuttra Secures FDA Approval For Expanded Use For Rare Heart Disease, Stock Jumps
Alnylam's Amvuttra Secures FDA Approval For Expanded Use For Rare Heart Disease, Stock Jumps
The U.S. Food and Drug Administration on Thursday approved Alnylam Pharmaceuticals Inc.'s (NASDAQ:ALNY) supplemental application for Amvuttra (vutrisiran) in adults with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis, or ATTR-CM, to reduce cardiovascular mortality, cardiovascular hospitalizations, and urgent heart failure visits.
美國食品藥品監督管理局週四批准了阿爾寧製藥公司（納斯達克：ALNY）針對成年人野生型或遺傳性轉甲狀腺素介導的澱粉樣變心肌病（ATTR-CM）的Amvuttra（vutrisiran）補充申請，以降低心血管死亡率、心血管住院率和急診心力衰竭就診率。
The approval expands the indication for Amvuttra, which now becomes the first and only therapeutic approved by the FDA for ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
此次批准擴大了Amvuttra的適應症，使其成爲FDA針對ATTR-CM和遺傳性轉甲狀腺素介導的澱粉樣變性多發性神經病（hATTR-PN）在成年人中獲批的首個也是唯一的治療藥物。
ATTR-CM affects approximately 150,000 people in the U.S. and over 300,000 people worldwide.
ATTR-CM在美國影響約15萬人，全球超過30萬人。
Also Read: Alnylam's Concedes Its Patent Infringement Case Against Moderna's COVID-19 Vaccine
另請閱讀：阿爾寧公司在對Moderna新冠疫苗的專利侵權案中承認其敗訴。
This approval is based on the HELIOS-B Phase 3 clinical trial. The trial achieved statistical significance compared to placebo on all 10 pre-specified primary and secondary endpoints.
此次批准基於HELIOS-B三期臨牀試驗。該試驗在所有10個預設的主要和次要終點上相較於安慰劑達到了統計學顯著性。
In the overall population, Amvuttra reduced the risk of all-cause mortality and recurrent cardiovascular (CV) events by 28% during the double-blind treatment period of up to 36 months.
在整體人群中，Amvuttra在最長達36個月的雙盲治療期間降低了全因死亡率和複發性心血管（CV）事件的風險28%。
Mortality in this population was significantly reduced by 36% through 42 months in a pre-specified secondary endpoint analysis.
在該人群中，通過42個月的預設次要終點分析，死亡率顯著降低了36%。
In the monotherapy population, Amvuttra reduced the risk of ACM and recurrent CV events by 33% and the risk of mortality by 35% through 42 months.
在單藥治療人群中，Amvuttra將全因死亡率（ACM）和複發性心血管事件的風險降低了33%，並在42個月內降低了死亡風險35%。
Amvuttra in hATTR-PN is covered by insurers for around 99% of patients, with the majority paying nothing out-of-pocket. Similar broad coverage and out-of-pocket costs are expected in ATTR-CM, given comparable payer dynamics and the clinical value demonstrated in the HELIOS-B clinical trial.
在hATTR-PN中，Amvuttra的保險覆蓋率約爲99%的患者，大多數患者沒有自付費用。在ATTR-CM中，預計會有類似的廣泛覆蓋和自付費用，因爲支付方動態相似，並且在HELIOS-B臨牀試驗中證明了其臨牀價值。
Marketing authorization applications based on HELIOS-B data are currently under review by several global health agencies including the European Medicines Agency, the Brazilian Health Regulatory Agency, and the Japanese Pharmaceuticals and Medical Devices Agency.
基於HELIOS-B數據的市場授權申請目前正在多個全球健康機構審查，包括歐洲藥品管理局、巴西健康監管局及日本藥品和醫療設備管理局。
Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025.
Alnylam仍按計劃於2025年推進vutrisiran的額外全球監管申報。
In 2024, Amvuttra sales reached $970.45 million, up from $557.84 million a year ago.
2024年，Amvuttra的銷售額達到97045萬，較去年55784萬增長。
Price Action: ALNY stock is up 8.46% at $274.98 at the last check Friday.
價格走勢：ALNY股票在上週五的最後檢查時上漲8.46%，報274.98美元。
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Image via Shutterstock
圖片來自Shutterstock
譯文內容由第三人軟體翻譯。
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，而今天再破新高， 最高 24076 ， 其後收市時轉跌約70點， 出了陰燭， 暫時走勢仍未有破壞， 不過由前底至今已經上漲接近至6千點， 本人覺得有貨者可以繼續持貨直到 明顯有走勢轉壞才止賺離場， 沒貨者可以等待回調后再上車， 其實本人也希望能夠快點有回調， 一來可以上車， 二來回一回氣也健康
，暫時看法都是跟之前一樣， 覺得即使有所回調應該都不會跌得太深，但假若期貨失守22350企不穩收，便可能還有下跌空間， 期貨短期要跌破21400的機會應該也不大， 所以本人覺得如果有大幅的回調也是一個機會分注做多。近日都堅持不過夜持倉，暫只做即市， 因為不高追，也不隨便做空。支持阻力以現貨作參考
支持位 23150，23250，2...
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