Europe Approves Biogen's Tofersen For Adult Patients With Rare Type Of Neurodegenerative Disorder
Europe Approves Biogen's Tofersen For Adult Patients With Rare Type Of Neurodegenerative Disorder
On Thursday, the European Commission granted marketing authorization under exceptional circumstances and maintained orphan designation for Biogen Inc's (NASDAQ:BIIB) Qalsody (tofersen) for amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS).
週四，歐盟委員會在特殊情況下批准了上市許可，並維持了百健公司（納斯達克股票代碼：BIIB）Qalsody（tofersen）的孤兒稱號，用於治療與超氧化物歧化酶1基因突變相關的肌萎縮性側索硬化症（ALS）（SOD1-ALS）。
Qalsody is the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).
Qalsody是歐盟批准的第一種針對ALS遺傳病因的療法，也稱爲運動神經元疾病（MND）。
SOD1-ALS is an ultra-rare genetic form of ALS estimated to affect less than 1,000 people in Europe.
SOD1-ALS是一種超罕見的肌萎縮性側索硬化症遺傳形式，估計在歐洲影響不到1,000人。
Qalsody is Biogen's third rare disease therapy to be approved in the EU.
Qalsody是Biogen第三種獲得歐盟批准的罕見疾病療法。
Related: Tale Of Two Ionis-Partnered Neurology-Focused Early Stage Assets-Biogen Discontinues One, Decides Not To Exercise Licensing Option For Another.
相關： Tale Of Two Ionis合作的專注於神經病學的早期資產——Biogen停產了一家資產，決定不對另一家資產行使許可權。
In the Phase 3 VALOR study (n=108), patients were randomized 2:1 to receive treatment with either Qalsody 100 mg (n=72) or placebo (n=36) for 24 weeks.
在3期VALOR研究（n=108）中，患者以 2:1 的比例隨機分配，接受Qalsody 100 mg（n=72）或安慰劑（n=36）的治療，爲期24周。
The primary efficacy endpoint was the change from baseline to Week 28 in the ALS Functional Ratings Scale-Revised total score.
主要療效終點是ALS功能評級量表修訂後的總分從基線改爲第28周。
The results numerically favored tofersen but were not statistically significant.
結果在數值上有利於託弗森，但在統計學上並不顯著。
At Week 28, mean plasma neurofilament light chain (NfL), a marker of axonal injury and neurodegeneration, was reduced by 55% in the tofersen-treated participants (ITT), compared to a 12% increase with placebo.
在第28周，接受tofersen治療的參與者（ITT）的平均血漿神經絲輕鏈（nFL）（軸突損傷和神經變性的標誌）減少了55％，而安慰劑的平均血漿神經絲輕鏈（nFL）增加了12％。
About 330 people with SOD1-ALS have received Qalsody across 18 EU countries through the Biogen early access program.
大約 330 人有 SOD1-ALS已通過Biogen搶先體驗計劃在18個歐盟國家接待了Qalsody。
Last April, the FDA granted accelerated approval for Qalsody. In 2023, the drug generated sales of $5.9 million.
去年4月，美國食品和藥物管理局加快了對Qalsody的批准。2023年，該藥物的銷售額爲590萬美元。
Biogen licensed Qalsody from Ionis Pharmaceuticals Inc (NASDAQ:IONS) under a collaborative development and license agreement. Ionis discovered Qalsody.
根據合作開發和許可協議，Biogen向愛奧尼斯製藥公司（納斯達克股票代碼：IONS）許可了Qalsody的許可。愛奧尼斯發現了 Qalsody。
In addition to the ongoing open-label extension of the Phase 3 VALOR study, Qalsody is being studied in the Phase 3 ATLAS study to evaluate whether Qalsody can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity.
除了正在進行的3期VALOR研究的開放標籤延期外，Qalsody的3期ATLAS研究還在研究中，以評估Qalsody在症狀前患者中啓動時是否可以延緩臨床發作 SOD1 基因突變和疾病活動的生物標誌物證據。
Price Action: BIIB shares are up 1.90% at $224.26 at the last check on Friday.
價格走勢：在週五的最後一次支票中，BIIB股價上漲1.90％，至224.26美元。
譯文內容由第三人軟體翻譯。