Friedreich's ataxia is a genetic, debilitating and life-shortening neuromuscular disease1
Milestone highlights Biogen's growing portfolio in rare diseases and focus on addressing unmet needs of patients living with neuromuscular diseases
CAMBRIDGE, Mass., Dec. 15, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended marketing authorization for SKYCLARYS (omaveloxolone) for the treatment of Friedreich's ataxia (FA) in people aged 16 years and older. If approved by the European Commission (EC), SKYCLARYS will be the first treatment authorized within the European Union for this rare, genetic, progressive neuromuscular disease.1 If approved, omaveloxolone will be marketed as SKYCLARYS.
"The CHMP's recommendation for SKYCLARYS is a significant milestone toward our goal of bringing a treatment that slows the progression of FA to the patient community in the region," said Priya Singhal, M.D., M.P.H., Head of Development at Biogen. "Upon approval of SKYCLARYS, we look forward to leveraging Biogen's rare disease expertise and capabilities to bring this groundbreaking treatment to patients in the European Union living with this debilitating disease."
The CHMP's positive opinion for SKYCLARYS is based on efficacy and safety data from the placebo-controlled MOXIe Part 2 trial. At the end of the 48-week MOXIe Part 2 study, patients who received SKYCLARYS had less physical impairment compared to patients who received placebo, as measured by the modified Friedreich Ataxia Rating Scale (mFARS). Improvements across subscales of mFARS, including upright stability, lower limb coordination, ability to swallow and upper limb coordination, were also observed in patients treated with SKYCLARYS compared to placebo. Additional data was provided from a post hoc, propensity-matched analysis in which patients treated with SKYCLARYS in MOXIe (Parts 1 and 2) had lower mFARS score at 3 years, as compared to a matched natural history group. The most common side effects are increased liver enzymes, decreased weight and appetite, nausea, vomiting, diarrhea, headache, fatigue, oropharyngeal and back pain, muscle spasms, and influenza.
The CHMP's recommendation for SKYCLARYS will now be reviewed by the EC for marketing authorization in the European Union with a final decision expected in the first quarter of 2024. The U.S. Food and Drug Administration (FDA) approved omaveloxolone, marketed as SKYCLARYS, in February 2023 for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.
About SKYCLARYS (omaveloxolone)
SKYCLARYS (omaveloxolone) is an oral, once-daily medication indicated for the treatment of Friedreich's ataxia (FA) in adults and adolescents aged 16 years and older in the U.S. SKYCLARYS has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration. The European Commission has granted Orphan Drug designation in Europe to omaveloxolone for the treatment of FA.
About Friedreich's Ataxia
Friedreich's ataxia (FA) is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder. It is the most common inherited ataxia.2,3 Early symptoms of FA, such as progressive loss of coordination, muscle weakness and fatigue, typically appear in childhood and can overlap with other diseases.4 Most people living with FA will need to use a wheelchair within 10-20 years of their first symptoms.2 The reported average age of death for FA patients is just 37 years old, although with appropriate and targeted care, individuals may live many years after confinement to a wheelchair.5-7
About Biogen
Founded in 1978, Biogen is a leading global biotechnology company that has pioneered multiple breakthrough innovations including a broad portfolio of medicines to treat multiple sclerosis, the first approved treatment for spinal muscular atrophy, two co-developed treatments to address a defining pathology of Alzheimer's disease, the first treatment to target a genetic form of ALS, the first oral treatment approved for postpartum depression, and the first approved treatment for Friedreich's ataxia. Biogen is advancing a pipeline of potential novel therapies across neurology, neuropsychiatry, specialized immunology and rare diseases and remains acutely focused on its purpose of serving humanity through science while advancing a healthier, more sustainable and equitable world.
馬薩諸塞州劍橋，2023年12月15日（GLOBE NEWSWIRE）——Biogen Inc.（納斯達克股票代碼：BIIB）宣佈，歐洲藥品管理局（EMA）人用藥品委員會（CHMP）建議批准用於治療16歲及以上人群弗裏德賴希共濟失調（FA）的SKYCLARYS（奧馬維洛索龍）。如果獲得歐盟委員會（EC）的批准，SKYCLARYS將成爲歐盟範圍內批准的首款治療這種罕見、遺傳、進行性神經肌肉疾病的藥物。1 如果獲得批准，奧馬維洛酮將作爲SKYCLARYS上市。
Biogen研發主管Priya Singhal表示：“CHMP對SKYCLARYS的建議是朝着向該地區患者社區提供一種減緩FA進展的治療方法的目標邁進的重要里程碑。”“在獲得SKYCLARYS批准後，我們期待利用Biogen的罕見病專業知識和能力，爲患有這種使人衰弱的疾病的歐盟患者提供這種開創性的治療方法。”
CHMP對SKYCLARYS的正面評價是基於安慰劑對照的MoxiE第2部分試驗的療效和安全性數據。根據修改後的弗裏德賴希共濟失調評級量表（mFars），在爲期48周的MoxiE第2部分研究結束時，與接受安慰劑的患者相比，接受SKYCLARYS治療的患者的身體損傷較少。與安慰劑相比，在接受SKYCLARYS治療的患者中，還觀察到包括直立穩定性、下肢協調性、吞嚥能力和上肢協調性在內的各子量表都有所改善。其他數據來自一項事後傾向匹配的分析，在該分析中，與匹配的自然史組相比，在MoxiE（第1部分和第2部分）中接受SKYCLARYS治療的患者在3年時的mFars分數較低。最常見的副作用是肝酶升高、體重和食慾下降、噁心、嘔吐、腹瀉、頭痛、疲勞、口咽和背部疼痛、肌肉痙攣和流感。
CHMP對SKYCLARYS的建議現在將由歐盟委員會審查，以獲得歐盟的上市許可，最終決定預計將在2024年第一季度作出。美國食品藥品監督管理局 (FDA) 於 2023 年 2 月批准以 SKYCLARYS 的名義上市的 omaveloxolone 用於治療 16 歲及以上成人和青少年的弗裏德賴希共濟失調。
關於 SKYCLARYS（omaveloxolone）
SKYCLARYS（omaveloxolone）是一種每日一次的口服藥物，適用於治療美國16歲及以上成人和青少年的弗裏德賴希共濟失調（FA）。SKYCLARYS已獲得美國食品藥品監督管理局頒發的孤兒藥、快速通道和罕見兒科疾病稱號。歐盟委員會已在歐洲授予用於治療FA的奧馬韋洛酮的孤兒藥稱號。
關於弗裏德賴希的共濟失調
弗裏德賴希共濟失調（FA）是一種罕見的遺傳性、縮短壽命、衰弱和退行性神經肌肉疾病。它是最常見的遺傳性共濟失調。2,3 FA的早期症狀，例如漸進性失去協調能力、肌肉無力和疲勞，通常出現在兒童時期，並可能與其他疾病重疊。4 大多數FA患者在首次出現症狀後的10-20年內需要使用輪椅。2 據報告，足協患者的平均死亡年齡僅爲37歲，儘管如果有適當和有針對性的護理，個人在坐輪椅後可能會活很多年 chair.5-7
關於 Biogen
Biogen成立於1978年，是一家全球領先的生物技術公司，開創了多項突破性創新，包括治療多發性硬化的廣泛藥物組合、第一種批准的脊髓性肌萎縮症治療藥物、兩種針對阿爾茨海默氏病決定性病理學的共同開發的治療方法、第一種針對ALS遺傳形式的治療藥物、第一種獲准用於產後抑鬱症的口服療法，以及第一種批准的弗裏德賴希共濟失調治療藥物。Biogen正在推進神經病學、神經精神病學、特種免疫學和罕見病等一系列潛在的新型療法，並始終堅定不移地致力於通過科學服務人類，同時推動更健康、更可持續和更公平的世界。
