Sarepta’s Big DMD Approval, Alnylam’s Clinical Win and ADA Highlights

2024-06-26

临床3期基因疗法临床结果上市批准临床2期

The biggest news of the last week was easily the expanded approval of Sarepta’s Duchenne muscular dystrophy gene therapy, Elevidys. CBER Director Peter Marks again overruled FDA staff members and review teams to grant the therapy full approval and a broad label expansion despite its missing the primary endpoint in a Phase III confirmatory study. This approval has us thinking about other big FDA decisions to watch this year, first and foremost, Eli Lilly’s anti-amyloid antibody donanemab. If given the green light, donanemab will be a direct competitor to Biogen and Eisai’s Leqembi, also a disease-modifying anti-amyloid antibody. Beyond that, Verona Pharma is expecting a decision Wednesday on ensifentrine, which could be the first novel mechanism for chronic obstructive pulmonary disease in over a decade, and in August, the FDA is set to decide on Lykos’ MDMA-assisted PTSD therapy, which an advisory committee voted against earlier this month. Then this week, Alnylam Alnylam scored big with a Phase III win in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). In what Alnylam CMO Pushkal Garg called “overwhelmingly positive data,” Amuvttra significantly lowered the risk of death and recurrent cardiovascular events in patients with ATTR-CM. Meanwhile, biopharma conference season continues with the American Diabetes Association Diabetes Association’s annual conference held this past weekend. Eli Lilly’s blockbuster drug Zepbound “significantly improved” disease severity in patients with obesity and sleep apnea, and Altimmune racked up a Phase II victory for pemvidutide, which was effective at helping patients lose weight while retaining lean muscle. Greg Slabodkin is the news editor at BioSpace. You can reach him at greg.slabodkin@biospace.com. Follow him on LinkedIn.  Heather McKenzie is a senior editor at BioSpace. You can reach her at heather.mckenzie@biospace.com. Follow her on LinkedIn. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn. > Listen on Spotify > Listen on Apple Podcasts > Listen on Amazon Music > Listen on iHeart

更多内容，请访问原始网站

文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。

机构