Ahead of ad­comm, FDA ques­tions ef­fi­ca­cy of Al­ny­lam's patisir­an in dis­abling rare con­di­tion AT­TR-CM

The FDA’s Car­dio­vas­cu­lar and Re­nal Drugs Ad­vi­so­ry Com­mit­tee will meet to­mor­row to dis­cuss the ef­fi­ca­cy of Al­ny­lam’s On­pat­tro (patisir­an) for the treat­ment of car­diomy­opa­thy of wild-type or hered­i­tary transthyretin-me­di­at­ed amy­loi­do­sis (AT­TR-CM) in adults.

While Al­ny­lam has tout­ed the treat­ment’s sta­tis­ti­cal sig­nif­i­cance in this set­ting, hit­ting its pri­ma­ry end­point and meet­ing its first sec­ondary end­point in a piv­otal tri­al, the FDA is rais­ing ques­tions on the ef­fect size. If ap­proved in this in­di­ca­tion, On­pat­tro would have to com­pete with Pfiz­er’s Vyn­damax (tafamidis) cap­sules, which FDA ap­proved in 2019 as the first treat­ment for AT­TR-CM.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER