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.

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