FDA re­jects Al­ny­lam’s On­pat­tro la­bel ex­pan­sion af­ter ad­comm vot­ed in fa­vor

The FDA has re­ject­ed Al­ny­lam Phar­ma­ceu­ti­cals’ ex­pan­sion plans for its siR­NA in­jec­tion On­pat­tro, serv­ing a com­plete re­sponse let­ter for the treat­ment of AT­TR car­diomy­opa­thy.

The Mon­day morn­ing sur­prise no-go comes weeks af­ter the agency’s pan­el of out­side ad­vi­sors had vot­ed 9 to 3 in fa­vor of the drug’s ben­e­fits out­weigh­ing its risks. The FDA raised ques­tions at the ad­comm on the ef­fect size in this in­di­ca­tion, and in­de­pen­dent ex­perts had agreed that On­pat­tro’s ben­e­fit was small, but risks were small­er for the pa­tients who ex­pe­ri­ence mal­formed pro­tein buildup in the heart.

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