Al­ny­lam nears FDA fil­ing for 4th RNAi ther­a­py vutrisir­an af­ter late-stage da­ta back use in pro­tein mis­fold­ing dis­or­der

With three ap­proved RNAi ther­a­peu­tics al­ready on the mar­ket, Al­ny­lam has se­cured its place as the leader in that rare dis­ease-fo­cused field. Now, a fourth can­di­date is rapid­ly ap­proach­ing reg­u­la­tors’ desks, and Al­ny­lam hopes it will com­ple­ment an­oth­er of its drugs tar­get­ing a rare pro­tein mis­fold­ing dis­or­der.

On Thurs­day, Al­ny­lam un­veiled da­ta from its open-la­bel Phase III He­lios-A study show­ing vutrisir­an topped a his­tor­i­cal place­bo con­trol in re­duc­ing the symp­toms of polyneu­ropa­thy tied to transthyretin-me­di­at­ed (AT­TR) amy­loi­do­sis from base­line af­ter nine months of treat­ment. On sev­er­al sec­ondary end­points, in­clud­ing changes in qual­i­ty of life and pa­tient gait speed, vutrisir­an al­so beat out his­tor­i­cal place­bo.

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