Alnylam’s Patisiran ATTR-CM Opportunity Boosted By New APOLLO-B Data

Phase III results for Onpattro (patisiran) in ATTR cardiomyopathy pave a path to approval – though the competitive profile is in question – and foreshadow potential success for follow-on drug vutrisiran.

stethoscope and red heart on blue background
ATTR cardiomyopathy significantly increases the market size for Onpattro • Source: Shutterstock

The full results of the Phase III APOLLO-B clinical trial testing Alnylam Pharmaceuticals Inc.’s Onpattro (patisiran) in transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) should support approval, but whether or not the data give it a competitive profile remains to be seen. One upside to the results, however, was the potential readthrough to the ongoing Phase III HELIOS-B trial of follow-on drug Amvuttra (vutrisiran) in ATTR-CM.

Both drugs are approved for ATTR polyneuropathy, an indication much smaller than the 250,000-patient global ATTR-CM market, but Onpattro is administered intravenously and Amvuttra is a subcutaneous injection. Given the relative ease of Amvuttra’s administration, analysts expect it to be the higher-selling of the two products

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