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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