Alnylam's Phase III Results For Vutrisiran Are 'Foundation' For Amyloidosis Expansion
FDA Filing Anticipated In Early 2021
The results are in for hATTR amyloidosis with polyneuropathy, while results for hereditary and wild-type ATTR with cardiomyopathy are expected later this year.
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The company won approval in hATTR amyloidosis polyneuropathy for the RNAi drug, which offers quarterly subcutaneous dosing versus Onpattro’s I.V. administration every three weeks.
The company presented 18-month data at a conference that it thinks will support strong uptake. European regulators required the 18-month data for filing; an FDA decision based on 9-month data is expected in April.
Alnylam’s second NDA for an RNA interference treatment for the rare disease rests on a comparison of vutisiran patients in the Phase III HELIOS-A study against an external control arm of placebo patients from Onpattro’s APOLLO study.