Alnylam’s lumasiran is latest RNAi therapeutic to hit pivotal study goal
Lumasiran’s success in a pivotal study to treat a form of hyperoxaluria represents another step toward Alnylam meeting its updated goal of having four approved medicines by the end of 2020 -- all since the summer of 2018.
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) said Tuesday that RNAi therapeutic lumasiran met the primary endpoint of the Phase III ILLUMINATE-A trial to treat primary hyperoxaluria type 1 (PH1). The company is aiming to submit regulatory applications in the U.S. and EU early next year. The therapy could receive expedited review, given that it has Orphan Drug designations in the U.S. and EU, breakthrough therapy designation from FDA, and PRIority MEdicines (PRIME) designation from EMA...
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