Alnylam Completes Enrollment in ENVISION Phase 3 Study of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias

Alnylam Pharmaceuticals (Nasdaq:ALNY), the leading RNAi therapeutics company, today announced that it has achieved full patient accrual in its ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHPs).

As quoted in the press release:

The Company reiterated its previous guidance that it expects to report topline results of the interim analysis by the end of September in support of a potential accelerated approval, and topline results on the primary endpoint of annualized attack rate after six months of treatment in early 2019. The interim analysis is based on lowering of urinary aminolevulinic acid (ALA) levels from approximately 30 patients at three months of treatment as a surrogate biomarker that is reasonably likely to predict clinical benefit. Pending Company and FDA review of the program at the time of interim analysis and assuming positive results and acceptable safety, the Company continues to expect to submit an NDA at or around year-end 2018, seeking an accelerated approval.

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