(RTTNews.com) - Alnylam Pharmaceuticals Inc. ( ALNY ) said it has achieved full patient accrual in its Envision Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 or ALAS1 for the treatment of acute hepatic porphyrias, or AHPs.
Enrollment was completed ahead of schedule, with 94 AHP patients randomized across 36 sites in 18 countries, surpassing the initial target of approximately 75 patients due to high patient demand.
The Envision Phase 3 trial is a randomized, double-blind, placebo-controlled, global, multi-center study to evaluate the efficacy and safety of givosiran in patients with a documented diagnosis of AHPs.
Patients were randomized on a 1:1 basis to receive 2.5 mg/kg of givosiran or placebo subcutaneously administered monthly, over a 6-month treatment period.
The primary endpoint is the annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit or hemin administration at home over the 6-month treatment period.
Alnylam Pharma 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 or 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 as well as FDA review of the program at the time of interim analysis and assuming positive results and acceptable safety, Alnylam continues to expect to submit an NDA at or around year-end 2018, seeking an accelerated approval.
Read the original article on RTTNews (http://www.rttnews.com/2928340/alnylam-pharma-completes-enrollment-in-envision-phase-3-study-of-givosiran.aspx)
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