Alnylam Announces Plan to Initiate Rolling Submission of a New Drug Application

- October 15th, 2018

Alnylam Pharmaceuticals (Nasdaq:ALNY), the leading RNAi therapeutics company, today announced that in consultation with the U.S. Food and Drug Administration (FDA), the Company plans to pursue a full approval based on the complete results of the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of … Continued

Alnylam Pharmaceuticals (Nasdaq:ALNY), the leading RNAi therapeutics company, today announced that in consultation with the U.S. Food and Drug Administration (FDA), the Company plans to pursue a full approval based on the complete results of the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), rather than filing based on the interim Phase 3 results.

As quoted in the press release:

“The AHPs are devastating diseases and our goal is to help address the significant unmet need that exists today for people living with AHP. Based on our positive Phase 1/2 clinical results presented earlier this year and the positive interim analysis results from the ENVISION Phase 3 trial, we’re encouraged by givosiran’s potential to make a difference in the lives of AHP patients,” said Akin Akinc, Ph.D., Vice President and General Manager, Givosiran Program at Alnylam. “Our constructive discussions with the FDA led to our decision to pursue a full approval path with complete ENVISION study results, including porphyria attack data, which are expected much earlier than originally planned. The FDA has also agreed to a rolling submission of the NDA which will begin this year. This filing plan creates the potential to achieve full approval as rapidly as possible and aligns with our strategy in all other countries.”

Click here to read the full press release.

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