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Alnylam Plans To Pursue Full Approval Of Givosiran, Rolling Submission Of NDA


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(RTTNews.com) - Alnylam Pharmaceuticals Inc. ( ALNY ), an RNAi therapeutics company, said that in consultation with the U.S. Food and Drug Administration, it plans to pursue a full approval of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 or ALAS1 for the treatment of acute hepatic porphyria or AHP.

The full approval will be based on the complete results of the ENVISION Phase 3 study of givosiran, rather than filing based on the interim Phase 3 results.

The FDA has also agreed to a rolling submission of a New Drug Application or NDA, which will be initiated in 2018 with full clinical sections submitted in mid-2019, assuming positive study results.

"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, Vice President and General Manager, Givosiran Program at Alnylam.

Alnylam previously reported positive topline results from the interim analysis of the ENVISION Phase 3 study of givosiran demonstrating a statistically significant reduction in urinary ALA levels, a surrogate biomarker that is reasonably likely to predict clinical benefit.

Alnylam said it continues to dose patients in the ongoing ENVISION study, where enrollment was completed ahead of schedule with 94 AHP patients. The company expects to report topline full study results of the primary endpoint - the annualized attack rate after six months of treatment - in early 2019.


Read the original article on RTTNews (http://www.rttnews.com/2943449/alnylam-plans-to-pursue-full-approval-of-givosiran-rolling-submission-of-nda.aspx)


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Referenced Symbols: ALNY



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