Reuters Health Information: Alnylam's gene-silencing drug shows efficacy in late-stage trial

Alnylam's gene-silencing drug shows efficacy in late-stage trial

Last Updated: 2018-09-27

By Reuters Staff

(Reuters) - Alnylam Pharmaceuticals Inc said on Thursday its gene-silencing drug for a rare, painful genetic disease was found to be effective in a late-stage trial, putting the firm on track for a faster approval from U.S. regulator.

The drug, givosiran, showed significant reduction in urinary aminolevulinic acid (ALA) in patients with acute hepatic porphyria, the company said, citing interim data from the trial.

Acute hepatic porphyria (AHP) is a family of rare diseases that affects the liver and has no approved treatments.

"The AHPs are devastating diseases in which patients suffer from both debilitating neurovisceral attacks as well as chronic pain and fatigue," Dr. Akshay Vaishnaw, President of Research and Development at Alnylam, said in a news release. "We are pleased and encouraged that the interim analysis of the ENVISION Phase 3 study demonstrated that givosiran treatment was associated with statistically significant lowering of ALA, a disease biomarker reasonably likely to predict clinical benefit."

The company plans to apply for accelerated approval by the end of the year, which will allow the firm to launch the drug in the United States even when the results of the full trial are pending.

Last month, the U.S. FDA approved Alnylam's gene-silencing drug Onpattro (patisiran) for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

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