The U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals’ (NASDAQ:ALNY) lead drug candidate AMVUTTRA (Vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
The regulatory approval was supported by positive clinical results from HELIOS-A Phase 3 study that evaluated the efficacy and safety of AMVUTTRA across a diverse group of patients with hATTR amyloidosis with polyneuropathy.
During the Phase 3 clinical trial, Vutrisiran met primary and all secondary endpoints, with Significant Improvement in Polyneuropathy, Quality of Life and Gait Speed Relative to External Placebo.
Yvonne Greenstreet, MBChB, Chief Executive Officer, commented : “Twenty years ago, Alnylam was founded with the bold vision for RNA interference to make a meaningful impact on the lives of people around the world in need of new approaches to address serious diseases with significant unmet medical needs, such as hATTR amyloidosis. Today, AMVUTTRA has the potential to change the standard of care for people living with the polyneuropathy of this devastating disease.”
The company said that Vutrisiran is the first and only FDA-approved Treatment Demonstrating Reversal in Neuropathy Impairment with Subcutaneous Administration Once Every Three Months.
Alnylam anticipates commercial launch of AMVUTTRA (Vutrisiran) in the month of July, 2022, with Value-Based Agreements to Accelerate Access.
Alnylam Pharmaceuticals is trading higher 6.5 percent at $136 in the after hours session.
