Alnylam Pharmaceuticals has scored FDA approval for Amvuttra (vutrisiran), the first and only approved treatment demonstrating reversal in neuropathy impairment.
The drug is injected once every three months and is now greenlighted for the treatment of the polyneuropathy of transthyretin-mediated (hATTR) amyloidosis in adults. While rare, hATTR is a rapidly progressive and fatal disease that presents debilitating polyneuropathy episodes, and there are few treatments for it.
The recent approval hinged on data from a phase 3 study, HELIOS-A, in which the drug significantly improved the signs of neuropathy, and more than 50% of patients experienced some reversal of their symptoms. The study met all the secondary endpoints in the most recently reported data, demonstrating a promising safety and tolerability profile as there were no drug-related discontinuous or deaths.
Amvuttra is under review by other international regulatory agencies for hATTR treatment and is also being evaluated in the HELIOS-B phase 3 study for the treatment of patients with ATTR amyloidosis with cardiomyopathy, including both hATTR and wild-type ATTR (wtATTR) amyloidosis.
