FDA approves polyneuropathy treatment for adults
The FDA has approved Amvuttra, an RNA interference therapeutic for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Amvuttra (vutrisiran, Alnylam Pharmaceuticals) is administered through subcutaneous injection once every 3 months, the company said in a press release. The FDA approval was based on positive results from a global, randomized phase 3 study, which assessed the safety and efficacy of vutrisiran in participants with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy.

Source: Adobe Stock.
Source: Adobe Stock.
“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,” Alnylam CEO Yvonne Greenstreet, MBChB, said in the release. “Today, vutrisiran has the potential to change the standard of care for people living with the polyneuropathy of this devastating disease.”

According to the release, 164 patients with hATTR amyloidosis were randomized 3:1 to receive 25 mg of vutrisiran by subcutaneous injection once every 3 months (n = 122) or 0.3 mg/kg of patisiran via IV infusion once every 3 weeks for 18 months (n = 42). The vutrisiran group was also compared with a placebo group (n = 77).

Among 114 participants who received vutrisiran, there was a 2.2-point mean decrease, or improvement, in the modified Neuropathy Impairment Score +7 after 9 months of treatment, compared with a 14.8-point mean increase reported in the placebo group. In addition, 50% of patients who received vutrisiran experienced improvement in neuropathy impairment compared with baseline.

All safety and tolerability endpoints were met after 9 months of treatment with vutrisiran. The most reported adverse events were arthralgia (11%), dyspnea (7%) and a vitamin A decrease (7%).

“We are so thankful to the patients, families and investigators involved in making Amvuttra a reality for the hATTR amyloidosis community,” Greenstreet said.