June 28, 2022
Winter Garden, FL
The FDA recently approved a new sub-cutaneous, specialty therapy, Amvuttra (vutrisiran) from Alnylam Pharmaceuticals, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
Hereditary ATTR including polyneuropathy is a rare condition that has had few treatment options. It affects about 50,000 people worldwide. The disease is rapidly progressive with a high mortality rate.
Amvuttra is administered via subcutaneous injection once every three months (quarterly). The prescribing information for the product states that it should be administered only by a medical professional.
Amvuttra joins the market with another Alnylam product, Onpattro, with the same indication. Amvuttra is sub-q, whereas Onpattro is infused. Amvuttra has a potential to treat another form of ATTR that affects the heart. That indication, called ATTR cardiomyopathy, may reach $1.8 billion in global sales by 2026.
Alnylam is pricing Amvuttra at an annual list price of $463,500. Given the small patient population in the US, it is expected that Amvuttra will launch through limited distribution (direct-to-office). By way of reference, Onpattro launched in 2018 via LD provided by US Bioservices and Orsini Healthcare.
Alnylam Announces FDA Approval of Amvuttra (vutrisiran)
RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
CAMBRIDGE, Mass.– Jun 13, 2022 — Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) approved Amvuttra (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease with debilitating polyneuropathy manifestations, for which there are few treatment options. The FDA approval is based on positive 9-month results from the HELIOS-A Phase 3 study, where Amvuttra significantly improved the signs and symptoms of polyneuropathy, with more than 50 percent of patients experiencing halting or reversal of their disease manifestations.
“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,” said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam Pharmaceuticals. “We are so thankful to the patients, families and investigators involved in making Amvuttra a reality for the hATTR amyloidosis community. As the fifth RNAi therapeutic developed by Alnylam to receive regulatory approval in less than four years, we believe Amvuttra represents an important milestone that brings us one step closer to achieving our P 5 x25 goals aimed at Alnylam’s transition to a leading biotech company.”