The US Food and Drug Administration has approved Alnylam's Onpattro, an RNAi-based therapy, Stat News reports.
"This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms," FDA Commissioner Scott Gottlieb says in a statement. "In this case, the effects of the disease cause a degeneration of the nerves, which can manifest in pain, weakness, and loss of mobility."
The agency approved Onpattro (patisiran) for the treatment of peripheral nerve disease in adults with hereditary transthyretin-mediated amyloidosis, a condition in which amyloid protein builds up in nerves, the heart, and other organs. This scope, Stat News notes, is narrower than what European regulators described, as it doesn't encompass the drug's effect on cardiac damage caused by the condition.
"Until today, there were no approved medications to treat these patients in the United States," Alnylam CEO John Maraganore said on a conference call, according to Stat News. "We do look forward to working with the FDA to expand the Onpattro label in the future."
Stat News adds the annual average list price of the drug will be $450,000.