The U.S. Food and Drug Administration on Aug. 10 OK’d the first-ever rare disease treatment that uses RNA interference, a technique which works to neutralize disease-causing genes.
The agency gave the drug, Alnylam’s patisiran (Onpattro), approval for treatment of neuropathy, according to STAT.
“Until today, there were no approved medications to treat these patients in the United States,” Alnylam CEO John Maraganore said Friday. “We do look forward to working with the FDA to expand the Onpattro label in the future.”
According to STAT, the annual average list price for the drug will be $450,000, a figure which rests in a range predicted by analysts.
FDA Commissioner Scott Gottlieb in a press release said the drug’s approval is “part of a broader wave of advances that allow us to treat disease by actually targeting the root cause … .”
