The U.S. FDA has approved Alnylam Pharmaceuticals’ Onpattro, making it the first medicine specifically cleared to treat a rare and deadly disease called hereditary transthyretin amyloidosis (hATTR).
The FDA decision marks the first-ever approval for a medicine that uses RNA interference (RNAi), a method cells can use to silence a gene before it makes a harmful protein.
Hereditary transthyretin (TTR)-mediated amyloidosis is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene.
Onpattro was reviewed by the FDA under Priority Review and had previously been granted Breakthrough Therapy and Orphan Drug Designations.
As reported by Xconomy, Alnylam asaid the drug’s average list price–which doesn’t include rebates to insurers—is $450,000 per patient, per year. The average net price, taking rebates into account, is $345,000 per year, Alnylam said.
Read the press release