Weeks after a landmark approval in the USA, Alnylam Pharmaceuticals’ (Nasdaq: ALNY) RNA interference (RNAi) therapeutic has been approved by the European Commission (EC).
Onpattro (patisiran) has been granted marketing authorization for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage one or stage two polyneuropathy. As an RNAi therapeutic, it targets the faulty protein that is causing the disease rather than treating the symptoms.
Theresa Heggie, head of Europe for Alnylam, called the approval “the start of a new chapter in the treatment of this rare, rapidly progressive, fatal disease,” but securing routine reimbursement for patients across the continent might not be an easy process given Onpattro’s potential price.
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