Complete New Drug Application for FAP Therapy Patisiran Submitted to FDA

Alnylam Pharmaceuticals has submitted a complete new drug application (NDA) for its investigational therapy patisiran to the U.S. Food and Drug Administration for the treatment of adults with hereditary ATTR amyloidosis, also called familial amyloid polyneuropathy (FAP).

The application has been submitted on a rolling basis — a practice that aims to speed up the regulatory review of treatments for serious conditions. The company has asked the FDA to grant the application priority review, which could cut the review process from 10 to six months.

Alnylam, together with its partner Sanofi Genzyme, started the submission in November, after a Phase 3 trial (NCT01960348) showed robust responses among treated patients.

“In November, we reported promising results from the APOLLO Phase 3 study of patisiran which showed amelioration of neurological impairment, improved quality of life, and reduced disease symptoms and disability in hATTR amyloidosis patients with polyneuropathy,” Eric Green, vice president and general manager of the TTR program at Alnylam, said in a press release.

“In less than 90 days from first reporting the top-line data, the completion of Alnylam’s first NDA submission is a historic event, bringing patisiran one step closer to patients living with hATTR amyloidosis. We look forward to the exciting prospect of introducing the first FDA-approved RNAi therapeutic, marking the arrival of a new class of medicines,” Green added.

Patisiran is a so-called RNAi drug that prevents the faulty TTR protein, which causes the disease, to form.

The drug’s regulatory path has been lined with efforts to speed its development. The drug was previously granted fast track and breakthrough therapy designations from the FDA, and recently received an expanded orphan drug status for ATTR amyloidosis.

Meanwhile, the treatment has been granted accelerated assessment by the European Medicines Agency. Alnylam and Sanofi Genzyme are working to file an application for the drug’s approval in the EU by year’s end.

“People living with hATTR amyloidosis currently have no FDA-approved options to treat this devastating, often fatal disease,” said Isabelle Lousada, president and CEO of the Amyloidosis Research Consortium (ARC).

“The ARC applauds this significant milestone for patisiran and we are hopeful this therapy will be approved for patients in the U.S. who are desperately awaiting treatment options,” she said.