Rare Disease Report

Patisiran NDA Accepted, hATTR Drug Granted Priority Review

FEBRUARY 01, 2018
Mathew Shanley
Alnylam Pharmaceuticals, Inc. announced this afternoon that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary (hATTR) amyloidosis.

Additionally, the FDA granted Alnylam’s request for Priority Review and set a Prescription Drug User Fee Act (PDUFA) action date of August 11, 2018.

In November, one month after Alnylam reported that its Phase 3 clinical trial of patisiran met its primary endpoint in hATTR amyloidosis with polyneuropathy, the company initiated the submission of the NDA.

“We are delighted to have received Priority Review status for patisiran and look forward to continuing to work closely with the FDA during the review process,” said Eric Green, Vice President and General Manager of the TTR program at Alnylam in a press release. “Together with the previous grant of Breakthrough Therapy Designation, the Priority Review underscores the robust evidence of the potential of patisiran and the urgent need for an FDA-approved treatment for hATTR amyloidosis, an aggressive, rapidly progressive, debilitating and fatal disease.”

Previously, the FDA had granted Fast Track Designation, Breakthrough Therapy Designation, and an expanded Orphan Drug Designation to the investigational RNAi therapeutic for ATTR amyloidosis.

In Europe, the drug had been granted accelerated assessment, and last week, the company announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA), initiating their review.

ATTR amyloidosis is a genetic condition that progresses rapidly and can result in the misfolding of TTR proteins. Over time, these mutations encourage the development of amyloid fibrils that can be deposited into different organs. Patisiran is being developed to target TTR, thus reducing accumulation of the misfolded protein.

The disease is characterized by a clinical presentation that includes sensory and motor, autonomic and cardiac symptoms.

In an interview with Rare Disease Report last year, Michael Polydefkis, M.D., Professor of Neurology at Johns Hopkins University and lead investigator on the Phase 3 APOLLO study emphasized the potential of patisiran: “This opens the door to change people’s lives,” he said. “This drug provides an opportunity to stop, and maybe even reverse, what was once a fatal disease.”

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