Rare Disease Report

EMA Grants Alnylam Accelerated Assessment for Potential hATTR Amyloidosis Treatment

NOVEMBER 13, 2017
Mathew Shanley
Patisiran, an investigational RNAi therapeutic being developed by Alnylam, has been granted an accelerated assessment by the European Medicines Agency (EMA).

The drug targets transthyretin (TTR) and is designed to treated hereditary transthyretin amyloidosis (hATTR) amyloidosis. With the accelerated assessment, Alnylam can expect the review timeline to be reduced from the typical 210 days to 150 once the marketing authorization application (MAA) is filed and validated in the European Union (EU).

In hATTR, the misfolding of TTR proteins leads to a deposition of amyloid fibrils in different organs. In patients with the genetic, life-threatening condition, the buildup of TTR fibrils interferes with the intended functionality in a variety of tissues and organs, leading to further damage, a poor quality of life, and eventually death.

Parts of the body that are most commonly affected in patients with hATTR include: peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system (CNS), thyroid, and bone marrow.

“We are pleased the CHMP has granted accelerated assessment for patisiran and believe this underscores the urgent need to improve outcomes for patients living with hATTR amyloidosis,” said Eric Green, Vice President and General Manager of the TTR program in a press release. “The results from the APOLLO Phase 3 study in hATTR amyloidosis patients with polyneuropathy provide evidence that patisiran has the potential to improve neurologic impairment, quality of life, and cardiac parameters. With this accelerated assessment award from the EMA, we hope to be able to make this promising treatment option available to patients in Europe sooner.”

The accelerated assessment was granted partly because of promising results from the APOLLO Phase 3 study in which 225 patients were enrolled into a randomized, double-blind, placebo-controlled, global trial designed to evaluate the safety and efficacy of patisiran in hATTR amyloidosis patients with polyneuropathy. At the ATTR Amyloidosis Meeting in Paris earlier this month, data from the study was presented and exhibited a profound improvement in patients when treated with Alnylam’s compound. Michael Polydefkis, MD, Professor of Neurology at Johns Hopkins University noted that the drug’s capabilities can “open the door to change people’s lives.”

The primary outcome measure of the APOLLO study was modified Neuropathy Impairment Score +7 (mNIS+7), and the study observed that after 18 months, clinical benefit was found in patients across all defined subgroups when treated with patisiran.

“The news that patisiran has been accepted for accelerated assessment by the EMA signals to us that everyone involved recognizes the serious impact hereditary ATTR amyloidosis has on the lives of people affected by this devastating, progressive disease,” said Eric Low, Chairman, Amyloidosis Research Consortium UK. “The need for new treatment options is urgent. The patient community really welcomes when everything possible is being done to assess promising new treatments quickly and efficiently to see if patients may benefit earlier.”

As it pertains to patisiran in the U.S., Alnylam plans to file a New Drug Application with the Food and Drug Administration (FDA) by the end of 2017. Regulatory filings for the drug in other countries including Japan and Brazil are currently being prepared by the company.

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