Rare Disease Report

EMA Accepts Marketing Authorization Application for Patisiran

JANUARY 26, 2018
Mathew Shanley
In November, Alnylam was granted accelerated assessment by the European Medicines Agency (EMA) for Patisiran, its potential therapy for hereditary transthyretin amyloidosis (hATTR) amyloidosis. Today, the company announced that its Marketing Authorisation Application (MAA) was accepted.

The review process for the investigational RNAi therapeutic has been initiated. With today’s acceptance, Alnylam can expect the review timeline to be reduced from the typical 210 days to 150.

Patients with hATTR suffer from the misfolding of transthyretin (TTR) proteins leading to a deposition of amyloid fibrils in different organs. The buildup of TTR fibrils interferes with the intended functionality in a variety of tissues and organs, resulting in further damage, a poor quality of life, and eventually death.

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

“By evaluating the patisiran application under accelerated assessment, the EMA acknowledges the urgent need for a new treatment approach to address the debilitating and devastating effects of hATTR amyloidosis,” said Eric Green, Vice President and General Manager of the TTR program at Alnylam in a press release. “We plan to work closely with the EMA and Committee for Medicinal Products for Human Use (CHMP) toward the goal of bringing patisiran to patients with hATTR amyloidosis in the EU as quickly as possible.”

In addition to the news from the EMA, Alnylam also announced today that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted designation of patisiran as a Promising Innovative Medicine (PIM), supportive of its consideration for entry into the UK’s Early Access to Medicines Scheme (EAMS).

The safety and efficacy of patisiran have not yet been evaluated by any health authority, but it is being developed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made.

In a November interview with Rare Disease Report, 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.”

For the latest updates on patisiran and other drugs making their way through the pipeline, follow Rare Disease Report on Facebook and Twitter.

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