Alnylam Pharmaceuticals and its partner Sanofi Genzyme have submitted an application that asks European regulators to approve patisiran for adults with hereditary transthyretin-mediated (hATTR) amyloidosis.
The European Medicines Agency (EMA) announced earlier that it will review the so-called Marketing Authorisation Application (MAA) under an accelerated assessment pathway, which will reduce the evaluation time from 210 to 150 days.
An accelerated assessment may be granted to therapies that are considered of major interest for public health and therapeutic innovation.
The accelerated assessment status is granted to therapies considered to be therapeutic innovations that will cover a public health need in order to provide access to therapies more quickly.
Patisiran is an RNAi treatment that prevents faulty transthyretin (TTR) protein from being produced. Buildup of faulty TTR protein causes hATTR amyloidosis, also called familial amyloid polyneuropathy (FAP).
“The MAA submission for patisiran represents another important milestone for Alnylam and a critical step toward bringing RNAi therapeutics to people living with hATTR amyloidosis,” Eric Green, vice president and general manager of the TTR program at Alnylam, said in a press release.
An earlier Phase 3 clinical trial, called APOLLO (NCT01960348), showed that patisiran robustly decreased TTR protein, as well as symptoms and impairment caused by amyloid accumulation in various organs. Moreover, the study showed that patients’ quality of life improved with the treatment.
“Based on the results of the APOLLO study, we believe patisiran has the potential to become the standard of care for the treatment of hATTR amyloidosis. We look forward to working with the EMA and the Committee for Medicinal Products for Human Use (CHMP) during the review process,” said Green.
“People with hATTR amyloidosis have limited treatment options,” added Rand Sutherland, MD, Therapeutic Area Head, Rare Diseases Development at Sanofi.
“With this MAA submission, we are one step closer to making patisiran available in Europe and executing on our shared vision to bring this RNAi treatment to patients globally,” Sutherland said.
Meanwhile, Alnylam recently completed its application with the U.S. Food and Drug Administration to approve the treatment. The application had been submitted on a rolling basis, a practice that speeds up the application process.
Sanofi also is preparing regulatory filings for the treatment in Japan, Brazil and elsewhere. The company plans to start submitting the applications in the first half of 2018.