Alnylam Pharmaceuticals, a RNAi therapeutics company, has announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted patisiran, an investigational RNAi therapeutic for the treatment of hATTR amyloidosis, a positive scientific opinion through the Early Access to Medicines Scheme (EAMS).
With this decision, eligible adults in the UK with hATTR amyloidosis can gain access to patisiran before the drug is granted marketing authorisation by the European Commission (EC).
The aim of EAMS is to provide early availability of innovative, new, unlicensed medicines to UK patients who have a high degree of unmet clinical need.
Patisiran is an investigational, intravenously administered RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to reduce the deposition and facilitate the clearance of TTR amyloid in peripheral tissues and potentially restore function to these tissues.
Hereditary TTR-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue. hATTR amyloidosis represents a major unmet medical need with significant morbidity and mortality, affecting approximately 50,000 people worldwide.
The MHRA’s decision is based on the evaluation of the effects of patisiran in hATTR amyloidosis patients with polyneuropathy and its safety profile as demonstrated in the APOLLO Phase 3 study. Within EAMS, patisiran will be made available for eligible hATTR amyloidosis patients presenting with symptoms of polyneuropathy and/or cardiomyopathy.
MHRA’s decision follows the recent positive opinion by the CHMP for patisiran for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. If approved by the EC, the medicine will be commercialised under the brand name ONPATTRO™. Patisiran is currently under priority review as a Breakthrough Therapy with the U.S. Food and Drug Administration(FDA).
Brendan Martin, Country Manager, UK & Ireland at Alnylam said, “The EAMS positive scientific opinion reflects Alnylam’s commitment to patients with hATTR amyloidosis and their families, for whom it will be welcome news. New treatment options that impact the underlying cause of the disease, improve neuropathy and a patient’s ability to function on a daily basis, are urgently needed and this decision will allow patients to have access to patisiran without delay.”