Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced on 8/3/18 that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted patisiran, an investigational RNAi therapeutic in development 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 authorization 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. The medicines included in the scheme are those that are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where there are no adequate treatment options.
“The EAMS positive scientific opinion reflects Alnylam’s commitment to patients with hATTR amyloidosis and their families, for whom it will be welcome news,” said Brendan Martin, Country Manager, UK & Ireland at Alnylam. “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.”

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. The results of the APOLLO study were published July 5, 2018 in The New England Journal of Medicine (NEJM). Within EAMS, patisiran will be made available for eligible hATTR amyloidosis patients presenting with symptoms of polyneuropathy and/or cardiomyopathy.

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.

hATTR amyloidosis represents a major unmet medical need with significant morbidity and mortality, affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy. Treatment options that can modify the course of the disease are limited and there remains a pressing need for novel medicines to help treat patients with hATTR amyloidosis.