Alnylam Announces that the United Kingdom’s MHRA Grants Early Access to Lumasiran

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). With this decision, eligible PH1 patients in the UK, many of whom are children, can gain access to lumasiran before the drug is granted marketing authorization by the European Commission (EC).

The aim of EAMS is to provide early availability of innovative, 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.

PH1 is an ultra-rare orphan disease characterized by excessive oxalate production, which can lead to end-stage kidney disease (ESKD) and other systemic complications. PH1 affects 1-3 individuals per million in Europe and the United States; in the United Kingdom, it is estimated that there are 100 patients diagnosed with PH1. Current treatment approaches do not prevent oxalate overproduction and aim to lessen damage to the kidneys and delay progression to ESKD. PH1 patients with advanced kidney disease require dialysis to help filter waste products from their blood, until they are able and eligible to receive a dual or sequential liver/kidney transplant. However, long-term dialysis and post-transplant complications can severely impact patients’ quality of life.

“This positive scientific opinion to make lumasiran available through the EAMS is wonderful news for PH1 patients and their families, who currently have limited treatment options,” said Brendan Martin, Country Manager, UK & Ireland at Alnylam. “New medicines that address the underlying cause of this ultra-rare condition, and have the potential for a favorable impact on disease manifestations, are urgently needed. This decision will allow eligible PH1 patients in the UK to have access to lumasiran at the earliest opportunity.”