MHRA grants early access to lumasiran in UK

13 July 2020

US RNAi therapy specialist Alnylam Pharmaceuticals (Nasdaq: ALNY) says 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 in Europe. A marketing authorization application (MAA) for the drug was submitted to the European Medicines Agency (EMA) in April, at the same time as it was filed for US approval.

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.

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