13 July 2020 - Positive scientific opinion under Early Access to Medicines Scheme will make lumasiran treatment available for UK primary hyperoxaluria type 1 patients before marketing authorisation.

Alnylam Pharmaceuticals announced today that the UK’s Medicines and Healthcare Products Regulatory Agency 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.

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.

Read Alnylam Pharmaceuticals press release