MHRA to allow for early access to Alnylam’s ultra-rare disease treatment, lumasiran
The MHRA has granted Alnylam’s lumasiran a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). The decision will allow patients with the ultra-rare disease primary hyperoxaluria type 1 (PH1) access to the drug before its official approval by the European Commission. PH1 is an ultra-rare orphan disease affecting around 90 patients in