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The Medicines and Healthcare Products Regulatory Agency (MHRA) has granted Alnylam's lumasiran a positive scientific opinion through the Early Access to Medicines Scheme (EAMS).
The decision allows 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 the UK, that causes an abnormal build-up of toxic oxalate in the liver, resulting in painful and recurrent kidney stones and ultimately irreparable damage to the kidneys and other vital organs.
Richmond Pharmacology is proud to have been involved in the early stage of the drug and supported the development through a first-in-human combined healthy volunteer and patient trial.
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