UK's MHRA allows early access to lumasiran for ultra-rare disease

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.

There are currently no treatment options for the condition. Existing strategies focus on disease management and patients with advanced disease require kidney dialysis until they are able, and eligible, to receive a dual kidney/liver transplant.

Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1), which encodes glycolate oxidase (GO). By silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1.

Clinical data, from the ILLUMINATE-A trial, show that the drug achieved a 65.4% mean reduction in urinary oxalate relative to baseline, with a mean treatment difference of 53.5% relative to placebo.

“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.”

The drug is currently being review by regulators in Europe and the US.