Alnylam Pharmaceuticals said Thursday that the European Commission approved the RNAi therapeutic Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1) in all age groups in the EU. The treatment works by degrading HAO1 mRNA and depleting the glycolate oxidase enzyme, which in turn inhibits the hepatic overproduction of oxalate that drives progression in PH1 disease.
CEO John Maraganore said "Alnylam has taken [Oxlumo] from identification of compound to regulatory approval in just six years and we will progress with the same sense of urgency as we work with national reimbursement bodies across Europe." The regulatory approval follows a positive opinion issued last month from the European Medicines Agency's Committee for Medicinal Products for Human Use based on data from two Phase III trials.
In ILLUMINATE-A, which included adults and children six years or older, Oxlumo achieved the primary endpoint with a 53.5% mean reduction in urinary oxalate versus placebo and also showed a 65.4% mean reduction in urinary oxalate relative to baseline. The majority of Oxlumo-treated patients also achieved normal or near-normal levels of urinary oxalate, compared to none in the placebo group. Meanwhile, efficacy results and the safety profile of Oxlumo in the ILLUMINATE-B study, which enrolled infants and children under the age of six, were comparable to those seen in ILLUMINATE-A.
The treatment is administered via subcutaneous injection by a healthcare professional once monthly for three months, then once quarterly thereafter. Oxlumo is also under priority review in the US, with the FDA expected to make a decision by December 3.
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