The US Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ supplemental new drug application (sNDA) for the expanded use of Oxlumo (lumasiran) to treat advanced primary hyperoxaluria type 1 (PH1).
Oxlumo, which is administered through subcutaneous injection, has now been indicated to treat PH1 to reduce the levels of urinary oxalate (UOx) and plasma oxalate (POx) in paediatric and adult patients.
It is an RNAi therapeutic that targets hydroxyacid oxidase 1 (HAO1), which encodes glycolate oxidase (GO).
The regulatory approval is based on the positive six-month data obtained from the ILLUMINATE-C Phase III study of Oxlumo that was conducted in PH1 patients with severe renal impairment, including those on hemodialysis.
In the study, the treatment with Oxlumo led to substantial reductions in POx and also showed better safety and tolerability profile in compromised renal function patients, including people with kidney failure and who are undergoing treatment by hemodialysis.
The company’s sNDA also included data from open-label extensions of Phase III studies, ILLUMINATE-A and ILLUMINATE-B, conducted in PH1 paediatric and adult patients.
Alnylam Pharmaceuticals Global Rare Disease lead vice-president Jorge Capapey said: “Today’s label expansion exemplifies Alnylam’s commitment to advancing research and innovation in support of the PH1 community.
“We also believe this expansion will strengthen prescribers’ confidence in Oxlumo for patients.
“Through the findings of the ILLUMINATE clinical development program, I am thrilled to see the potential benefit of Oxlumo, which remains the first and only FDA-approved PH1 treatment option, now be available for a broad range of people living with the ultra-rare disease, including those advanced PH1 patients undergoing hemodialysis.”