FDA approval of Oxlumo, the first and only treatment approved for primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients. - Alnylam Pharma.

Alnylam Pharmaceuticals, Inc.has announced that the FDA approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and systemic organ dysfunction. In ILLUMINATE-A – the largest controlled Phase III study ever conducted in PH1 – Oxlumo was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients achieving normal or near-normal levels. Oxlumo demonstrated an encouraging safety and tolerability profile, with injection site reactions (ISRs) as the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase III study, the safety and efficacy of Oxlumo were demonstrated in patients under the age of six, and results showed reduction of urinary oxalate and an overall safety and tolerability profile consistent with that demonstrated in ILLUMINATE-A.