Alnylam's Oxlumo Indicated For Treatment Of PH1 To Lower Urinary And Plasma Oxalate Levels

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) said that the U.S. Food and Drug Administration approved a label expansion for Oxlumo (lumasiran), an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 or PH1 to lower urinary oxalate or UOx and plasma oxalate or POx levels in pediatric and adult patients.

The approval was based on positive efficacy and safety results of the ILLUMINATE-C Phase 3 study of Oxlumo in patients with severe renal impairment, including those on hemodialysis.

Oxlumo was approved in in November of 2020 by the FDA for the treatment of PH1 to lower UOx levels in pediatric and adult patients. It was also approved by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups.

Primary hyperoxaluria type 1 is an ultra-rare genetic disease characterized by oxalate overproduction in the liver. 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 and nephrocalcinosis, which can progress to kidney failure. PH1 can also lead to oxalate deposition in multiple organs beyond the kidney, a condition known as systemic oxalosis.