Alnylam’s Oxlumo for PH1 Disease Wins FDA Approval

Alnylam Pharmaceuticals has received FDA approval for Oxlumo (lumasiran), a treatment for primary hyperoxaluria type 1 (PH1) that is an ultra-rare orphan disease which can lead to renal failure.

Oxlumo is an RNA interference drug meant to halt production of oxalate, a toxin that leads to the disease. Patients with PH1 produce too much oxalate, which can combine with calcium to cause kidney stones and deposits in the kidneys. Patients can experience progressive kidney damage, which can lead to kidney failure and the need for dialysis.

PH1 affects an estimated one to three individuals per million in North America and Europe. The European Medicines Agency has also issued the drug a marketing authorization.