Alnylam's Lumasiran Scores Expanded FDA Approval For Ultra Rare Genetic Disease

The FDA approved a label expansion for Alnylam Pharmaceuticals Inc's ALNY for Oxlumo (lumasiran), an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate (UOx) and plasma oxalate (POx) levels in pediatric and adult patients.
PH1 is an ultra-rare genetic disease characterized by oxalate overproduction in the liver.
The approval is 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.
Related: Analysts Put Alnylam On Upside Catalyst Watch Ahead Of Upcoming Data From Lead Program.
Oxlumo treatment resulted in substantial reductions in POx and demonstrated encouraging safety and tolerability profile in patients with compromised renal function.
The supplemental application also included results from the open-label extensions of the ILLUMINATE-A and ILLUMINATE-B Phase 3 studies of pediatric and adult patients with PH1.
The label has correspondingly been updated to highlight the maintenance of sustained reductions in UOx through Month 24 and Month 12, respectively.
Price Action: ALNY shares traded lower by 0.28% at $201.81 on the last check Friday.