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

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  • 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.
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