Alnylam Announces FDA Approval of Supplemental New Drug Application for OXLUMO (lumasiran) in Advanced Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) approved a label expansion 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. 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.

PH1 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.

The FDA approval is based on positive six-month results from the ILLUMINATE-C Phase 3 study, in which OXLUMO treatment resulted in substantial reductions in POx and demonstrated an encouraging safety and tolerability profile in patients with compromised renal function, including those with kidney failure and undergoing treatment by hemodialysis. Elevated POx is directly related to the pathophysiology of oxalosis and results in systemic deposition of oxalate in extra-renal tissues, potentially leading to bone fractures, cardiomyopathy, impaired erythropoiesis, vision loss, skin ulcers and other serious manifestations.¹

The supplemental New Drug 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.

“Today’s label expansion exemplifies Alnylam’s commitment to advancing research and innovation in support of the PH1 community. We also believe this expansion will strengthen prescribers’ confidence in OXLUMO for patients,” said Jorge Capapey, Vice President, Global Rare Disease Lead at Alnylam Pharmaceuticals. “Through the findings of the ILLUMINATE clinical development program, I am thrilled to see the potential benefit of OXLUMO, which remains the first and only FDA-approved PH1 treatment option, now be available for a broad range of people living with the ultra-rare disease, including those advanced PH1 patients undergoing hemodialysis. We extend our sincere gratitude to the patients, families and investigators involved in the research supporting this approval, without whom it would not be possible to bring this therapy to those living with PH1.”