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Alnylam submits regulatory applications to the FDA and EMA to support label expansion for Oxlumo for the treatment of advanced primary hyperoxaluria type 1.

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Published:15th Dec 2021
Alnylam Pharmaceuticals, Inc. announced the submission of a supplemental New Drug Application (sNDA) to the FDA and Type II Filing Variation to the European Medicines Agency (EMA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the reduction of plasma oxalate in the treatment of patients with advanced primary hyperoxaluria type 1 (PH1).

“Compromised renal function in patients with advanced PH1 often leads to elevated levels of plasma oxalate and subsequent pathologic deposition of oxalate in multiple organs, including the bones, eyes, heart, and skin – a highly morbid and potentially fatal condition known as systemic oxalosis.

Treatment options for these patients are limited to intensive hemodialysis and liver/kidney transplantation. With that in mind, we believe the ILLUMINATE-C data package demonstrates the potential for lumasiran to provide a therapeutic option that substantially reduces plasma oxalate levels that result in systemic oxalosis in adult and pediatric PH1 patients with advanced disease, including for patients on hemodialysis,” said Jeroen Valkenburg, General Manager, Lumasiran program at Alnylam.

The applications to the FDA and EMA are based on positive results from the ILLUMINATE-C single-arm, open-label Phase III study of lumasiran in patients of all ages with advanced PH1. The six-month primary analysis results from the study were presented in November 2021 at the American Society of Nephrology (ASN) Kidney Week annual meeting and showed a substantial reduction in plasma oxalate from baseline in PH1 patients with chronic kidney disease (CKD) stage 3b-5, with or without dialysis. The most common drug-related adverse events were injection site reactions, all of which were mild and transient.

Condition: Hyperoxaluria
Type: drug

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