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Alnylam Announces Innovative Value-Based Agreement Framework for OXLUMO™ (lumasiran) to Accelerate Access for Patients with Primary Hyperoxaluria Type 1 and Deliver Ultra-Rare Orphan Disease Pricing Solutions to U.S. Payers
- Expedited Access to OXLUMO Aims to Support Children and Adults Living with PH1 who Face Inevitable Disease Progression and Irreversible Kidney Damage in
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Expedited Access to OXLUMO Aims to Support Children and Adults Living with PH1 who Face Inevitable Disease Progression and Irreversible Kidney Damage in the Absence of New Treatment Options -\n\n- New Value-Based Agreement Framework Includes an Innovative Patient Need Adjustment that Offers Payers Increased Cost Predictability Across the Entire Spectrum of PH1 Patient Ages, from Infant to Adult - \n\n- Express Scripts, Harvard Pilgrim, and Highmark are Among Leading Payers Pursuing Agreements in Principle -\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today a new framework for value-based agreements (VBAs) designed to help people with primary hyperoxaluria type 1 (PH1) gain access to OXLUMO™ (lumasiran). Now approved by the U.S. Food and Drug Administration (FDA) for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients, OXLUMO is the first-ever approved targeted therapeutic that substantially curbs oxalate production in patients living with PH1, an ultra-rare genetic disease characterized by oxalate overproduction.\n\nAlnylam is in active discussions with leading payers and has reached an agreement in principle with Express Scripts, Harvard Pilgrim, and Highmark to pursue VBAs for OXLUMO.\n\nOxalate overproduction in PH1 results in elevated urinary oxalate and the deposition of calcium oxalate crystals in the kidneys and urinary tract. People with PH1 typically endure intensive management of debilitating, painful and recurrent kidney stones. With limited treatment options previously available, the disease would inevitably progress to kidney failure, nephrocalcinosis (calcification of the kidneys), and multi-organ dysfunction as a consequence of systemic oxalosis (the spread of oxalate to organs and tissues outside of the kidneys).1 People with PH1 often present with kidney failure at the time of diagnosis.2 PH1 patients with renal failure undergo dialysis almost daily, for up to 10-12 hours per day and night.2 A dual or sequential liver/kidney transplant is then typically performed to resolve the underlying metabolic defect in the liver and restore kidney function,2 but these interventions are associated with life-long immunosuppression and a high risk of morbidity and mortality. Until now, there have be...