With its third FDA approval in less than two-and-a-half years, Alnylam Pharmaceuticals, Inc. is bringing another orphan drug for a rare condition to the U.S. market. And similar to the first two, Oxlumo (lumasiran) comes with a costly price tag. To help provide payers with some predictability around costs, the company is extending a new value-based agreement, as well as offering another VBA that the company introduced with its previous approval.
On Nov. 23, the FDA approved Alnylam’s Oxlumo for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. The agency says it is the first treatment approved for the rare genetic disorder, in which the body overproduces oxalate. This, in turn, results in the deposition of calcium oxalate in the kidneys and urinary tract, which can lead to kidney stones, nephrocalcinosis, kidney failure and organ damage throughout the body.