Primary Hyperoxaluria Drug’s Label Receives Expansion to Include Patients with More Severe Disease

According to a press release published on, the US Food and Drug Administration (FDA) has expanded the label for Oxlumo, a drug developed by Alnylam Pharmaceuticals as a treatment for the rare disease primary hyperoxaluria type 1 (PH1). The drug is available for pediatric and adult patients living with the disease and is intended to lower plasma oxalate and urinary oxalate levels. The expanded label allows for use in the patients with low kidney function that are undergoing hemodialysis.

About Primary Hyperoxaluria

Primary hyperoxaluria is a rare progressive condition in which the body produces an excess of oxalate. This primarily leads to the development of deposits of oxalate that appear in the kidney and bladder, resulting in frequent kidney stones. There are three different types of primary hyperoxaluria which are distinguished by distinct causal mutations that affect different genes. The symptoms that result from these disorders are the result of oxalate deposits appearing in different areas of the body. Oxalate may appear in the bone marrow, joints, and bones. Frequent kidney stones result in symptoms such as acute pain, secondary infection of urine, and ultimately damage to the kidneys. As these stones appear early in life, kidney failure is of serious concern to primary hyperoxaluria patients. There aren’t any medications currently approved for this condition. Treatment approaches may include dialysis (as the kidneys fail) and kidney/liver transplant. To learn more about primary hyperoxaluria, click here.

About Oxlumo and the Expanded Label

The FDA first approved Oxlumo in November 2020. The updated, expanded label approval follows positive results in a phase 3 clinical trial in which the drug substantially lowered plasma oxalate and displayed a good tolerability and safety profile in patients whose disease has progressed to kidney failure and were undergoing hemodialysis. With this new label, a greater number of people living with the disease will be able to continue to receive treatment with Oxlumo. 

“The significance of the label expansion of Oxlumo cannot be overstated, as this milestone provides crucial reassurance among a patient population with the highest unmet need, as well as their caregivers and loved ones, that Oxlumo is available in the U.S. for patients living with PH1, including those with advanced disease.” – Kim Hollander, Executive Director, Oxalosis and Hyperoxaluria Foundation


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