FDA approves first drug to treat rare kidney-related condition

The Food and Drug Administration on Monday approved Oxlumo to treat a rare genetic disorder affecting the kidneys, among other organs.

“The approval of Oxlumo represents a great triumph of community involvement to address a rare disease,” Dr. Norman Stockbridge, director of the division of cardiology and nephrology in the FDA’s Center for Drug Evaluation and Research, said in a news release. 

The condition is called “primary hyperoxaluria type 1” (PH1) and results from an overproduction of the chemical oxalate. The FDA said the approval of Oxlumo marks the first treatment for PH1. 

The first sign of the disease is often a kidney stone, per the Mayo Clinic. (iStock)


“Hyperoxaluria can be caused by inherited (genetic) disorders, an intestinal disease, or eating too many oxalate-rich foods. The long-term health of your kidneys depends on early diagnosis and prompt treatment of hyperoxaluria,” according to the Mayo Clinic.

The first sign of the disease is often a kidney stone, which results from a combination of calcium and the overproduction of oxalate, per the Mayo Clinic. The FDA added that the damage from oxalate can progress beyond the kidneys to the heart, bones and eyes.

The drug approved Monday works to tamp down on oxalate production. Small clinical studies showed a monthly injection of Oxlumo followed by maintenance doses dropped oxalate levels in the urine by 65%, compared to a 12% decrease in the placebo group. 

Further, after six months, over half of 26 trial participants reached normal levels of the chemical in the urine, whereas none of the 13 placebo patients saw the same result. A second study among 16 kids showed a larger drop in oxalate, at 71% after six months.

The FDA said the most frequent side effects of Oxlumo are stomach pain and reaction at the injection site.


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