FDA Approves Treatment for Patients With Urea Cycle Disorder

The U.S. Food and Drug Administration (FDA) approved Olpruva (sodium phenylbutyrate) to treat patients with urea cycle disorders (UCDs).

The drug developed by Acer Therapeutics Inc. and its collaboration partner, Relief Therapeutics, was authorized to treat UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

Olpruva was approved as an oral suspension for adults and children weighing 44 pounds (20 kg) or more and with a body surface area (BSA) of 1.2 m2 or greater.

“The FDA’s approval of OLPRUVA™, an innovative formulation of sodium phenylbutyrate packaged for the first time in single-dose envelopes, marks the culmination of our ongoing dedication to develop new and differentiated treatment options for those affected by rare diseases,” Chris Schelling, chief executive officer and founder of Acer, said in a press release.

A urea cycle disorder is a group of genetic disorders that result in a deficiency of one of the six enzymes responsible for removing ammonia from the body. Ammonia is toxic and can cause life-threatening side effects, such as irreversible brain damage, coma, or death if the body cannot get rid of it.

Urea cycle disorder is a genetic disorder affecting around 1 in 35,000 people in the U.S.

According to the drug manufacturers, the most common side effects of Olpruva are absent or irregular menstrual periods, decreased appetite, body odor, bad taste, or avoiding foods one ate before getting sick.

Olpruva may also cause serious side effects, such as nervous system problems (neurotoxicity), low potassium levels in the blood (hypokalemia), and conditions related to swelling (edema).

Certain medicines may increase the ammonia level in the blood or cause serious side effects when taken during treatment with Olpruva. It is especially important to inform your doctor about taking corticosteroids, valproic acid, haloperidol, and probenecid.

The symptoms of UCDs are usually present a few days after the baby is born. They include:

  • Lethargy or feeling tired
  • Fussiness in babies
  • Being nauseous or vomiting
  • Not being able to eat
  • Breathing too fast or too slow
  • Confusion


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