Monday, March 1, 2021

FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A - Drug Information Update

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FDA Center for Drug Evaluation and Research Division of Drug Information

FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A

The U.S. Food and Drug Administration approved Nulibry (fosdenopterin) for injection to reduce the risk of death due to Molybdenum Cofactor Deficiency Type A, a rare, genetic,  metabolic disorder that typically presents in the first few days of life, causing intractable seizures, brain injury and death.

The most common side effects included complications related to the intravenous line, fever, respiratory infections, vomiting, gastroenteritis and diarrhea.

Phototoxicity (injury to the skin and eyes from certain types of light, such as sunlight) was seen in animals, so patients treated with Nulibry should avoid exposure to sunlight and wear sunscreen, protective clothing, and sunglasses when exposed to the sun.

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