image credit: The U.S. FDA / Wikimedia

FDA approves Nulibry to treat Molybdenum Cofactor Deficiency Type A

March 1, 2021


MoCD Type A is an ultra-rare and progressive condition which is known to affect less than 150 patients globally with a median survival of four years.

The condition presents shortly after birth, often accompanied with severe encephalopathy and intractable seizures.

People with MoCD Type A cannot produce cyclic pyranopterin monophosphate (cPMP) – Nulibry is an intravenous medication that replaces the missing cPMP.

The effectiveness of the therapy was demonstrated in 13 treated patients compared to 18 matched, untreated patients with MoCD Type A.

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