The U.S. Food and Drug Administration has approved Nulibry (cyclic pyranopterin monophosphate) to lower the risk of death in people with molybdenum cofactor deficiency (MoCD) Type A. This is a rare, inherited disorder that usually appears in infancy and causes severe damage to the nervous system. Until now, there was no approved treatment for this condition.
Nulibry is given as a once-daily infusion into a vein. The dose depends on the patient's age and, for newborns, their gestational age. The approval was based on studies that compared patients who received Nulibry to a group of similar patients who did not receive the drug. The results showed that Nulibry helped reduce the risk of death.
This approval is a significant step for families affected by MoCD Type A. However, it is important to know that Nulibry should be started as soon as a diagnosis is suspected. If genetic testing later shows that the patient does not have MoCD Type A, the drug should be stopped.
If you or a loved one has been diagnosed with MoCD Type A, talk to your doctor about whether Nulibry is an option. Your doctor can help you understand the benefits and risks based on your specific situation.