THD NewsDesk, New Delhi: BridgeBio Pharma and Origin Biosciences’ Nulibry (fosdenopterin) is making the first authorized treatment to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. It has been reported that, FDA (The US Food and Drug Administration) has approved Nulibry to treat Molybdenum Cofactor Deficiency Type A.
MoCD Type A is an ultra-rare and progressive condition. This is known to affect less than 150 patients globally with a median survival of four years. Babies with this condition appear normal at birth, but within a week they have difficulty feeding and develop seizures that do not improve with treatment. Basically, the condition presents shortly after birth, often accompanied with severe encephalopathy and intractable seizures
Even, cyclic pyranopterin monophosphate (cPMP), as an experimental treatment for MoCD Type A, is seen to have failed in curing it, however the recent intravenous medication, Nulibry is expected to replace the cPMP. The effectiveness of the therapy was demonstrated in 13 treated patients compared to 18 matched, untreated patients with MoCD Type A. Patients treated with Nulibry had a survival rate of 84% at three years compared to 55% for the untreated patients.
Neil Kumar, founder and chief executive officer of BridgeBio said “Patients with MoCD Type A and their families have an approved therapy for the first time after the FDA approved for Nulibry”,
He further added “We would like to thank who played an essential role in achieving this important milestone, because without the community effort from those of patients, caregivers, physicians, scientists, and advocates, the drug development of this rare disease would not have been possible”
