The US Food and Drug Administration (FDA) has approved BridgeBio Pharma and Origin Biosciences’ Nulibry (fosdenopterin), making it the first treatment to be authorised to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.
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.
Patients treated with Nulibry had a survival rate of 84% at three years compared to 55% for the untreated patients.
“The FDA’s approval of Nulibry means that patients with MoCD Type A and their families have an approved therapy for the first time. It also reflects our belief that every life matters and that no disease is too rare to address,” said Neil Kumar, founder and chief executive officer of BridgeBio.
“As is often true in rare disease drug development, this was a community effort in which we were able to play a part – we’d like to thank the patients, caregivers, physicians, scientists, and advocates who played an essential role in achieving this important milestone,” he added.