MHRA approves Nulibry (fosdenopterin) to treat molybdenum cofactor deficiency (MoCD) type A
Sentynl Therapeutics, Inc./Zydus Lifesciences, Ltd. announced The Medicines and Healthcare products Regulatory Agency (MHRA) authorization of Nulibry (fosdenopterin) for Injection as the first therapy for the treatment of patients in Great Britain (GB) with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants
MoCD Type A is known to impact fewer than 150 patients globally with a median survival age of four years.
Nulibry is a first-in-class synthetic cPMP substrate replacement therapy that was approved by the U.S. Food and Drug Administration (FDA) in 2021 to reduce the risk of mortality in patients with MoCD Type A. Following this decision by the MHRA, Nulibry is the first and only approved therapy in GB for MoCD Type A.
Nulibry's MHRA approval was supported by data from three clinical trials that demonstrated the safety and efficacy of Nulibry for the treatment of patients with MoCD Type A compared to data from a natural history study. These studies showed that Nulibry-treated patients had a 5.5 times lower risk of death than that of the untreated patients. Moreover, the survival probability at 3 years of age was 85.5% for Nulibry-treated patients and 55.1% for untreated control patients.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
3 mins