The approval was based on findings from three clinical studies that demonstrated Nulibry's safety and effectiveness in MoCD Type A treatment compared to data from a natural history study
Sentynl Therapeutics is a fully-owned subsidiary of Zydus Lifesciences. (Credit: Zydus Group)
Sentynl Therapeutics has secured authorisation from the UK Medicines and Healthcare products Regulatory Agency (MHRA) of Nulibry (fosdenopterin) for Injection to treat molybdenum cofactor deficiency (MoCD) Type A.
Nulibry is a substrate replacement therapy that offers a synthetic source of cyclic pyranopterin monophosphate (cPMP). It received the US Food and Drug Administration (FDA) approval in 2021 for the same indication.
In July 2022, the drug was approved by the Israel Ministry of Health to lower the risk of mortality in patients with MoCD Type A. It was approved by the European Medicines Agency (EMA) in September 2022.
Nulibry’s approval in the UK was based on findings from three clinical trials that showed Nulibry’s safety and efficacy in MoCD Type A treatment compared to data from a natural history study.
According to the results, patients receiving Nulibry had a death risk that was 5.5 times lower than that of individuals not receiving the medication.
Furthermore, the survival rate at three years of age for patients treated with substrate replacement therapy was 85.5%, whereas the rate for untreated control patients was 55.1%.
Sentynl Therapeutics president and CEO Matt Heck said: “The MHRA approval of Nulibry opens the door for healthcare providers in Great Britain to utilise this innovative treatment to meet the previously unmet needs of patients and their caregivers.
“This approval advances our mission to make a positive impact in the lives of patients suffering from rare diseases, especially one as devastating as MoCD Type A.”
The US-based Sentynl Therapeutics, which is a fully-owned subsidiary of Zydus Lifesciences, obtained the worldwide rights to the drug from BridgeBio Pharma in March 2022. Sentynl Therapeutics is handling the continued development, manufacturing, and commercialisation of the substrate replacement therapy on a global scale.