Sentynl Therapeutics gets UK MHRA approval for its drug ‘NULIBRY’ to treat rare genetic disorder

NULIBRY® is the first and only treatment in the United Kingdom (UK) for patients with Molybdenum Cofactor Deficiency (MoCD) Type A. MoCD Type A is an ultra-rare, life- threatening genetic disorder

0
50
Biovoice News DNA Genetics
New Delhi: Sentynl Therapeutics, the wholly owned subsidiary of Zydus Lifesciences has announced that it has received marketing authorization from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for NULIBRY® (fosdenopterin) for Injection as the first therapy for the treatment of patients in Great Britain with Molybdenum Cofactor Deficiency (MoCD) Type A.
MoCD Type A is an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants. It 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 UK MHRA, NULIBRY® is the first and only approved therapy in GB for MoCD Type A.
Speaking on the development, Dr. Sharvil Patel, Managing Director, Zydus Lifesciences Ltd., said, “We are happy that with the UK MHRA approval of NULIBRY® which will enable access to this critical drug and treat an ultra-rare disorder. Healthcare providers in Great Britain can now bridge the unmet needs of patients with this innovative treatment.”
Matt Heck, President & Chief Executive Officer of Sentynl said, “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.”
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.
In March 2022, Sentynl acquired the global rights to NULIBRY® from BridgeBio Pharma, Inc. and is responsible for the ongoing development, manufacturing and commercialization of fosdenopterin globally.