Sentynl Therapeutics’ Nulibry Injection Gets UK MHRA Approval for MoCD Type A

Sentynl Therapeutics’ Nulibry Injection Gets UK MHRA Approval for MoCD Type A

By, Admin 19 April 2024

UK MHRA approves Sentynl Therapeutics’ Nulibry for injection as the first therapy for treatment of MoCD type A

Overview 

Sentynl Therapeutics, Inc, a US-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (Zydus Group), has 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 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.

About Nulibry

  • Nulibry is a first-in-class synthetic cPMP substrate replacement therapy that was approved by the US 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. 
  • The MHRA decision is based on efficacy and safety data collected to date.

First of Its Kind Treatment

Nulibry is the first and only treatment in Great Britain for patients with molybdenum cofactor deficiency (MoCD) type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants with a median overall survival age of about four years.

Sentynl CEO on Nulibry

  • "The MHRA approval of Nulibry opens the door for healthcare providers in Great Britain to utilize this innovative treatment to meet the previously unmet needs of patients and their caregivers,"said Matt Heck, president & chief executive officer of Sentynl. 
  • "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."
  • Behind approval
  • 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 per cent for Nulibry-treated patients and 55.1 per cent for untreated control patients.

Global Rights Acquisition

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.

About MoCD 

  • Molybdenum Cofactor Deficiency (MoCD) type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity. 
  • The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.
  • MoCD type A is an ultra-rare disease. 
  • The incidence and prevalence of MoCD type A in Great Britain are not known, but the estimated prevalence is 0.005 per 10,000. 
  • Based on these estimates, MoCD type A is likely to be underdiagnosed.

MoCD type A: Symptoms & Severity

  • The most common presenting symptoms of MoCD type A are seizures, feeding difficulties and encephalopathy. 
  • Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging. 
  • This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.

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