The Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for the use of fosdenopterin (Nulibry) in the treatment of Molybdenum Cofactor Deficiency (MoCD) Type A, a rare genetic disorder.
What is MoCD Type A? MoCD Type A is a genetic disorder characterised by the body’s inability to produce cyclic pyranopterin monophosphate (cPMP). This deficiency leads to the accumulation of toxic sulphites, which can cause brain damage. Symptoms typically manifest in newborns and include seizures, involuntary movements, and feeding difficulties.
Fosdenopterin (Nulibry) Authorisation:
- The MHRA has authorised the use of fosdenopterin (Nulibry) in Great Britain.
- The decision was based on an European Commission (EC) decision (EMEA/H/C/005378), following advice from the Committee for Medicinal Products for Human Use (CHMP). This process is known as the EC Decision Reliance Procedure.
Safety and Administration:
- Patient safety and access to high-quality medical products are top priorities for the MHRA.
- The regulatory standards for fosdenopterin’s approval have been met.
- Close monitoring of safety will continue.
- Fosdenopterin is administered intravenously via a catheter as a 9.5 mg powder for solution for injection.
Understanding MoCD Type A:
- MoCD Type A results from an error in the MOCS1 gene, which impairs cPMP production.
- The absence of cPMP prevents the breakdown of certain compounds (sulphites) in the body.
- These sulphites are toxic to the brain and can impact a child’s development.
The approval of fosdenopterin represents a significant advancement in the treatment of MoCD Type A, offering hope to patients and their families. As with all medications, ongoing safety vigilance remains essential.
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