Your Monthly Medicines Roundup for January 2024

Metabolic Support UK would like to share that during the month of January, one medication for inherited metabolic disorders was approved in Scotland. We also discuss the different treatments we are currently expecting to hear progress about this year.

You can find details below: 

The Scottish Medicines Consortium have released the outcome of the ultra-orphan initial assessment of fosdenopterin for the treatment of people with molybdenum cofactor deficiency (MoCD) type A. It is approved for an initial time period of approximately three years to allow for additional evidence and data to be collected. The Scottish Government will confirm exactly when fosdenopterin will become available on the NHS in Scotland as part of the ultra-orphan pathway. 

For further details, click the button below:

• Arginase 1 deficiency– pegzilarginase for the treatment of people with ARG1d is currently being assessed by NICE in England. We are hoping a recommendation will be issued by the end of the year. 

• Batten disease– the Managed Access Agreement (MAA) for cerliponase alfa for the treatment of Batten disease is expected to come to an end this May, 2025. Based on the data gathered during the MAA period, a new assessment of cerliponase alfa for the treatment of Batten disease will be undertaken by NICE in England. 

• Leber hereditary optic neuropathy–Idebenone for the treatment of LHON by NICE in England is expected to receive a recommendation in May 2025. 

• Long-chain fatty acid oxidation disorders (CACT, CPTI, CPTII, LCHAD, TFP, VLCAD): the assessment of triheptanoin, also known as Dojolvi or C7, for the treatment of LC-FAODs by NICE in England is expected to start this summer. 

• Molybdenum cofactor deficiency type A: fosdenopterin, also known as cPMP, for the treatment of MoCD type A is currently being assessed by NICE in England. We are hoping that a recommendation will be issued soon. 

• Niemann-Pick Type AB and B: Olipudase alfa for the treatment of Niemann-Pick type A and AB is currently being assessed by NICE in England. We are hoping a recommendation will be issued by the end of the year.