Your Monthly Medicines Roundup for March 2025
Your Monthly Medicines Roundup for March 2025
Metabolic Support UK would like to share that during the month of March, two medications for inherited metabolic disorders have seen updates.
You can find details below:
Neuronal Ceroid Lipofuscinosis-Type 2 (batten disease type 2)
In January of this year, we let you know that we were expecting an update shortly as the Managed Access Agreement (MAA) for cerliponase alfa for the treatment of Batten disease, active in England (NICE) is expected to come to an end in May. A Committee Meeting to discuss the pharmaceutical company’s additional information, will be held on the 3rd of April.
For further details about the assessment, click the button below:
Leber hereditary optic neuropathy
Idebenone for the treatment of LHON by NICE in England was discussed during a committee meeting in February. The draft guidance was published on 5 March: Idebenone for the treatment of LHON was not recommended. Between 5 and 26 March, NICE welcomed feedback which is currently being deliberated. It is currently unclear when the final decision will be published. For further support, we recommend reaching out to the LHON Society.
For further details about the assessment, click the button below:
