Your Monthly Medicines Roundup for October 2024

Metabolic Support UK would like to share that during the month of October, two medications for inherited metabolic disorders were approved in the UK. We also have an update on the Managed Access Agreement for Batten disease type 2 and a suspension update for a treatment for Gangliosidoses.

You can find details below: 

Back in August 2024, we provided an update on Venglustat for treating gangliosidoses in people 2 years and over. This outlined that the appraisal had been discontinued by NICE in England. At the end of September, 2024, NICE provided an update to share that, in line with the August update, they had decided to suspend their assessment, but will continue to monitor any developments.

For further details, click the button below:

Galsulfase for the treatment of people with mucopolysaccharidosis VI (N-acetylgalactosamine 4-sulfatase deficiency; Maroteaux-Lamy syndrome), was recommended as an option for use within NHS Wales. Galsulfase was historically available in Wales, a lapse in availability for people newly diagnosed with MPS VI occurred after this availability was stopped recently. Now that AWMSG has concluded its review, galsulfase will again be available in Wales.

For further details, click the button below:

Since 2019, a Managed Access Agreement (MAA) has been in place for cerliponase alfa to treat people with neuronal ceroid lipofuscinosis type 2 in England. An MAA generally lasts for 5 years after which a decision is made regarding permanent access. NICE have share that the MAA will be extended to 27 May 2025 to allow all involved parties to reach an agreement to secure access people with Neuronal Ceroid Lipofuscinosis-Type 2.

For further details, click the button below:

Miglustat for the treatment of people with Niemann-Pick type C, was recommended as an option for use within NHS Wales. Miglustat was historically available in Wales, a lapse in availability for people newly diagnosed with MPS VI occurred after this availability was stopped recently. Now that AWMSG has concluded its review, miglustat will again be available in Wales.

For further details, click the button below:

For more updates throughout the medicine approval pathway, check out our Medicines Consultations page by clicking the button below:

For further details, click the button below: