Your Monthly Medicines Roundup for May 2025
Your Monthly Medicines Roundup for May 2025
It has been a quiet month in terms of public announcements of new access developments for metabolic medicines. There is one update in relation to batten disease type 2, which should come as a positive and welcome announcement while negotiations are ongoing. Next month, we expect to be able to share an update for arginase-1 deficiency and molybdenum co-factor deficiency type A.
You can find details below:
Neuronal Ceroid Lipofuscinosis-Type 2 (batten disease type 2)
Last month, we let you know that a the Managed Access Agreement had been extended for a duration of three months. NICE in England have now disclosed this has been extended to December 2025, unless they reach a final recommendation earlier. Irrespective of the final outcomes, BioMarin and the NHS have announced that people living with batten disease type 2 who are currently accessing Cerliponase alfa through the managed access agreement will have access to the treatment for life
For further details about the assessment, click the button below:
