Your Monthly Medicines Roundup for February 2025

In this month’s update, Metabolic Support UK share that during the month of February, one medication for inherited metabolic disorders received a negative recommendation in England. We also discuss the three treatments which have been identified for inherited metabolic disorders and are now awaiting development.

You can find details below: 

The National Institute for Health and Care Excellence (NICE) have released the final recommendation for Olipudase alfa in the treatment of people living with Niemann-Pick type AB and B in England. Unfortunately, NICE do not recommend Olipudase alfa. They reached this conclusion based on the evidence presented by the Company.

NICE stated that the evidence shows that, after one year of treatment, lung function is improved and the size of the spleen is reduced. These improvements become more gradual as the condition stabilises. This means that the treatment shows a positive effect in managing the condition. Unfortunately, the cost associated with the treatment are considered too high for NICE to recommend this as acceptable use of NHS resources. For further support, we recommend reaching out to Niemann-Pick UK .

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• Familial Chylomicronaemia Syndrome– Olezarsen for treating people with familial chylomicronaemia syndrome was selected for assessment by NICE In England. Timelines for the next steps of this assessment are currently unclear. We will provide updates as we receive them. Further details can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11678 

• Familial hypercholesterolaemia– Inclisiran for treating adolescents with familial hypercholesterolaemia and elevated low density lipoprotein cholesterol in people 12 to 17 years was selected for assessment by NICE In England. Timelines for the next steps of this assessment are currently unclear. We will provide updates as we receive them. Further details can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11698
  

• Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo Syndrome type A– UX111 for treating people with mucopolysaccharidosis type IIIAwas selected for assessment by NICE In England. Timelines for the next steps of this assessment are currently unclear. We will provide updates as we receive them. Further details can be found here: https://www.nice.org.uk/guidance/awaiting-development/gid-ta11701