Your Monthly Medicines Roundup for February 2026

February was an eventful month, with a number of new medicines for inherited metabolic disorders identified for appraisal, as well as a few final decisions. Sadly not all of these decisions were positive and if you do find yourself needing a listening ear, you can reach out to our Support team via 08006523181 or email us at contact@metabolicsupport.org.  

OTL-101 for treating people with adenosine deaminase deficiency–severe combined immunodeficiency was discontinued by NICE in England in February 2026. NICE shared that the company had not provided any information to enable an assessment. 

Further details can be found here:  

Final draft guidance recommending pegzilarginase for the treatment of people with arginase-1 deficiency by NICE in England was published on the 17th of February2026. Metabolic Support worked on this assessment, together with the ARG1d community and healthcare professionals since 2024 and are very pleased this has come to a positive recommendation.

Further details about the assessment can by clicking the first button. Our full news article about this development can be accessed via the second button.

Since 2019, a Managed Access Agreement has been in place for people with Batten disease to receive cerliponase alfa. From 2023, the results from the managed access agreement have been under assessment by NICE in England to decide whether cerliponase alfa can become routinely available on the NHS. By mid-2025, NICE had reached the conclusion that they were unable to recommend the treatment. This was appealed by the company and patient organisation, BDFA, and a hearing was held late 2026. The final recommendation from NICE was announced in February and unfortunately details that cerliponase alfa will not be made routinely available on the NHS. 

Full details of the final recommendation can be found here: 

Atidarsagene autotemcel for treating people with metachromatic leukodystrophy has been identified by SMC in Scotland for assessment. Evidence submission to SMC is expected for the second half of the year, with the full assessment starting shortly after.

We will keep you updated as this develops. In the meantime, full details of this upcoming assessment can be found here:  

Similar to OTL-101 for treating people with adenosine deaminase deficiency–severe combined immunodeficiency, VTS-270 for treating people with Niemann-Pick type C1 was discontinued by NICE in England in February 2026. NICE shared that the company had not provided any information to enable an assessment.

Further details can be found here: 

Sepiapterin for treating hyperphenylalaninaemia in phenylketonuria in people of any age has been identified by NICE in England for assessment. Timelines for this submission are still unclear. We will keep you updated as this develops.

In the meantime, full details of this upcoming assessment can be found here: