May 2025 decisions news release
The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday, May 12) published advice on six medicines.
An ultra-orphan initial assessment report has been published for exagamglogene autotemcel (Casgevy®) a treatment for transfusion-dependent beta-thalassemia, a rare genetic condition that affects the production of haemoglobin. It is for patients aged 12 and over, where a stem cell transplant is appropriate but there is no suitable donor available. The Scottish Government will confirm when it is available for prescribing and further evidence will be gathered. Following this SMC will review it and decide on routine availability in NHSScotland.
Sodium thiosulfate (Pedmarqsi®) was accepted to reduce the risk of hearing loss, caused by cisplatin-based chemotherapy, when it is used to treat solid tumours that have not spread in children aged one month to 18 years.
Erdafitinib (Balversa®) was accepted to treat adults with urothelial carcinoma, a type of bladder cancer, that has spread to other parts of the body or can’t be removed by surgery, in patients who have received previous treatment.
Mepolizumab (Nucala®) was accepted, for use together with other medicines, to treat a type of asthma called eosinophilic asthma, in adults and children aged six and over. It is used where the asthma is severe and hasn’t been well controlled with previous treatments.
Donanemab (Kisunla®) was not recommended to treat the early stages of Alzheimer's disease in adults who carry one or no copies of a gene called ApoE4.
Fruquintinib (Fruzaqla®) was not recommended as a treatment for adults with advanced colorectal cancer who have already received at least two lines of treatment.
SMC Chair Dr Scott Muir said: “The committee is pleased to be able to accept these new medicines for use by NHSScotland.
“Sodium thiosulfate offers the first licensed treatment option for reducing the risk of hearing loss in children undergoing cisplatin-based chemotherapy.
“Erdafitinib offers a targeted treatment option that can be taken at home for people with bladder cancer who have already received one line of treatment.
“SMC welcomed the submission for donanemab and recognises the need for new therapies for Alzheimer’s disease. However, having carefully considered all the evidence, the committee was unable to accept donanemab for treatment in the NHS in Scotland.
“The committee considered that there remains uncertainty around what the modest clinical benefit means for patients and their families. We would welcome a resubmission from the company with changes to address the uncertainties.
“The committee was unable to accept fruquintinib for the treatment of adults with advanced colorectal cancer. The company’s evidence around the cost effectiveness of the treatment compared to currently available options was not sufficient.”