We are delighted to announce that velmanase alfa, the ERT treatment for alpha mannosidosis has been made accessible under the Scottish Medicines Consortium’s (SMC) ultra-orphan pathway. This means that eligible patients in Scotland, can have access to the treatment under this process for the next 3 years. This is great news for patients diagnosed with alpha mannosidosis in Scotland.
Velmanase alfa meets the Scottish Medicines Consortium (SMC) definition of an ultra-orphan medicine, which is a medicine to treat an extremely rare condition. This document summarises the initial SMC assessment of velmanase alfa for treating mild to moderate mannosidosis. It is used for treating effects of the condition that do not involve the brain (non-neurological manifestations).
More information:
To find out more about velmanase alfa and how it works click on the links below.
ae-velmanase-alfa-lamzede-smc2466.pdf (pdf - 574449)
umar-velmanase-alfa-lamzede-final-aug-2022-for-website.pdf (pdf - 823758)
If you need the support of the advocacy team, please email Steve Cotterell at steve.cotterell@mpssociety.org.uk.