(CIPA) plus miglustat is an enzyme replacement therapy that mimics the naturally occurring enzyme (alpha-glucosidase) which is lacking in late-onset Pompe disease. Miglustat is a medicine that helps the cipaglucosidase alfa enzyme be absorbed more readily by the cells in your body that are affected by late-onset Pompe disease.
People with late-onset Pompe disease have low levels of an enzyme called alpha-glucosidase. This enzyme helps the body control levels of glycogen (a type of carbohydrate) within the cells. Glycogen provides the body with energy. However, in late-onset Pompe disease the levels of glycogen can get too high, causing a build-up of glycogen in the muscles of the body which prevents them from working properly.
In June 2023 the National Institute for Health and Care Excellence (NICE) published final draft guidance recommending cipaglucosidase alfa (CIPA) plus miglustat as an option for treating late-onset Pompe disease in adults. It joins alglucosidase alfa (ALGLU) and avalglucosidase alfa (AVAL) as treatment options.
When a NICE highly specialised technologies guidance recommends the use of a drug or treatment, or other technology, the NHS in Wales must usually provide funding and resources for it within two months of the first publication of the final draft guidance.
Northern Ireland also usually mirrors NICE guidance and there is a separate treatment appraisal process in Scotland.
Full information on the recommendation is available on the NICE website here.