Skip to content

Cipaglucosidase alfa

Cipaglucosidase alfa (CIPA) plus miglustat (also known as (Pombiliti) is a treatment for adults with late-onset Pompe disease  developed by Amicus Therapeutics. 

How does it work?

(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.

Recommendation for use

In August 2023 the National Institute for Health and Care Excellence (NICE) published final draft guidance (CIPA) plus miglustat as an option for treating late-onset Pompe disease in adults.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.  

Full information on the recommendation is available on the NICE website here.

NICE’s recommendation means that it is also available in Wales and Northern Ireland. 

In December 2023 (CIPA) plus miglustat was also accepted for use within NHS Scotland by the Scottish Medicines Consortium. More information on this is available on the SMA website here. 

Support and information

Metabolic myopathy

Pompe disease is a metabolic myopathy. Also known as metabolic muscle conditions, these are conditions that interfere with the way muscles provide energy.

Research

We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.

Our support services

We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.

Our helpline is open Monday-Thursday 10am-2pm.