1 Recommendations

1.1 Cipaglucosidase alfa (CIPA) plus miglustat is recommended, within its marketing authorisation, as an option for treating late-onset Pompe disease in adults. It is recommended only if the company provides it according to the commercial arrangement.

Why the committee made these recommendations

The standard treatment for late-onset Pompe disease is enzyme replacement therapy (ERT) with alglucosidase alfa (ALGLU) or avalglucosidase alfa (AVAL). CIPA plus miglustat is an alternative ERT.

The results of clinical trials show that CIPA plus miglustat seems to improve walking and breathing compared with ALGLU in the short term, but the long-term effects are uncertain. CIPA plus miglustat has only been compared indirectly with AVAL. It appears to be as effective, but this is uncertain.

There are also uncertainties in the cost-effectiveness model. But, compared with AVAL and ALGLU, there is a positive net health benefit for CIPA plus miglustat. This implies that overall population health will be increased if CIPA plus miglustat is an available treatment option. So, considering the total annual costs of the treatment options and despite the uncertainties in the clinical-effectiveness results and the model, CIPA plus miglustat is recommended for routine use.

  • National Institute for Health and Care Excellence (NICE)