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NICE recommends cipaglucosidase alfa plus miglustat.

NICE recommends cipaglucosidase alfa plus miglustat as a treatment option for late onset Pompe in adults.

The National Institute for Health and Care Excellence has today published final guidance that recommends cipaglucosidase alfa (CIPA) plus miglustat, within its marketing authorisation, as an option for treating late-onset Pompe disease in adults.

The recommendation means that cipaglucosidase alfa would become a third enzyme replacement therapy option alongside alglucosidase alfa (ALGLU) and the more recent avalglucosidase alfa (AVAL).

The decision follows a committee meeting in early May which reviewed evidence from clinical trials, the biotechnology company Amicus and an expert clinician, as well as from other stakeholders. This included powerful testimony from AGSD-UK’s nominated patient expert on their experience of the condition and of the therapy. AGSD-UK had previously submitted evidence based on survey responses and interviews with community members.

Explaining the committee’s recommendations NICE commented:

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.

NICE go on to say that CIPA plus miglustat is indicated both for people who have had previous ERT treatment and for people who have not had ERT.

They refer to evidence from the clinical expert at the committee meeting who explained that data for CIPA plus miglustat and for AVAL suggested a more durable treatment effect than ALGLU so it was likely that newly diagnosed people would start on AVAL or CIPA plus miglustat. The expert indicated that clinicians would look at someone holistically when considering whether to switch treatment and suggested that people currently taking ALGLU would also be switched to AVAL or CIPA plus miglustat when response to ALGLU began to wane.

The recommendation highlights that because CIPA plus miglustat has been available through the early access to medicines scheme, NHS England and integrated care boards have agreed to provide funding to implement this guidance 30 days after publication. It also points out that 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 generally reflects NICE guidance, while there is a separate appraisal process in Scotland.

In July the Scottish Medicines Consortium concluded its assessment of AVAL, which had previously been approved by NICE in 2022, accepting the therapy for use throughout NHS Scotland for the treatment of patients with Pompe. AGSD-UK was also involved in submitting evidence as part of this process. The Product Update document for AVAL published by the Scottish Medicine Consortium is available here.

Scottish Medicines Consortium Product Update for AVAL

Full information on the NICE recommendation for CIPA plus miglustat is available on the NICE website:

Recommendations on NICE website

The availability of different treatment options for Pompe is extremely welcome, given the varying responses to therapies among people affected.