Decision

Cipaglucosidase alfa with miglustat in the treatment of late-onset Pompe disease

EAMS scientific opinion issued to Amicus Therapeutics UK Limited for cipaglucosidase alfa with miglustat in the treatment of adult patients with late-onset Pompe disease previously treated with alglucosidase alfa.

Documents

Cipaglucosidase alfa with miglustat: Public Assessment Report (PAR)

Request an accessible format.
If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Cipaglucosidase alfa with miglustat: Treatment protocol: Information for healthcare professionals

Request an accessible format.
If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Cipaglucosidase alfa with miglustat: Treatment protocol: Information for patients

Request an accessible format.
If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Cipaglucosidase alfa with miglustat: Treatment protocol: Information on the Pharmacovigilance System

Request an accessible format.
If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Cipaglucosidase alfa with miglustat: Information for NHS Medical Director

Request an accessible format.
If you use assistive technology (such as a screen reader) and need a version of this document in a more accessible format, please email webupdates@mhra.gov.uk. Please tell us what format you need. It will help us if you say what assistive technology you use.

Details

The treatment protocol for healthcare professionals has been updated to include new safety data from ongoing clinical trials in section 4.8 as well as information on miglustat interaction with food (section 4.5).

Detail

For the full EAMS indication please see section 4.1 of the Treatment protocol: Information for healthcare professionals

The scientific opinion includes:

  • a public assessment report (PAR)
  • a treatment protocol:
    • for healthcare professionals
    • for patients
    • on the pharmacovigilance system
  • Information for NHS Medical Directors

Information and details regarding patient access

For new patients wishing to access EAMS medicines in England, trusts must register patients on the NHS England web-based registration system. Queries to NHS England regarding the scheme can be submitted to england.eams@nhs.net

For information about access in Northern Ireland, contact Chief Pharmaceutical Officer and secondary.care@health-ni.gov.uk

For information about access in Scotland contact the Scottish Government Directorate General Health & Social Care at medicines.policy@gov.scot

For information about access in Wales, contact the Welsh Government Health and Social Services Group by emailing Andrew Evans, Chief Pharmaceutical Officer or Lynne Schofield, Head of Pharmacy and Prescribing policy at Pharmacyand.PrescribingBranch@gov.wales

Updates to this page

Published 8 June 2021
Last updated 21 September 2022 + show all updates
  1. The treatment protocol for healthcare professionals has been updated to include new safety data from ongoing clinical trials in section 4.8 as well as information on miglustat interaction with food (section 4.5).

  2. First published.

Sign up for emails or print this page