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
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
Last updated 21 September 2022 + show all updates
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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).
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First published.