Avalglucosidase alfa in the treatment of Pompe disease
EAMS scientific opinion issued to Aventis Pharma Ltd t/a Sanofi for avalglucosidase alfa in the treatment of late- and infantile-onset Pompe disease for patients who have already received enzyme replacement therapy with alglucosidase alfa.
Documents
Details
Detail
For the full EAMS indication please see section 4.1 of the Treatment protocol: Information for healthcare professionals.
The scientific opinion includes:
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a public assessment report (PAR)
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a treatment protocol:
- for healthcare professionals
- for patients
- on the pharmacovigilance system
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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 Ryan Wilson, Secondary Care Directorate at ryan.wilson@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
Last updated 29 March 2022 + show all updates
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Added Annex to Public Assessment Report first renewal PDF
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The treatment protocols have been updated to allow for home administration of avalglucosidase alfa. Changes have been made to the: Treatment protocol for healthcare professionals – update to section 4.2; Treatment protocol for patients – update to section 3; Treatment protocol Information on the Pharmacovigilance System.
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Updated the treatment protocol for healthcare professionals
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First published.