Decision

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.

• From: Medicines and Healthcare products Regulatory Agency
• Published: 8 March 2021
• Last updated: 29 March 2022 — See all updates

This publication was withdrawn on 15 July 2022

Withdrawn due to granting of marketing authorisation.

See Expired early access to medicines scheme scientific opinions for further information.

Documents

Avalglucosidase alfa Public Assessment Report (PAR)

PDF, 730 KB, 4 pages

Avalglucosidase alfa - Annex to Public Assessment Report (PAR) - first renewal

PDF, 707 KB, 2 pages

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Avalglucosidase alfa Treatment Protocol Information for healthcare professionals

PDF, 554 KB, 17 pages

Avalglucosidase alfa Treatment protocol Information for patients

PDF, 259 KB, 6 pages

This file may not be suitable for users of assistive technology.

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Avalglucosidase alfa Treament protocol Information on the Pharmacovigilance System

PDF, 153 KB, 5 pages

Avalglucosidase alfa Information for NHS Medical Director

PDF, 119 KB, 5 pages

Details

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 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

Published 8 March 2021
Last updated 29 March 2022 + show all updates

1. 29 March 2022

   Added Annex to Public Assessment Report first renewal PDF

2. 4 October 2021

   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.

3. 19 April 2021

   Updated the treatment protocol for healthcare professionals

4. 8 March 2021

   First published.