Decision

Voxelotor in the treatment of sickle cell disease

EAMS scientific opinion issued to Global Blood Therapeutics (UK) Ltd for Voxelotor in the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide.

Documents

WITHDRAWN - Voxelotor: Public Assessment Report (PAR)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.

Voxelotor: 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.

Voxelotor: 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.

Voxelotor: 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.

Voxelotor: 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

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 28 January 2022
Last updated 25 October 2022 + show all updates
  1. Public Assessment Report (PAR) withdrawn

  2. 'Treatment protocol: Information on the Pharmacovigilance System' and 'Treatment protocol: Information for healthcare professionals' have been revised to include a new email address for adverse drug reaction reporting to the scientific opinion holder.

  3. First published.

Sign up for emails or print this page