Skysona

Withdrawn

This medicine's authorisation has been withdrawn

elivaldogene autotemcel

Overview

On 18 November 2021, the European Commission withdrew the marketing authorisation for Skysona (elivaldogene autotemcel) in the European Union (EU). The withdrawal was at the request of the marketing authorisation holder, bluebird bio (Netherlands) B.V., which notified the European Commission of its decision not to market the product in the EU for commercial reasons. 

Skysona was granted marketing authorisation in the EU on 16 July 2021 for treatment of early cerebral adrenoleukodystrophy. No patients had been treated with Skysona since its marketing authorisation. 

The European Public Assessment Report (EPAR) for Skysona is updated to indicate that the marketing authorisation is no longer valid.

Skysona : EPAR - Medicine overview

Reference Number: EMA/413176/2021

Skysona : EPAR - Risk-management-plan summary

Product information

Skysona : EPAR - Product information

Skysona : EPAR - All authorised presentations

Product details

• Name of medicine:
  Skysona
• Active substance:
  elivaldogene autotemcel
• International non-proprietary name (INN) or common name:
  elivaldogene autotemcel
• Therapeutic area (MeSH):
  Adrenoleukodystrophy
• Anatomical therapeutic chemical (ATC) code:
  N07

Pharmacotherapeutic group

Other nervous system drugs

Therapeutic indication

Treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA) matched sibling haematopoietic stem cell donor is not available.

Authorisation details

• EMA product number:
  EMEA/H/C/003690
• Advanced therapy:

  This medicine is classified as an advanced therapy medicinal product (ATMP): a medicine for human use that is based on genes, tissues or cells. It offers groundbreaking new opportunities for the treatment of disease and injury. For more information, see Advanced therapy medicinal products: Overview.

• PRIME: priority medicine:

  This medicine was granted entry to the EMA Priority Medicines (PRIME) scheme during its development. PRIME is a scheme launched by EMA to enhance support for the development of medicines that target an unmet medical need. This voluntary scheme is based on enhanced interaction and early dialogue with developers of promising medicines, to optimise development plans and speed up evaluation so these medicines can reach patients earlier. For more information, see PRIME: priority medicines.

• Marketing authorisation holder:
  bluebird bio (Netherlands) B.V.

  Stadsplateau 7; 3521 AZ Utrecht; The Netherlands

• Opinion adopted:
  20/05/2021
• Marketing authorisation issued:
  16/07/2021

Assessment history

Initial marketing authorisation documents

Skysona : Orphan maintenance assessment report (initial authorisation)

Adopted Reference Number: EMADOC-1700519818-686727

Skysona : EPAR - Public assessment report

Adopted Reference Number: EMA/332184/2021

CHMP summary of positive opinion for Skysona

Adopted Reference Number: EMA/CHMP/273940/2021