Skysona becomes first and only gene therapy approved in EU for CALD

Skysona has become the first and only gene therapy approved in the EU to treat early Cerebral Adrenoleukodystrophy (CALD), a rare neurodegenerative disease that can lead to progressive, irreversible loss of neurologic function, and death.

The European Commission (EC) granted marketing authorisation for Skysona (elivaldogene autotemcel, Lenti-D) to bluebird bio for the treatment of early CALD in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available.

Previously, the only therapeutic option available to CALD patients was transplantation of stem cells from a donor, called allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with severe potential complications and mortality that increase in patients without a matched sibling donor (MSD). It is estimated that more than 80% of patients diagnosed with CALD do not have an MSD.

Skysona was reviewed as part of the European Medicines Agency’s Priority Medicines scheme (PRIME) and was previously granted Orphan Medicinal Product status. The marketing authorization is valid in all 27 member states of the EU, as well as Norway, Liechtenstein, and Iceland.

A message from the Editor:

Thank you for reading this story on our news site - please take a moment to read this important message:

As you know, our aim is to bring you, the reader, an editorially led news site but journalism costs money and we rely on advertising and digital revenues to help to support them.

With the Covid-19 lockdown having a major impact on our industry as a whole, the advertising revenues we normally receive, which helps us cover the cost of our journalists and this website, have been drastically affected.

As such we need your help. If you can support our news sites with a small donation of even £1, your generosity will help us weather the storm and continue in our quest to deliver quality journalism.

In the meantime may I wish you the very best.

- Advertisement -

Related news