Bluebird Closes In On First Approval For Neurodegenerative Disease Gene Therapy

The EU is set to be the first market to approve bluebird bio’s one-time gene therapy for cerebral adrenoleukodystrophy.

European flags in front of the Berlaymont building, headquarters of the European commission in Brussels.
bluebird's gene therapy Skysona could be approved in the EU soon • Source: Alamy

Skysona (elivaldogene autotemcel), bluebird bio’s gene therapy for cerebral adrenoleukodystrophy (CALD), is set to win its first ever marketing authorization now that the European Medicines Agency has recommended that the one-time treatment for the potentially fatal neurodegenerative disease be approved throughout the EU.

Skysona will be the first one-time gene therapy approved to treat CALD, a rare disease that occurs almost exclusively in...

Pink Sheet Podcast

Learn what's happening at the US FDA. On the go.

Derrick Gingery and the team bring you a weekly "Drug Fix".

More from Europe

EU Biosimilar Filings, Opinions And Approvals

 
• By 

<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>

EU Pharma Reform: What You Need to Know Now

 

As the reform of the EU pharmaceutical legislation progresses through the legislative process, the Pinks Sheet offers an infographic highlighting some of the key changes being proposed that will reshape the way drug companies run their business.

New EU Approvals

<p>Executive Summary<p> <p>The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add six new products, incl/ding Ixchiq, Valneva's chikungunya virus vaccine</p>

More from Geography

Canada Cuts Red Tape With Single Ethics Review For Trials

A new research ethics review system for multi-site clinical trials in Canada is expected to help improve study start-up times, avoid unnecessary duplication and boost the country’s competitiveness on a global stage.

Nancy Test Circulable Section Geography

 

This is a short description

Diversity Action Plans: What To Submit And When

 
• By 

With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.