The US Food and Drug Administration (FDA) has awarded Accelerated Approval for bluebird bio’s Skysona (elivaldogene autotemcel; eli-cel) for slowing neurologic dysfunction progression in early, active cerebral adrenoleukodystrophy (CALD) patients. 

The treatment is indicated for usage in boys aged four to 17 years with CALD.

Through certain Qualified Treatment Centers in the country, Skysona is anticipated to be made available commercially by the end of this year.

The regulatory agency carried out the review of the Biologics License Application (BLA) for the therapy under the Priority Review approach.

bluebird bio was granted a rare paediatric priority review voucher following approval. 

The latest development is based on findings from the single-arm, open-label Phase II/III ALD-102 (Starbeam) and Phase III ALD-104 clinical trials.

In a post-hoc enrichment assessment, symptomatic patients were evaluated for Major Functional Disability (MFD)-free survival from symptom onset (NFS ≥ 1) in Skysona-treated and untreated subjects. 

Subjects who received Skysona had 72% chances of MFD-free survival at 24 months from the time of first NFS ≥ 1 versus 43% in untreated subjects.

Mucositis, vomiting, nausea, febrile neutropenia, alopecia and reduced appetite among others were reported to be the most prevalent non-laboratory adverse reactions reported.

A one-time gene therapy, Skysona leverages ex-vivo transduction with the Lenti-D lentiviral vector for adding the ABCD1 gene’s functional copies into the hematopoietic stem cells of the patient. 

bluebird bio CEO Andrew Obenshain said: “Children with CALD and their families have been at the heart of bluebird’s mission since the company was founded more than a decade ago. 

“For the ALD community, this long-awaited approval represents significant hope and offers families a new option where, for many, there had been none.”

In June 2019, the European Commission granted conditional marketing authorisation for bluebird bio’s Zynteglo to treat transfusion-dependent β-thalassemia.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.