View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. News
September 19, 2022

FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy

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

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.

Free Whitepaper
img

Optimise your cell therapy process: a guide to cell thawing

Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.
by Cytiva Thematic

By clicking the Download Free Whitepaper button, you accept the terms and conditions and acknowledge that your data will be used as described in the Cytiva Thematic privacy policy By downloading this Whitepaper, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services.

Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. A weekly roundup of the latest news and analysis, sent every Friday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Pharmaceutical Technology