Bluebird Bio's Skysona (elivaldogene autotemcel;) has received Accelerated Approval from the US Food and Drug Administration (FDA). eli-cel) to slow the progression of neurologic dysfunction in early, active patients with Adrenoleukodystrophy Drugs Development Market.
The treatment is demonstrated for use in young men matured four to 17 years with CALD.
Through specific Qualified Treatment Communities in the country, Skysona is expected to be made accessible economically before the current year's over.
Using the Priority Review method, the regulatory body examined the therapy's Biologics License Application (BLA).
After receiving approval, bluebird bio received a rare voucher for pediatric priority review.
The most recent development is based on the results of the Phase III ALD-104 and Phase II/III ALD-102 (Starbeam) open-label, single-arm clinical trials.
The Major Functional Disability (MFD)-free survival from symptom onset (NFS 1) in symptomatic Skysona-treated and untreated subjects was evaluated in a post-hoc enrichment assessment.
At 24 months after the first NFS 1, subjects who received Skysona had a 72% chance of MFD-free survival, compared to 43% in subjects who were not treated.
The most common non-laboratory adverse reactions included, among others, mucositis, vomiting, nausea, febrile neutropenia, alopecia, and decreased appetite.
Skysona is a one-time gene therapy that adds functional copies of the ABCD1 gene to the patient's hematopoietic stem cells by using ex-vivo transduction with the Lenti-D lentiviral vector.
Andrew Obenshain, CEO of bluebird bio, stated: Since the company's inception more than a decade ago, the mission of bluebird has placed children with CALD and their families at its center.
"This long-awaited approval represents significant hope for the ALD community and provides families with a new option where many previously had none,"
Zynteglo, a treatment for transfusion-dependent beta-thalassemia, received conditional marketing authorization from the European Commission in June 2019.