The drug is branded as Skysona. The approval is restricted to patients less than 18 years old with an ABCD1 genetic mutation.
The one-time gene therapy is targeted at patients who do not have a matched sibling donor for hematopoietic stem cell (HSC) transplant.
The marketing authorization is supported by efficacy and safety data from the Phase 2/3 Starbeam study (ALD-102), where 90% of patients treated the drug met the primary endpoint of major functional disability (MFD)-free survival at two years follow-up, reported the biotech.
Positive review of Zynteglo
The go-ahead for that gene therapy follows the positive recommendation earlier this month from the EMA in its review of buebird bio’s Zynteglo.
The European Medicines Agency’s (EMA) safety committee, Pharmacovigilance Risk Assessment Committee (PRAC), concluded, that there is no evidence betibeglogene autotemcel (beti-cel) gene therapy, licensed as Zynteglo in the EU, causes the blood cancer, acute myeloid leukemia (AML).
Based on the review of all available data, the PRAC said that the benefit-risk balance of medicinal products containing Zynteglo remains favorable.
As a result, Bluebird bio lifted the voluntary suspension on the drug.
Zynteglo is a gene therapy for the blood disorder beta thalassemia. It is a one-time treatment for patients that are 12 years and older who require regular blood transfusions. It was granted conditional marketing authorization in the EU in May 2019.
It uses a viral vector to deliver a working gene into the patient’s blood cells.