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news.The US Food and Drug Administration (FDA) has accepted bluebird bio’s Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) gene therapy and granted priority review for the product’s use in the treatment of sickle cell disease (SCD).
lovo-cel gene therapy is intended for the treatment of sickle cell disease. Credit: Arek Socha from Pixabay.
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Bluebird is seeking approval for treating a rare genetic disease, SCD, in patients aged 12 years and above having a history of vaso-occlusive events.
A modified form of the β-globin gene added to lovo-cel produces anti-sickling adult hemoglobin thereby treating the underlying cause of SCD.
The BLA includes efficacy data from the HGB-206 study’s Group C cohort including 36 patients with median 32 months of follow-up and HGB-210 study including two patients, each followed for 18 months.
It also includes safety data from 50 patients including six patients followed for six or more years. It is claimed to be the longest follow-up of any gene therapy programme for SCD.
Commenting on the development, bluebird bio CEO Andrew Obenshain said: “The FDA’s acceptance of our BLA for lovo-cel moves us one step closer in bringing a potentially transformative therapy to the sickle cell disease community that is long overdue, and we are grateful to the patients, caregivers, researchers, clinicians, and community leaders who have enabled this exciting milestone.
“We look forward to working with the agency on its review.”
A Prescription Drug User Fee Act (PDUFA) goal date of 20 December this year has been assigned by the agency.
lovo-cel previously secured various designations from the FDA including fast track, orphan drug, rare pediatric disease, and regenerative medicine advanced therapy (RMAT).
If approved, lovo-cel will be the company’s third ex-vivo gene therapy.
