FDA lifts partial clinical hold on studies of gene therapy for sickle cell disease
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The FDA lifted its partial clinical hold on bluebird bio’s sickle cell disease studies evaluating lovotibeglogene autotemcel among patients aged younger than 18 years, according to the agent’s manufacturer.
Lovotibeglogene autotemcel (bluebird bio), also called lovo-cel, is an investigational one-time gene therapy being studied for sickle cell disease.
Lovo-cel clinical studies had been placed on partial hold for children and adolescents amid an investigation by the manufacturer into “an adolescent patient with persistent, nontransfusion-dependent anemia following treatment with lovo-cel,” a bluebird bio press release stated.
Results of the investigation — shared at ASH Annual Meeting and Exposition earlier this month, in addition to findings of a separate persistent anemia case following treatment with lovo-cel — showed both patients had alpha-thalassemia trait. Those two patients are the only two in the study with such a trait.
The specific genotype has been added to exclusion criteria for ongoing studies.
“We are very pleased to have addressed the FDA’s questions and resolved the partial clinical hold,” Richard Colvin, MD, PhD, chief medical officer of bluebird bio, said in the press release. “We are working closely with study investigators and clinical trial sites to resume enrollment and treatment of pediatric and adolescent patients in the first quarter of next year.”
Bluebird bio previously suspended clinical trials using its LentiGlobin product for sickle cell disease due to reported cases of hematologic malignancies among patients who received the treatment.
The temporary suspension applied to a phase 1/phase 2 HGB-206 trial and phase 3 HGB-210 trial of bluebird bio’s LentiGlobin-based investigational therapy for patients with sickle cell disease.
According to the manufacturer, enrollment and dosing for adults in the HGB-210 study continued as planned while the partial hold was in effect for patients aged younger than 18 years. Patients in HGB-206 Group C have completed treatment and will form the primary basis for efficacy in its lovo-cel biologics license application submission.
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