Safety Concerns Hit Bluebird Sickle Cell Trial, Again
The FDA placed a partial hold on its clinical program for lovo-cel due to an adolescent patient experiencing anemia 18 months after treatment, raising uncertainty over filing plans.
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San Rocco, whose potential rival gene therapy for thalassemia is in preclinical development, had wanted FDA to delay approval of bluebird’s BLA until resolution of a patent case and a ‘careful investigation’ of Zynteglo’s risks.
Interpretation of efficacy from single-arm studies in patients with cerebral adrenoleukodsytrophy complicated by comparability with external controls and limited follow-up, FDA says in advisory committee briefing documents which also cite three cases of myelodysplastic syndrome and raise broader questions about lentiviral vector safety.
The firm is laying off a big piece of its workforce and deprioritizing some investments to free up capital for potential upcoming launches, but that could add commercial risks.