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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Ovum preservation and sperm banking are options for post-puberty patients, but busulfan – used for myeloablative conditioning before certain gene therapies – may cause hesitation to use some treatments in children, experts at ASH told Scrip.
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.