ASH 2023 | Exagamglogene autotemcel for severe sickle cell disease
Haydar Frangoul, MD, The Children’s Hospital at TriStar Centennial, Nashville, TN, shares insights into the safety and efficacy of exagamglogene autotemcel (exa-cel) for patients with severe sickle cell disease (SCD), highlighting recent data from the CLIMB SCD-121 study (NCT03745287). Results demonstrated an extensive benefit for patients receiving exa-cel, with 96% of patients remaining free of vaso-occlusive crisis (VOC). The study also showed an improvement in health-related quality of life (HRQoL) for patients. This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (edited for clarity)
The data that we presented shows a truly transformational benefit to patients with sickle cell disease. In our cohort, the 30 subjects that are evaluable for the primary endpoint, 96% remained vaso-occlusive disease-free, or crisis-free. So, 96% of the patients did not have any event related to sickle cell disease, and 100% of them did not get hospitalized, or have a vaso-occlusive crisis, after receiving exa-cel...
The data that we presented shows a truly transformational benefit to patients with sickle cell disease. In our cohort, the 30 subjects that are evaluable for the primary endpoint, 96% remained vaso-occlusive disease-free, or crisis-free. So, 96% of the patients did not have any event related to sickle cell disease, and 100% of them did not get hospitalized, or have a vaso-occlusive crisis, after receiving exa-cel. There was one patient that was hospitalized much later, close to two years out, because of a viral infection. But truly, the results are quite remarkable.
I’m hoping that now that this drug is available in the United States and the United Kingdom, that it will hopefully be more and more available for patients to consider doing it, to remove the suffering from sickle cell disease.
And we also actually, companion to that, presented data on the patient-reported outcomes that showed improvement in all categories, including pain, well-being, health and psychological benefit. So this therapy went beyond not getting only hospitalized, but actually patients are feeling better, and that’s based on their patient-reported outcomes.
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Disclosures
Jazz Pharmaceuticals: Speakers Bureau; Editas Medicine: Consultancy; Vertex Pharmaceuticals: Consultancy, Board of Directors/Advisory Committee; Rocket Pharmaceuticals: Consultancy, Member of DSMB for a study.
