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Casgevy (exagamglogene autotemcel) is indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). Casgevy was evaluated in an ongoing single-arm, multicenter, phase 1/2/3 trial with patients who had a history of at least 2 protocol-defined severe VOC events during each of the 2 years prior to screening. Of the 63 patients enrolled in the trial, 44 patients went on to receive Casgevy to form the full analysis set (FAS). Of the 44 patients from the FAS, 31 had adequate follow-up to allow evaluation of the primary efficacy endpoint and formed the primary efficacy set (PES). The primary efficacy outcome was the proportion of VF12 responders, defined as patients who did not experience any protocol-defined severe VOCs for at least 12 consecutive months within the first 24 months after treatment with Casgevy. The VF12 response rate was 29/31 (93.5%, 98% one-sided CI: 77.9%, 100.0%). The proportion of patients who did not require hospitalization due to severe VOCs for at least 12 consecutive months within the 24-month evaluation period (HF12) was also assessed. One patient was not evaluable for HF12 response; the remaining 30 patients achieved the endpoint of HF12 (100% [98% one-sided CI: 87.8%, 100.0%]). The most common grade 3 or 4 adverse reactions with an incidence ≥25% were mucositis, febrile neutropenia, and decreased appetite. The most common grade 3 or 4 laboratory abnormalities (≥ 50%) were neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.

Casgevy™ (exagamglogene autotemcel)

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