Retrophin announced that the independent Data Monitoring Committee for the pivotal Phase 3 FORT Study, which is evaluating fosmetpantotenate for the treatment of pantothenate kinase-associated neurodegeneration, has completed its scheduled clinical safety review required to open enrollment for pediatric patients. Upon review of the available safety and tolerability data of fosmetpantotenate in adult patients with PKAN in the study to date, the DMC recommended that the pivotal trial continue as planned, and supported initiation of enrollment in pediatric patients aged 6 to 17. The FORT Study is a pivotal Phase 3 clinical trial conducted under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration. Under the terms of the SPA agreement, the FORT Study protocol required an initial group of adult patients be treated with fosmetpantotenate to allow a safety assessment by the DMC prior to opening enrollment to pediatric patients aged 6 to 17. The Company anticipates completion of patient enrollment around year-end 2018, and top-line data to become available in the second half of 2019.
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