The clinical development of omigapil in congenital muscular dystrophy (CMD)
Santhera Non-confidential report
Santhera is developing omigapil, an investigational medicinal product with anti-apoptotic properties, as a potential therapy for two forms of congenital muscular dystrophy (CMD) based on both in vitro and in vivo pre-clinical data. The Phase 1 clinical study (CALLISTO, NCT01805024) in patients with CMD has been completed. In response to the application, TACT gave an evaluation of a proposed clinical trial to investigate the efficacy of omigapil in patients with CMD, answered several specific questions about trial design and made recommendations about the need for further pre-clinical studies and suitable biomarkers to be used in a future study.
Name of applicant: Irinel Coserea Reviewed: November, 2018 in Chicago, IL, USA
Santhera is developing omigapil, an investigational medicinal product with anti-apoptotic properties, as a potential therapy for two forms of congenital muscular dystrophy (CMD) based on both in vitro and in vivo pre-clinical data. The Phase 1 clinical study (CALLISTO, NCT01805024) in patients with CMD has been completed. In response to the application, TACT gave an evaluation of a proposed clinical trial to investigate the efficacy of omigapil in patients with CMD, answered several specific questions about trial design and made recommendations about the need for further pre-clinical studies and suitable biomarkers to be used in a future study.
