New data further supports the clinical relevance of Peak Expiratory Flow as a predictor of disease progression in Duchenne muscular dystrophy.- Santhera Pharma.
Santhera Pharmaceuticals announces that new data further supporting the clinical relevance of Peak Expiratory Flow (PEF) and providing a link between the treatment effect of idebenone observed in the pivotal phase III DELOS study and measures of disease progression will be presented at the 15th International Congress on Neuromuscular Diseases (ICNMD) from July 6-10, 2018, in Vienna, Austria. At Santhera's lunchtime symposium on Sunday, July 8, a panel of clinical experts will present new data from natural history studies that support Peak Expiratory Flow as clinically relevant primary study endpoint.
This new data shows that Peak Expiratory Flow as percent predicted (PEF%p) is a sensitive and early marker of respiratory function decline in DMD applicable over a wide age range. Both PEF and Forced Vital Capacity (FVC) are predictors of time to clinically relevant events including time to hospitalization due to respiratory causes, time to initiation of assisted ventilation and death. Comparative analysis of the outcome of the DELOS trial with data from natural history studies allows the extrapolation of the observed treatment benefit of idebenone in PEF%p. This analysis shows that the treatment effect with idebenone observed in the DELOS trial can be linked to a delay in the initiation of assisted ventilation by 3 years, which is of high clinical relevance.
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