The investigational drug omigapil, under development by Santhera Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of congenital muscular dystrophy (CMD).
Preclinical studies in disease models have shown the drug inhibits cell death and reduces body weight loss and skeletal deformation while increasing mobility and improving life span.
In collaboration with the U.S. National Institutes of Health (NIH), Santhera is conducting an ongoing phase 1 clinical study (CALLISTO) evaluating omigapil’s pharmacokinetics (how the drug is absorbed, distributed and metabolized in the body), safety, and tolerability in 20 ambulatory and non-ambulatory patients ages 5 to 16, affected by either Ulrich or MDC1A subtypes of CMD. Completion of the study is expected by early 2017.
To learn more about CALLISTO, including site locations and complete inclusion and exclusion criteria, visit ClinicalTrials.gov and enter NCT01805024 into the search box.