A phase 1 trial to test omigapil in congenital muscular dystrophy (CMD) conducted at the National Institutes of Health is complete and data indicate it met its primary objective. Results from the trial, also known as CALLISTO, show a favorable pharmacokinetic profile of omigapil and demonstrated that the drug was safe and well-tolerated in children and adolescents with MDC1A and Collagen VI subtypes of CMD. (“Pharmacokinetics” refers to how the drug is absorbed, distributed and metabolized in the body.)

Omigapil is an anti-apoptotic drug designed to prevent cell death. In previous studies in a mouse model with type 1A congenital muscular dystrophy (MDC1A), results have indicated that treatment with omigapil inhibited cell death, reduced weight loss and skeletal deformation, improved motor function and increased life span.

Under development by Santhera Pharmaceuticals, the drug has received U.S. Food and Drug Administration (FDA) Orphan Drug and Fast Track designations for the treatment of CMD.

For more information about omigapil and the completed clinical trial, download and read an FAQ provided by Santhera Pharmaceuticals.