Santhera’s Congenital Muscular Dystrophies Drug Omigapil Receives FDA Fast Track Designation

Switzerland-based Santhera Pharmaceuticals‘ drug candidate omigapil has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of congenital muscular dystrophies (CMD).

Omigapil is a prenyl-analog with anti-apoptotic properties. Nonclinical studies in disease-relevant models show the drug inhibits cell death and reduces body weight loss and skeletal deformation while increasing locomotive activity and providing protection from early mortality. Santhera is now developing omigapil under an open Investigational New Drug (IND) application also granted by the FDA. In addition, omigapil holds an Orphan Drug designation for CMD in the U.S. and the European Union.

Santhera, in collaboration with the U.S. National Institutes of Health (NIH), is now conducting a Phase I clinical study (CALLISTO) evaluating omigapil’s pharmacokinetics, safety, and tolerability in 20 ambulatory and non-ambulatory patients ages 5 to 16, affected by either Ulrich or MDC1A subtypes of CMD.

Prof. Carsten Bönnemann, the principal investigator leading the study, expects completion by early 2017. The study is being conducted at the NIH’s National Institute of Neurological Disorders and Stroke in Bethesda, Maryland, and is funded by the American Cure CMD, the Swiss Foundation for Research on Muscle Diseases, and EndoStem (a European program).

“We are delighted that the FDA has granted Fast Track designation for omigapil, thereby underlining the unmet medical need for an effective therapy for CMD patients and the severity of this class of diseases,” said Santhera CEO Thomas Meier, Ph.D., in a press release. “After Raxone, omigapil is our second neuromuscular pipeline product and underpins our dedication to developing effective medicines to satisfy the needs of patients suffering from mitochondrial and other rare diseases.”

CMD is a group of inherited neuromuscular conditions characterized by distinct forms of progressive loss of muscle tissue. Complications associated with CMD include loss of body weight, skeletal deformations, and respiratory distress, resulting in immobility at a young age and early mortality.

Severe forms of CMD can affect newborns or young children with life-threatening progressive muscle weakness. There are currently no treatments available to slow or stop the progression of this disease, and options are confined to respiratory support and orthopedic surgery for scoliosis, as well as supplementary nutrition to avoid malnourishment.