New Advances in Santhera’s Therapeutic Drug Development for Duchenne Muscular Dystrophy

Santhera Pharmaceuticals, a Swiss company focused on the treatment of mitochondrial and neuromuscular diseases, recently announced that it has discussed with the U.S. Food and Drug Administration (FDA) its plans of submitting a New Drug Application (NDA) for Raxone ® /Catena ® (idebenone), intended as a therapy for Duchenne muscular dystrophy (DMD).

DMD is an inherited disorder caused by a defective gene called dystrophin and characterized by a rapid progressive skeletal muscle weakness caused by chronic inflammation and the degeneration of muscle cells and tissue, which can compromise locomotion and respiratory function, leading to breathing complications and cardio-respiratory failure. DMD has a rapid progression and affects mainly boys. The majority of the DMD patients require a wheelchair by the age of 12 and patients often succumb to the disease in their 20s.

Due to the loss of the dystrophin protein, DMD patients have a mitochondrial dysfunction in their muscle cells characterized by elevated oxidative stress (with increased production of harmful reactive oxygen species – ROS) and reduced energy production. Idebenone is an organic compound of the quinone family and an analogue of the coenzyme Q10 that is able to stimulate mitochondrial energy production in muscle cells and tissues and inhibit ROS production.

Idebenone has been granted orphan drug designation for DMD in both the United States and Europe, and was recently granted Fast Track designation for DMD treatment by the FDA, which will accelerate the development and review process in order to make this relevant drug available to the patients earlier. Recent data from the DELOS Phase III trial (NCT01027884) revealed that idebenone has a positive impact in preserving the respiratory function in DMD patients.

“In our meeting with the FDA we also communicated that we have recently started a collaboration with CINRG [Cooperative International Neuromuscular Research Group] to compare the outcomes of the successful Phase III DELOS trial with a comprehensive data-set collected by CINRG in their Duchenne Natural History Study over the past years. We are confident that these comparative analyses will further support the clinical relevance of the DELOS trial results which showed a clinical benefit on respiratory function in patients not taking concomitant glucocorticoid steroids. Our Fast Track designation allows us regular interactions with the FDA and we will now prepare for a second meeting with the Agency to discuss our plans for submitting an NDA in light of these emerging data” said the CEO of Santhera Dr. Thomas Meier in a news release. “In parallel to these ongoing activities we are continuing to prepare the NDA for submission.”

The NDA is the vehicle to obtain FDA approval for sale and marketing of new pharmaceuticals in the United States.