Santhera Starts Campaign to Help Duchenne MD Patients Learn More About Breathing Challenges

Santhera Pharmaceuticals has started a campaign to help Duchenne muscular dystrophy patients  and their families learn more about the breathing challenges the condition can pose.

The company’s Take a Breath DMD campaign is focusing on the importance of providing patients with proper respiratory care. It began in the United States but will be extended to Europe in 2018.

Santhera, which develops treatments for muscle disorders like Duchenne MD, will offer online information on breathing difficulties, coughing and pulmonary care. Those visiting the campaign website can sign up to receive additional resources and interactive tools that the company creates.

“I applaud Santhera for developing such an educational resource focused on the respiratory aspects of Duchenne muscular dystrophy,” Dr. Oscar H. Mayer, a pediatric pulmonologist who specializes in neuromuscular diseases, said in a press release. “DMD is one of the most common and devastating types of muscular degeneration, and results in progressive muscle weakness and respiratory function decline that typically start in the first decade of life and lead to substantial life limitation.”

One of the muscle disorder therapies that Santhera has developed is Raxone (idebenone).  The European Union and Israel have approved it as a treatment for Leber’s hereditary optic neuropathy.

The agency that reviews European Union treatment applications recommended against the European Commission approving Raxone as a DMD treatment. Santhera plans to appeal the decision that the Committee for Medicinal Products for Human Use made in September 2017. The company is in the process of gathering additional information to reapply.

Raxone is designed to boost muscle cells’ ability to produce energy. Its goal is to improve the performance of mitochondria, the cells’ powerhouses.

Santhera is developing another therapy, omigapil, to treat congenital muscular dystrophies.

Studies have shown that omigapil can prevent muscular dystrophy patients from losing weight and experiencing skeletal deformation, and help them move better and live longer.

The U.S. Food and Drug Administration approved Santhera’s Investigational New Drug application for omigapil in May 2017. The FDA has also granted the treatment fast track and orphan drug status, designations that can speed up its regulatory approval. It has received orphan drug status in the European Union as well.