According to a story from BioPortfolio, the specialty pharmaceutical company Santhera Pharmaceuticals recently announced that it has submitted its marketing application to the European Medicines Agency (EMA) for its drug idebenone (Puldysa) as a treatment for breathing problems associated with the rare degenerative disorder Duchenne muscular dystrophy. The drug indication is for those patients that are not currently using corticosteroids.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, boys are mostly affected, with girls only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.
Idebenone and Duchenne Muscular Dystrophy
The submission of the application follows a record of positive findings in the phase 2 and phase 3 clinical setting as well as data from a long term study that monitored Duchenne muscular dystrophy patients that were being treated with idebenone for a period of six years.
The company also plans to file an approval application with the US Food and Drug Administration (FDA) following the release of data from another phase 3 clinical trial that is testing the drug over an eighteen month period. This larger scale study is ultimately expected to include a total of 265 Duchenne muscular dystrophy patients. Idebenone has earned Orphan Drug status in both the US and EU and has also earned Fast Track designation in the US.
Decline in respiratory function is a major concern for Duchenne muscular dystrophy patients and is a major sign of disease progression. Slowing or halting the progression of breathing problems can offer vast improvements in quality of life for these patients.
