According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to file for Conditional Marketing Authorization with the European Medicines Agency (EMA). This authorization is intended for the company’s product Puldysa (active ingredient idebenone) as a treatment for breathing issues associated with Duchenne muscular dystrophy, a rare, progressive, degenerative disorder.
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.
A New Potential Treatment
The completion of this authorization is expected by the middle of 2019. The decision to file for the marketing approval is the result of extensive data that conclusively demonstrates the effectiveness of Puldysa in patients with Duchenne muscular dystrophy. For patients with this disorder, Puldysa was capable of providing meaningful relief from respiratory distress for a maximum of six years. This benefit means that patients will see significant delays in the progression of their disease. These delays could result in less hospitalizations related to adverse respiratory events. The therapy is intended for Duchenne muscular dystrophy patients that are not being treated with glucocorticoids.
Puldysa has earned Orphan Drug designations the US, Europe, and Australia as a therapy for Duchenne muscular dystrophy. If this Conditional Marketing Application is approved, then patients in the EU will have a new therapeutic option that can meaningfully improve their quality of life and delay the progression of Duchenne muscular dystrophy.