Investigational Therapy for Duchenne Muscular Dystrophy is Seeking Orphan Drug Designation

According to a story from, the biotechnology company BIOPHYTIS has submitted documentation for orphan drug designation from the European Medicines Agency (EMA). This designation will be for Sarconeos, a drug developed by the company for the treatment of Duchenne muscular dystrophy.
Duchenne muscular dystrophy is the most common type of muscular dystrophy and tends to affect the muscles of the upper arms, upper legs, and pelvis. People with Duchenne muscular dystrophy eventually lose the ability to walk, and experience other symptoms such as scoliosis and increased risk of neurobehavioral disorders. The muscle atrophy that occurs in the condition is a due to an absence of dystrophin, a protein that is normally found in the muscles. As the heart and breathing muscles become involved, respiratory assistance becomes necessary. Most people with Duchenne muscular dystrophy only live into their 20s and 30s. To learn more about this disorder, click here.

Orphan drug designation would be a major benefit for Sarconeos, as candidates with orphan drug designation can accelerate the development process for the drug, and also enjoy an 8-10 year period of market exclusivity if the product is approved for public use. In 2017 a proof-of-concept demonstration of Sarconeos was tested on an animal model of the disease, and was able to reduce muscles fibrosis and improve muscular strength and exercise tolerance.

Clinical trials are expected to begin for the treatment later this year. There are a very limited number of effective treatment options available for Duchenne muscular dystrophy. However, Sarconeos has a unique mechanism of action and the demonstration suggests that it has significant potential to slow the progression of the condition. The drug was originally developed to treat an age related type of skeletal muscle degeneration called sarcopenia.

Duchenne muscular dystrophy represents a major medical need that is currently not being met; many patients do not have access to treatments that can significantly slow down disease progression, and Sarconeos represents a new opportunity for patients that could significantly extend their lifespan. Hopefully, the clinical trials will prove that Sarconeos can be an effective and safe new treatment for Duchenne muscular dystrophy.

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