The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug Sarconeos, developed by the company Biophytis, for the treatment of Duchenne muscular dystrophy. The full story can be read here, at Financial Buzz.

Duchenne muscular dystrophy (DMD) is an inherited genetic condition that causes muscles to progressively weaken, causing disability and a shortened lifespan. It usually (but not always) affects young boys, one in every 3,500 of which will be born with the condition. People with DMD have a gene variation that causes them to lack the protein dystrophin, which is involved in muscle maintenance. Symptoms usually first appear between the ages of three and five and include weakness in the upper arm, shoulder, hip, and thigh muscles. As a result, children with the condition often find it difficult to stand up, climb stairs, raise their arms, and keep their balance. As the condition progresses, it will begin to affect the heart and respiratory system. Current treatments for DMD are limited and usually focus on managing symptoms and slowing disease progression. However, researchers are working on several types of potential new treatments, including gene therapies, gene repair, exon skipping, and stop-codon read through.

Sarconeos represents another current line of research. Sarconeos is the first drug of its kind and works by activating the MAS receptor to increase muscle growth. It also inhibits myostatin, a protein that prevents muscle growth. Studies on animal models of DMD have shown that the drug can improve the effects of the condition. It was found to reduce muscle fibrosis and slow or stop DMD progression, delaying the loss of walking ability and overall movement.

Based on these promising results, the FDA has awarded the drug Orphan Drug status in the US. The Orphan Drug designation grants the drug developers several benefits to support and encourage drug development. These can include a longer period of exclusive drug marketing rights, co-funding of clinical trials, and increased communication between the FDA and company. The developers, Biophytis, called the designation a “significant milestone” and says that they are aiming to achieve a similar designation in Europe in the next few weeks.

Following that, they hope to progress the drug on to clinical trials.

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