SGT-001 declared orphan drug for DMD

Release Date: 30-Nov-2016

Solid Biosciences and Solid GT announces that US Food and Drug Administration (FDA) have granted Orphan drug status to SGT001 for the treatment of Duchenne Muscular Dystrophy (DMD). It is a rare and progressive muscle wasting disease that is usually diagnosed in childhood. SGT001 is the gene therapy mediated to amino-associated viral (AAV) vector. The gene therapy functions by potentially restoring the dystrophin expression in the muscles.


The drug has proved to provide long term systemic expression of comparatively shorter form of dystrophin in the muscles by its clinical studies. The gene therapy also benefits as improved muscle power and prevention from contraction induced damage. The therapy is expected to commercialize in early 2017.


The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.


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