Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration (FDA) has given its experimental product ACE-083 Orphan Drug designation. The treatment was granted the designation for the treatment of rare facioscapulohumeral muscular dystrophy.

About Facioscapulohumeral Muscular Dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is a rare, inherited type of muscular dystrophy that is characterized by initially affecting the muscles of the face, upper arms, and shoulders. There are multiple variants of facioscapulohumeral muscular dystrophy that are caused by a variety of genetic alterations and mutations. Symptoms typically begin to appear in childhood and is typically pronounced in the teenage years. Other muscles also become affected, and the progression of muscle atrophy is usually asymmetrical. Symptoms include drooping eyelids, a fixed facial expression, difficulty raising the arms and moving them when raised, foot drop, problems with breathing, generalized muscle weakness, and hearing loss. There are a variety of treatments and therapies that can help alleviate symptoms, but there is currently no treatment specifically approved for facioscapulohumeral muscular dystrophy. Life expectancy can be affected when the breathing muscles begin to decline. To learn more about facioscapulohumeral muscular dystrophy, click here.

About Orphan Drug Designation

The designation is reflective of the positive data so far that has been collected in the early stage of Phase 2 trials for ACE-083. The complete data for the Phase 2 trial is expected to be available next year.

Orphan Drug designation is reserved for investigational drugs that fulfill an unmet medical need or offer significant advantages in comparison to currently available treatments. In addition, the therapy must treat a disease that is defined as “rare.” According to FDA guidelines, a disease is regarded as rare if its affects 200,000 people or less in the US. The designation provides considerable benefits to the company, such as the waiving of certain fees in the application and development process, some tax benefits, and a period of seven year market exclusivity if the treatment gains approval.

ACE-083 was also granted Fast Track designation in May, which could allow the FDA to more closely work with Acceleron to develop the drug and potentially lead to an expedited review process.

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