FDA Grants Fast Track Status to Marathon’s Duchenne Muscular Dystrophy Drug

Marathon Pharmaceuticals’ investigational Duchenne Muscular Dystrophy (DMD) drug was granted Fast Track designation by US health regulators.

The rare-disease focused biopharmaceutical company recently announced that the US Food and Drug Administration (FDA) granted fast track status for its deflazacort for treatment of patients with DMD, a fatal muscle disease found most often in boys. Marathon said that it hopes to receive federal approval for the drug by 2016.

DMD is a recessive X-linked form of muscle dystrophy, which results in muscle degeneration, difficulty walking, breathing and death. The disease affects approximately 20,000 people in the US. Currently, there is no cure for DMD, and treatment is typically aimed at controlling the onset of symptoms to maximize patients’ quality of life.

Deflazacort is a glucocorticoid with an anti-inflammatory and immunosuppressant properties. Marathon is currently conducting several clinical and preclinical studies evaluating the drug to support approval in patients with DMD. Previous published clinical studies show that deflazacort may be an important new treatment option for this patient population. Side effects associated with the drug reported to date include cushingoid appearance, irritability and cataract formation. In addition to fast track status, the FDA granted orphan drug designation for deflazacort as well.

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“Marathon is committed to improving the lives of patients with Duchenne Muscular Dystrophy and other rare diseases. Fast Track designation for deflazacort is an important milestone for DMD patients and their families. We will work to take full advantage of this opportunity to accelerate availability to patients,” said Jeffrey Aronin, chief executive officer of Marathon.

Deflazacort has been available in other countries such as Canada and Mexico for several years. It was originally developed by Marion Merrell Dow, now Aventis. It has been off patent for a long time, but the company never sought approval in the US. Therefore, Marathon expects to receive seven years of market exclusivity with FDA approval.

Source: Marathon Pharmaceuticals, LLC

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