Congenital Myotonic Dystrophy Treatment Gets FDA Fast Track

AMO Pharma announces FDA Fast Track designation for AMO-02 for treatment of congenital myotonic dystrophy.

AMO Pharma Limited, a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for AMO-02, the company’s investigational therapy in development for the treatment of congenital myotonic dystrophy.

Fast Track designation is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Drugs that receive Fast Track designation are eligible for more frequent meetings and written communications with the FDA, accelerated review and priority approval, and rolling New Drug Application review.

“Congenital myotonic dystrophy is a devastating neuromuscular disease where affected patients currently have no treatment options available,” said Michael Snape, chief executive officer of AMO Pharma. “The designation of Fast Track status for our development program for AMO-02 highlights both the urgent need for a treatment for patients and the potential for this novel therapy to offer a major advance in their care in the years ahead.”

AMO-02 (tideglusib) is in clinical stage development for the treatment of the severe form of myotonic dystrophy known as congenital DM1. In cellular and animal models of congenital DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to be increased. Inhibitors of GSK3ß have been shown to correct the activity of RNA binding proteins, such as CUGBP1, in animal models of DM1. AMO-02 is an inhibitor of GSK3ß that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell differentiation deficits in ex vivo tissue samples derived from patients with congenital DM1.

“Our ongoing phase 2 clinical trial for AMO-02 in the U.K. is the first sponsor-led clinical study in the treatment of congenital myotonic dystrophy and represents a historic milestone in research and a new era of hope for patients and their families affected by this serious condition,” Snape added. “In addition to advancing this development program, our research will provide many new insights to help shape the future of new research and to offer the prospect of better health for people living with myotonic dystrophy.”

(Source: PR Newswire)