FDA Grants ‘Breakthrough' Status To Novartis Muscle Drug, ‘First Real Option’ For Patients With sIBM

 @ZoeMintzz.mintz@ibtimes.com
on August 20 2013 11:46 AM

The U.S. Food and Drug Administration granted breakthrough therapy status to a newly developed compound used to treat a life-threatening muscle disease, Novartis said on Tuesday.

Novartis created the bimagrumab compound to treat patients with sporadic inclusion body myositis, a rare life-threatening condition that causes patients’ muscles to waste away until they eventually lose the ability to walk, Reuters reported.

"With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition," Timothy Wright,  the global head of development for Novartis, said in a statement.

The drug, also known as BYM338, is designed to boost muscle growth by blocking signals from inhibitory molecules. Novartis defines the compound as “a novel, fully human monoclonal antibody” to treat pathological muscle loss and weakness.

In clinical trials, patients who took the drug "substantially benefited" compared to those who took the placebo, Novartis said. BYM338 may also be used to treat patients with obstructive pulmonary disease (COPD), cancer cachexia, and sarcopenia, as well as mechanically ventilated patients.

The results from the Phase II proof-of-concept study that led to the FDA's special designation for the drug will be presented at the American Neurological Association meeting on Oct. 14.

While sporadic inclusion body myositis (sIBM) is rare, it is considered the most commonly acquired muscle disease associated with aging. Characterized by symptoms such as frequent falling episodes, difficulty climbing up stairs, weakened hand grip and difficulty swallowing, the disease usually affects elder Caucasian males over the age of 50.

The FDA developed the “breakthrough therapy” designation to help expedite the development of drugs for life-threatening conditions. Novartis said the new designation will involve “more intensive FDA interaction and guidance” while developing the new drug.  

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