Today, Novartis announced that its BYM338 (bimagrumab) has been granted breakthrough therapy designation by the US Food and Drug Administration (FDA) for treatment of sporadic inclusion body myositis (sIBM).
This is Novartis’ third drug to receive breakthrough therapy designation from the agency.
sIBM is a rare, potentially life-threatening muscle-wasting condition. Patients with the disease can gradually lose the ability to walk, experience falls and injuries, lose hand function, and have swallowing difficulties. Currently, there are no approved treatment options for the condition. sIBM is the most common degenerative disease of muscle in adults over the age of 65. Typically, after ten to fifteen years of onset, patients become wheelchair bound. Death can occur from injurious falls, infections, or malnutrition.
“BYM338 is the third example this year of Novartis’ leadership in bringing breakthrough therapies to patients reinforcing our commitment to innovation addressing significant unmet medical needs and enhancing the lives of patients,” Timothy Wright, MD, Global Head of Development at Novartis Pharmaceuticals, stated. “With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition.”
The novel, fully human monoclonal antibody developed to treat pathological muscle loss and weakness was developed by Novartis in collaboration with Morphosys. The drug binds with high affinity to type II activin receptors, preventing natural ligands from binding, including myostatin and activin. BYM338 fuels muscle growth by blocking signaling from these inhibitory molecules.
The drug was granted orphan drug designation in sIBM in 2012 by both US and European health regulators. BMY338 is also in clinical development for treatment of chronic obstructive pulmonary disease (COPD), cancer cachexia, sacropenia and in mechanically ventilated patients.
Last updated: 8/20/13; 1:30PM EST