Novartis Receives FDA breakThrough Therapy Designation for BYM338 (Bimagrumab) for Sporadic Inclusion Body Myositis

Novartis has announced that FDA has granted breakthrough therapy designation to BYM338 for sporadic inclusion body myositis (sIBM).

Breakthrough therapy designation was created by FDA to expedite the development and review of new drugs for serious or life-threatening conditions. This designation is based on the results of a Phase II proof-of-concept study that showed BYM338 substantially benefited patients with sIBM compared with placebo. The results of this study will be presented at the American Neurological Association meeting on 14 October and is expected to be published in a major medical journal later this year.

sIBM is a rare yet potentially life-threatening muscle-wasting condition. Patients who have the disease can gradually lose the ability to walk, experience falls and injuries, lose hand function, and have swallowing difficulties[1]. There are no currently approved, (or established), treatment options for sIBM[2]. 

"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," said Timothy Wright, MD, Global Head of Development, Novartis Pharmaceuticals. "With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition."

BYM338 (Bimagrumab) and the Novartis Commitment to Research in Muscle Therapeutics

BYM338 (bimagrumab) is a novel, fully human monoclonal antibody developed to treat pathological muscle loss and weakness. BYM338 was developed by the Novartis Institutes for Biomedical Research (NIBR), in collaboration with Morphosys, whose HuCAL library was used to identify the antibody. BYM338 binds with high affinity to type II activin receptors, preventing natural ligands from binding, including myostatin and activin. BYM338 stimulates muscle growth by blocking signaling from these inhibitory molecules. 

In addition to being developed for sIBM, BYM338 is in clinical development for chronic obstructive pulmonary disease (COPD), cancer cachexia, sarcopenia and in mechanically ventilated patients. BYM338 is administered by intravenous infusion. 

Breakthrough Therapy Designation

According to FDA, breakthrough therapy designation is intended to expedite the development and review of drugs that treat serious or life-threatening conditions. The designation requires preliminary clinical evidence that demonstrates substantial improvement over currently available therapy. The designation includes all of the fast track program features, as well as more intensive FDA interaction and guidance. The breakthrough therapy designation is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.

About Sporadic Inclusion Body Myositis (sIBM)

sIBM is a rare disease, yet it is the most common degenerative disease of muscle in adults older than 65 years. It is characterised by a slowly progressive, asymmetric, atrophy and weakness of muscles. Commonly, patients become wheelchair bound within 10 to15 years of onset. Death may occur due to injurious falls, infection (aspiration pneumonia), or malnutrition.

Bimagrumab also was granted orphan drug designation in sIBM in both the US and Europe in 2012.