Novartis gets FDA fast track for treatment against severe muscle wasting with monoclonal that blocks myostatin and activin

Novartis announced today that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation to BYM338 for sporadic inclusion body myositis (sIBM).

Breakthrough therapy designation was created by the 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 to placebo. The results of this study will be presented at the American Neurological Association meeting on October 14 and is expected to be published in a major medical journal later this year.

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.

Myostatin inhibition can occur naturally in about one person in a million. People and animals with myostatin inhibition are naturally extremely muscular. Myostatin inhibition can be four times more effective than steroids for promoting muscle growth.

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.

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. There are no currently approved, (or established), treatment options for sIBM.

Breakthrough therapy designation
According to the 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 characterized 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.

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