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Novartis scores FDA breakthrough-designation for rare disease product

Authored by Karl Simpson

Breakthrough Therapy designation has been given by the FDA to Novartis for their development asset , which is an orphan drug being developed to treat patients with sporadic inclusion body myositis, a rare and potentially life threatening disease which causes muscle-wasting.

Novartis already have achieved ‘breakthrough’ designation from the FDA for two other products in development for cardiovascular and cancer therapies respectively. The designation was introduced by the FDA to speed new therapeutic innovations to market and much has been made of the handful of designations which have been granted.

The Novartis asset is BYM338 (bimagrumab), a novel, fully human monoclonal antibody developed to treat pathological muscle loss and weakness.  was developed by the Novartis Institutes for Biomedical Research (NIBR) in collaboration with Morphosys, whose HuCAL library was used to identify the antibody. Morphosys recently hit the headlines when it announced a consider agreement with Celgene for the MOR202 product. 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.

The designation was awarded to Novartis on the back on a PoC study where it showed considerable benefits over the placebo. The read out for the trial is expected to be announced in October at the American Neurological Association meeting.

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