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Novartis orphan drug wins 'breakthrough' status from FDA

Bimagrumab could be first treatment for rare life-threatening muscle-wasting condition

FDA headquarters White Oak 

Novartis' investigational treatment for a rare life-threatening muscle-wasting condition has been awarded 'breakthrough therapy designation' by the FDA.

The US regulator's decision should speed up Novartis' development of bimagrumab, which will now benefit from all the features of the FDA's fast track programme, as well as more intensive interaction and guidance with the regulator.

Bimagrumab (BYM338) could become the first treatment for sporadic inclusion body myositis (sIBM), which can see patients 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 and the FDA's decision was based on a phase II proof-of-concept study of bimagrumab, which showed substantial benefits for patients with sIBM compared to placebo.

Results from the study will be presented at the American Neurological Association meeting in October and are also expected to be published in a major medical journal later this year.

A monoclonal antibody, bimagrumab was developed by the Novartis Institutes for Biomedical Research (NIBR) in collaboration with Morphosys, whose HuCAL library was used to identify the antibody.

The drug works by binding with high affinity to type II activin receptors, preventing natural ligands from binding, including myostatin and activin, and then stimulating muscle growth by blocking signalling from these inhibitory molecules.

Although it is a rare disease, sIBM is also the most common degenerative disease of muscle in adults older than 65 years and often causes patients to be wheelchair bound within 10 to15 years of onset.

Bimagrumab, which was granted orphan drug designation in sIBM in both the US and Europe in 2012, follows Novartis' lung cancer drug LDK378 and its heart failure candidate serelaxin in winning breakthrough status from the FDA.

Timothy Wright, global head of development at Novartis Pharmaceuticals, said: "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.

"With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition."

20th August 2013

From: Research



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