Another breakthrough status for Novartis!

In Clinical Development, Regulation & Government by André SingerLeave a Comment

After winning breakthrough status for its lung cancer drug LDK378 and the heart failure candidate Serelaxin, Novartis has managed again to win FDA’s blessing for the agency’s fast track programme, now for what could become the first treatment for sporadic inclusion body myositis (sIBM), a rare life-threatening muscle-wasting condition. The Swiss company’s candidate Bimagrumab (BYM338), a monoclonal antibody, has shown in its phase II proof-of-concept substantial benefits for patients with sIBM compared to placebo. The FDA’s decision and the promising data presented has also contributed to a more intensive interaction and guidance between the agency and Novartis. After receiving orphan drug designation in both US and Europe in 2012, this new status will boost Bimagrumab’s development pace even further. 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. So here’s a double opportunity for Novartis in terms of expanding the utility of the drug to a broader market. The study is being conducted by Novartis Institutes for Biomedical Research in collaboration with Morphosys. The Swiss drug maker does not have a specific unit for rare disease research, like Pfizer and Shire for example, however it has been really successful in approaching multiple opportunities to expand the clinical utility of oncology products to rare types of the disease. John Orloff, Novartis Chief Medical Officer, has twice presented at the World Orphan Drug Congress USA, and addressed important aspects about the company’s approach to narrowing unmet medical needs in the rare disease space.

After winning breakthrough status for its lung cancer drug, LDK378 and the heart failure candidate, Serelaxin, Novartis has again managed to win the FDA’s blessing for the agency’s fast track programme. This time it is for now for sporadic inclusion body myositis (sIBM), a rare life-threatening muscle-wasting condition.

The Swiss company’s candidate Bimagrumab (BYM338), a monoclonal antibody, has shown, in its phase II proof-of-concept, substantial benefits for patients with sIBM compared to a placebo. The promising data presented has contributed to a more intensive interaction and guidance between the FDA and Novartis. After receiving orphan drug designation in both the US and Europe in 2012, this new status will boost Bimagrumab’s development pace even faster.

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. So here, once again, is a double opportunity for Novartis in terms of expanding the utility of the drug to a broader market.

The study is being conducted by Novartis Institutes for Biomedical Research in collaboration with Morphosys. The Swiss drug maker does not have a specific unit for rare disease research, like Pfizer and GSK for example, however, it has been so successful in approaching multiple opportunities to expand the clinical utility of oncology products to rare types of a disease.

John Orloff, Novartis’s Chief Medical Officer, has often presented at the World Orphan Drug Congress USA, and addressed important aspects about the company’s approach to narrowing unmet medical needs in the rare disease space.

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