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Novartis Receives 18th FDA Breakthrough Therapy Designation

August 20, 2013

Novartis announces on August 20, 2013, that the FDA grants the 18th Breakthrough Therapy (BT) Designation to the company’s non-orphan drug BMY338 (Bimagrumab), for the treatment of the muscle-wasting rare disease, sporadic  Inclusion Body Myositis (sIBM).  The BT Designation is based on the results of a Phase II proof-of-concept study that shows a substantial benefit for patients with sIBM compared to placebo. The results of the Phase II study are to be presented at the American Neurological Association (ANA), October 2013, New Orleans.

This is the 3rd Novartis investigational treatment in 2013 to receive a FDA BT Designation. Novartis receives the 8th BT Designation for LDK378 for ALK+ Non-Small Cell Lung Cancer (NSCLC) and receives the 16th BT Designation for Serelaxin for Acute Heart Failure (AHF).

sIBM is a rare life-threatening muscle-wasting condition. Patients gradually lose the ability to walk, experience falls and injuries, lose hand function, and have swallowing problems. There are no currently approved treatment options for sIBM.  Per the Novartis Press Release, Bimagrumab has the potential to met an unmet medical need and, :

“If approved, BYM338 has the potential to be the first treatment for sIBM patients”.

Bimagrumab is a novel, fully human monoclonal antibody, developed in collaboration with MorphoSys and is administered by intravenous infusion. Bimagrumab “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.”

 Bimagrumab is also in clinical development for the following indications:


2) Cancer Cachexia

3) Sarcopenia

4) Mechanically Ventilated Patients.

The Blog Post, “FDA Breakthrough Therapy Designation Chart”, will always have the latest FDA Breakthrough Therapy (BT) designations, so please bookmark using URL

The latest “FDA Breakthrough Therapy Designation Statistics Chart” Blog Post is located at URL, so please bookmark.

Please Note: FDA Official Logo from FDA website.

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