Orphan drug lands Novartis third breakthrough therapy designation

In Regulation & Government by Tim Peplow

novartis bimagrumab orphan drug

Novartis has been granted breakthrough therapy designation from the FDA for its orphan drug bimagrumab.

The designation is for the treatment of sporadic inclusion body myositis (sIBM), a rare muscle-wasting condition which causes progressive loss of mobility, loss of hand function and difficulty swallowing. sIBM is potentially life-threatening and currently has no approved treatment options – qualifying bimagrumab for one of the FDA’s new breakthrough therapy designations.

Bimagrumab, which was granted orphan drug designation for sIBM in US and Europe in 2012, is a fully human monoclonal antibody. Also known as BYM338, the drug was developed in collaboration with Morphosys and works by blocking the activity of myostatin and activin on type II activin receptors.

The latest designation gives Novartis a hat-trick of breakthrough therapy designations, with the company already having received FDA breakthrough therapy status for the drugs LDK378 for non-small-cell lung cancer and serelaxin for acute heart failure.

The new regulatory pathway could slash the time taken for a new drug to be approved. The FDA says the breakthrough therapy designation is “intended to expedite the development and review of drugs for serious or life-threatening conditions”. The process is moved on as quickly as possible, with communications with the FDA taking minutes instead of weeks or months. Read about the breakthrough therapy putting drugs in the fast lane to approval >

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Read the press release >