FDA Grants Muscle-Wasting Disease Drug Breakthrough Status

(BLS.gov)

The global pharmaceutical company Novartis says the Food and Drug Administration granted its therapy candidate bimagrumab breakthrough status to treat a rare muscle-wasting condition for which there are currently no approved treatments. FDA designates new drugs for serious or life-threatening conditions as breakthroughs to expedite their review in the agency.

The disorder is known as sporadic inclusion body myositis or sIBM, an inflammatory muscle condition, characterized by the progressive weakness and wasting of the muscles. The condition usually affects the limbs, with symptoms appearing after age 50, causing the individual to become more disabled as the disease progresses, and often spreading to muscles used for speech and swallowing. According to Muscular Dystrophy Association, sIBM occurs in an estimated 1 in 200,000 individuals, affecting males 2 to 3 times as often as females.

Bimagrumab, code-named BYM338, is a monoclonal antibody developed to treat pathological muscle loss and weakness. BYM338, given by intravenous infusion, binds to protein receptors affecting muscle growth and prevents signals from proteins that retard muscle growth from binding to these receptors. Novartis says it is developing BYM338 as a treatment for two other muscle-wasting disorders, cancer cachexia and sarcopenia, as well as chronic obstructive pulmonary disease (COPD).

Novartis collaborated with the biotechnology company Morphosys to identify bimagrumab, using Morphosys’s Human Combinatorial Antibody Library or HuCAL technology. HuCAL antibodies, says Morphosys, are produced in bacteria, making it possible to isolate antibodies in the lab with the desired characteristics.

Novartis says FDA’s breakthrough designation is based on the results of a proof-of-concept clinical trial with a small number of sIBM patients that tested BYM338 against placebo. The results, says Novartis, show patients given BYM338 benefit more than the placebo patients. The company plans to present the findings in October at a meeting of the American Neurological Association.

FDA’s breakthrough therapy designation provides expedited review by the agency, including more frequent meetings and correspondence, as well as more guidance for developing the drug or biologic, and greater senior-level involvement in the review process.

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Hat tip: FirstWord Pharma

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