IBM: Novartis Drug Gets Breakthrough Therapy Designation from FDA

An experimental myostatin-blocking drug for sporadic inclusion-body myositis will get special attention from the U.S. Food and Drug Administration

by Quest Staff on August 26, 2013 - 3:45pm

Update (July 16, 2014): Some people have reported difficulty getting information about this trial when contacting the trial sites or Novartis. MDA is looking into these matters.

Update (Dec. 3, 2013): A 240-person trial of BYM338 is now open to men and women ages 36-85 who meet study criteria at several U.S. sites and at sites in Denmark and Italy. See Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients (RESILIENT); or enter NCT01925209 in the search box at

original story:

Multinational pharmaceutical company Novartis has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for BYM338, an experimental drug it is developing to treat sporadic inclusion-body myositis (IBM).

Sporadic IBM is IBM that occurs without a family history of the disease. Breakthrough therapy designation speeds up the FDA's review process for experimental compounds that appear to have benefit over available treatments.

The breakthrough designation is based on results from a phase 2 trial that compared BYM338 to a placebo in people with sporadic IBM. A larger trial is slated to open soon. (This trial has opened. See Dec. 3 update, above.)

BYM338 — also known as bimagrumab — is an antibody that blocks structures called activin receptors, thereby preventing myostatin and activin molecules — which limit muscle growth — from sticking to these receptors and sending signals to muscle fibers.

MDA has been supporting the strategy of “myostatin inhibition” as a way to compensate for muscle degeneration for a number of years. Current MDA grants to Tom Thompson at the University of Cincinnati and to Kathryn Wagner at the Kennedy Krieger Institute in Baltimore reflect continued commitment to this strategy, which has potential implications not only for IBM but for muscular dystrophies and other disorders involving muscle degeneration.

Read an excerpt from the Aug. 20, 2013, press release from Novartis below.

  • Designation highlights potential of BYM338 to address an unmet medical need in a serious disease.
  • If approved, BYM338 has the potential to be the first treatment for sIBM patients.
  • BYM338 is the third Novartis investigational treatment this year to receive a breakthrough therapy designation by the FDA, highlighting Novartis' leadership in the industry in breakthrough therapy designations.

Basel, August 20, 2013 Novartis announced today that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to BYM338 for sporadic inclusion-body myositis (sIBM).

Breakthrough therapy designation was created by the FDA to expedite the development and review of new drugs for serious or life-threatening conditions. This designation is based on the results of a phase 2 proof-of-concept study that showed BYM338 substantially benefited patients with sIBM compared to placebo. The results of this study will be presented at the American Neurological Association meeting on October 14 and is expected to be published in a major medical journal later this year.

sIBM is a rare yet potentially life-threatening muscle-wasting condition. Patients who have the disease can gradually lose the ability to walk, experience falls and injuries, lose hand function, and have swallowing difficulties[1]. There are no currently approved, (or established), treatment options for sIBM[2].

About BYM338 (bimagrumab) and the Novartis commitment to research in muscle therapeutics

BYM338 (bimagrumab) is a novel, fully human monoclonal antibody developed to treat pathological muscle loss and weakness. BYM338 was developed by the Novartis Institutes for Biomedical Research (NIBR), in collaboration with Morphosys, whose HuCAL library was used to identify the antibody. BYM338 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.

In addition to being developed for sIBM, BYM338 is in clinical development for chronic obstructive pulmonary disease (COPD), cancer cachexia, sarcopenia and in mechanically ventilated patients. BYM338 is administered by intravenous infusion.

Breakthrough therapy designation

According to the FDA, breakthrough therapy designation is intended to expedite the development and review of drugs that treat serious or life-threatening conditions. The designation requires preliminary clinical evidence that demonstrates substantial improvement over currently available therapy. The designation includes all of the fast track program features, as well as more intensive FDA interaction and guidance. The breakthrough therapy designation is a distinct status from both accelerated approval and priority review, which also can be granted to the same drug if relevant criteria are met.

About sporadic inclusion body myositis (sIBM)

sIBM is a rare disease, yet it is the most common degenerative disease of muscle in adults older than 65 years. It is characterized by a slowly progressive, asymmetric, atrophy and weakness of muscles. Commonly, patients become wheelchair bound within 10 to 15 years of onset. Death may occur due to injurious falls, infection (aspiration pneumonia) or malnutrition.

Bimagrumab also was granted orphan drug designation in sIBM in both the U.S. and Europe in 2012.

[1] Engel & Askanas, Inclusion-body myositis: Clinical, diagnostic, and pathologic aspects, Neurology 2006;66(Suppl 1):S20-S29
[2] Griggs, The current status of treatment for inclusion-body myositis, Neurology 2006;66(Suppl 1):S30-S32

Your rating: None Average: 4.8 (12 votes)
MDA cannot respond to questions asked in the comments field. For help with questions, contact your local MDA office or clinic or email See comment policy