Idebenone Appears Promising as Treatment for Duchenne Muscular Dystrophy

Kara Elam avatar

by Kara Elam |

Share this article:

Share article via email
Duchenne muscular dystrophy

A group of international researchers has published a review of clinical trials for a drug that shows significant potential as a therapy for people who suffer from Duchenne muscular dystrophy (DMD). The study, published in the European Neurological Review, is titled “Idebenone as a Novel Therapeutic Approach for Duchenne Muscular Dystrophy.

Idebenone was originally developed to treat Alzheimer’s disease, but with limited success. Researchers discovered in the process that the drug  improves secondary mitochondrial dysfunction caused by dystrophin deficiency, intracellular calcium accumulation, and increased reactive oxygen species (ROS).

Santhera Pharmaceuticals, a Swiss-based company, has completed both its exploratory and confirmatory clinical trials to determine the clinical level of efficacy the drug presents to reduce respiratory function decline in DMD patients.

The review study was led by Gunnar Buyse, M.D., Ph.D., a professor of pediatrics and child neurology at the University of Leuven in Belgium and deputy clinical chair of child neurology at University Hospitals Leuven, and a well-renowned leader in the field of clinical trial drug development research for muscular dystrophy.

Buyse and his colleagues reviewed trial data from two clinical studies investigating the efficacy of idebenone to decrease respiratory function decline in an exploratory phase II (DELPHI) and confirmatory phase III (DELOS) trial.

Results showed that idebenone significantly reduced the loss of respiratory function in 8- to 18-year-old DMD patients who were not using concomitant glucocorticoids, indicating idebenone can be used as a tool to modify the course of respiratory disease progression in patients with DMD with the potential to significantly decrease both morbidity and mortality.

“Considering its favorable safety and tolerability profile, this oral medication could therefore become the first treatment option for patients not using glucocorticoids with the possibility to ameliorate a life-threatening complication in the aging population of DMD patients,” the authors wrote.