The only drugs currently recommended for people with Duchenne muscular dystrophy (DMD) are glucocorticoid corticosteroids, but many patients still do not receive such treatment. A review in the journal US Neurology underscores the benefits of deflazacort, a glucocorticoid used as an anti-inflammatory and immunosuppressant, demonstrating that the drug preserves function and prolongs survival in DMD.
Guidelines for the treatment of DMD have existed since 2010, but surveys show that adherence to the recommendations varies between treatment centers and countries, particularly when it comes to corticosteroid treatment.
The review, “Deflazacort in Duchenne Muscular Dystrophy,” summarized studies comparing the drug to no treatment or to corticosteroid prednisone, the two most commonly used DMD treatments in the U.S., although neither are approved by the U.S. Food and Drug Administration (FDA). Deflazacort is currently available through Marathon Pharmaceuticals, the Illinois-based company behind the review, under the FDA’s open-label, expanded access program. The drug’s consideration by the agency as a DMD treatment is expected to begin soon.
Numerous studies show deflazacort preserves muscle function and strength while postponing loss of ambulation, sometimes by several years, the review reported. While most DMD-affected boys stop walking before the age of 12 without treatment, it said, 70 percent to 80 percent of those receiving the drug were still able to walk at that age
Moreover, the drug reduced rates of scoliosis development, a condition requiring surgery and associated with functional decline. Some studies also indicated that deflazacort treatment preserved heart and lung function. The prevention of heart disease is likely one of the main factors prolonging survival, as some studies also found better survival rates in treated groups.
Deflazacort seems to be as efficient in preventing decline as the steroid prednisone, as direct comparisons exploring muscle strength and physical function generally detected few differences between the drugs. Nevertheless, long-term glucocorticoid treatment is tied to an array of side effects, although some studies have suggested that deflazacort might lead to less weight gain than prednisone.