The corticosteroid Emflaza (deflazacort) is now approved by the U.S. Food and Drug Administration as treatment for Duchenne muscular dystrophy in patients aged 5 years old and above.
Corticosteroids are a common treatment option for DMD around the world but this is the first time that the FDA has approved any drug in the class to treat the condition and also the first time that Emflaza was approved for any kind of use. The third major proof point in the Muscular Dystrophy Association's research program, Emflaza joins two other drugs approved for treating muscular dystrophy: Exondys 51 and Spinraza.
"This approval follows decades of MDA researcher ... that was made possible by the hard work, dedication, and unwavering commitment of our donors and sponsors," said Steven M. Derks, MDA CEO and president.
"We hope that this treatment option will benefit many patients with DMD," said Dr. Billy Dunn, Division of Neurology director at the FDA Center for Drug Evaluation and Research.
The FDA also granted Emflaza priority review and fast track and orphan drug designations. The orphan drug designation comes with incentives to encourage and assist in drug development for rare diseases.
Marketed by Illinois' Marathon Pharmaceuticals, Emflaza is available as tablets and oral suspension. Both forms were approved by the FDA.
How Emflaza Works
As an immunosuppressant and anti-inflammatory, Emflaza was shown to slow loss of muscle function and strength, maintain respiratory and cardiac function, and reduce incidence of scoliosis in DMD patients. More importantly, the drug results in less severe side effects typically experienced when using corticosteroids, scuh as bone mass loss, weight gain, behavioral issues, and glucose intolerance.
The drug's effectiveness was observed in a clinical study involving 196 male patients between the ages of 5 and 15. All of the study participants had documented dystrophin gene mutation and had experience muscular weakness before 5 years old. By week 12, those given deflazacort had improved strength across various muscles and overall muscle strength ability was maintained until the study ended at week 52.
Similar results were observed in another trial that lasted 104 weeks with 29 male patients but this experiment also showed that participants on deflazacort seemed to lose walking ability later than those given placebo.
Duchenne Muscular Dystrophy
The most common of muscular dystrophies, DMD occurs as the result of the lack of dystrophin, a protein that aids in keeping muscle cells together. Symptoms first appear typically at 3 to 5 years old, worsening over time. DMD commonly manifests in individuals without known family histories of the condition and mainly affects boys. It is said that the condition occurs in about one in every 3,600 male infants across the globe.
People diagnosed with DMD experience progressive loss of ability to do activities independently, often requiring wheelchair use by the time they hit their early teen years. As the condition progresses, chances of life-threatening conditions developing involving the respiratory system and the heart grow. Life expectancy and disease severity vary but many patients usually succumb to DMD in their 20s or 30s.