FDA Approves Deflazacort Drug to Treat Duchenne Muscular Dystrophy

Highlights

• Duchenne Muscular Dystrophy is a disorder which causes progressive muscular weakness.
• Deflazacort (emflaza) drug is approved by the FDA for the treatment of Duchenne muscular dystrophy.
• The drug was found to show improved muscle strength in patients when compared to placebo.

The United States Food and Drug Administration (USFDA) has recently approved deflazacort (Emflaza) drug to treat Duchenne muscular dystrophy for patients above the age of 5 or older. Duchenne Muscular Dystrophy is a rare genetic disorder which causes progressive muscle weakness. One out of every 3600 male infants in the world is affected with Duchenne Muscular Dystrophy.

Corticosteroids are commonly used drugs for the treatment of Duchenne Muscular Dystrophy. Deflazacort is the first approved corticosteroid drug for Duchenne Muscular Dystrophy by the FDA.

The drug has also received an Orphan Drug Designation that provides incentives to encourage and develop drugs for rare diseases.

Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said, “This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy.”

“We hope that this treatment option will benefit many patients with DMD.”

Clinical Study
The efficacy of the drug was tested by conducting a clinical study of 196 male patients between the age of 5 and 15 years. These patients had a mutation in the dystrophin gene and had progressive muscle weakness before the age of 5.

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Patients who took deflazacort drug had improvements in muscle strength when compared to patients who took a placebo.

The stability in the average muscle strength was maintained throughout the end of 52 weeks in deflazacort treated patients.

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Another clinical trial that lasted for 104 weeks with 29 male patients, demonstrated an advantage on an assessment of average muscle strength when compared to placebo.

However, deflazacort patients appeared to lose their ability to walk when compared to those treated with placebo.

Common Side Effects of Deflazacort Drug
The side effects of deflazacort drug are similar to other corticosteroids that are being used for the treatment.

• Facial puffiness
• Weight gain
• Increased appetite
• Upper respiratory tract infections
• Cough
• Unwanted hair growth
• Excessive fat around the stomach
• Extraordinary daytime urinary frequency

Less common side effects of the drug may also include problems with endocrine function, increased risk of infection, elevated blood pressure, skin rashes, changes in the mood and behavior, decrease in bone density, vision problems such as cataract.

Duchenne Muscular Dystrophy
It is a most common type of muscular dystrophy which is caused by the absence of dystrophin protein that keeps the muscle cells intact. Symptoms of the disorder are usually seen between the age of 3 and 5.

People usually require a wheelchair during their teenage when affected with Duchenne Muscular Dystrophy.

Progression of the Duchenne Muscular Dystrophy may cause life-threatening heart and respiratory conditions. The patients were found to usually die between the age of 20 and 30.

References

1. FDA approves drug to treat Duchenne muscular dystrophy - (http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm540945.htm)

Source-Medindia