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Corticosteroid Drug approved by FDA to Treat Duchenne Muscular Dystrophy

March 10, 2017


The United States Food and Drug Administration has given approval FDAto Emflaza (deflazacort) tablets along with oral suspensions for the treatment of patients of the age 5 and above who suffer from a rare muscular genetic disorder called Duchenne muscular dystrophy (DMD) which causes continuous muscle deterioration and weakness. Emflaza is a corticosteroid which functions by reducing the amount of inflammation and decreasing the immune systemactivity.

Across the world many corticosteroids are commonly used for the treatment of DMD. This is the first ever corticosteroid which has been approved by FDA to treat DMD and the first approved deflazacort to be used in the United States.

The Director of the Division of Neurology Products in the FDA’s Centre for Drug Evaluation and Research, Billy Dunn, M.D., 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."

One of the most common type of muscular dystrophy is DMD and it happens because of the absence of dystrophin, which is a protein that assists the muscles in keeping intact. The initial symptoms are seen in the first 3 to 5 years of age which worsens over time. DMD mostly affects individuals with no hereditary condition and mainly affects the boys. It occurs in about one of every 3,600 male infants across the world.

People who have DMD eventually lose their ability to execute activities independently and most people need wheelchair even before they hit their teens. When the disease deteriorates, many respiratory and life-threatening conditions begin. Most patients are seen succumbing to DMD in their 20s or 30s; nevertheless, severity and life expectancy of the disease varies.

A clinical study done on 196 male (5 to 15 years old) patients established the efficiency of deflazacort at the initial stages of the clinical trials with familiar mutation of dystrophin gene and beginning of weakness before 5 years of age. By the 12th week, these patients who were consuming deflazacort had shown betterments in a clinical classification of muscle strength across several muscles as compared to those taking a placebo. At the end of 52nd week, there was a considerable proven stability in the muscle strength in the patients taking deflazacort.

Side effects that tag along with any corticosteroid drugs are also a part of Emflaza. Some of the common ones being weight gain, facial puffiness, cough, upper respiratory tract infection, excessive hair growth, excess urination during daytime, and excess fat around the stomach area.

Besides, there are a few less common side effects which include health issues such as escalated susceptibility to infection, endocrine function problems, risk of gastrointestinal perforation, high blood pressure, extreme mood fluctuations, skin rashes, reduced bone density, and cataracts. Also, patients who are acquiring immunosuppressive dosages of corticosteroids must not be given any live attenuated vaccines.

The FDA approved this particular application on priority and the drug also received an orphan drug destination, which renders inducements to help and motivate the development of drugs for any kind of rare diseases.

 

The sponsor is incurring a paediatric disease which is rare in natureFDA priority review voucher under a program which is meant to promote novel drugs and biologics for preventing and treating of rare paediatric diseases. Ever since the program started, this is the ninth rare paediatric disease priority review voucher which has been established by the FDA.

Emflaza is marketed by Marathon Pharmaceuticals of Northbrook, Illinois.