The US Food and Drug Administration (FDA) has approved the use of deflazacort, marketed under the brand name Emflaza, for the treatment of Duchenne muscular dystrophy (DMD) in children. The steroid drug is manufactured by Marathon Pharmaceuticals and is the first to be so approved by the FDA to treat people with the condition in the United States.
Muscle dystrophy is a muscle-wasting disease that affects boys aged three and above more than it affects girls. The FDA says it is a rare genetic disease that affects one in 3,600 male infants born worldwide, and it affects only about 15,000 boys in the US. It creates mobility problems in affected male children and confines them to a wheelchair until they pass on at around age 20. Patients also suffer from heart and breathing difficulties and the childhood disease is very life-threatening.
Emflaza will cost $89,000 for one year's treatment supply
Emflaza is produced by Northbrook, Illinois-based Marathon Pharmaceuticals and will cost $89,000 for one year's treatment supply. This is actually on the high side but six-year-old Marathon justifies this costly price with the argument that it took them six years to research, launch and get the neurological drug approved by the FDA; and that only a negligible percentage of the population requires it, while health insurance company will cover most of the costs. It will available for free through a Marathon program only for DMD patients without any insurance coverage.
The truth however remains that rare drugs for rare diseases are often costly, and these are often called "orphan drugs". According to Chicago Tribune, the top 100 orphan drugs in the United States go for an average cost of $111,820 annually per patient as at 2014. Even insured patients with high-deductible plans will still have to pay exorbitant amounts in out of pocket costs to acquire the drug.
FDA gave Emflaza a fast-track review and approval
In a FDA news release, DMD results when the body cannot produce dystrophin, a protein known to keep muscle cells intact and integrated. As the condition progresses, the muscles of the body starts to wear or waste away and affected children soon lose their ability to walk or feed themselves or carry out any other personal duties requiring muscle functions.
Emflaza was issued a priority review and a fast track designation to underscore the eagerness of the FDA to get a promising drug that could treat DMD in the US market. This is to encourage other pharmaceutical companies to fast-track the development of drugs for prevention and treatment of rare pediatric diseases.
"We hope that this treatment option will benefit many patients with DMD," said Dr. Billy Dunn, director of the Division of Neurology Products in FDA's Center for Drug Evaluation and Research. "This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy."