On Thursday, the U.S. Food and Drug Administration (FDA) approved a drug produced by the Marathon pharmaceutical company. The drug treats a deadly form of Duchenne muscular dystrophy that has its onset in childhood. It is the second drug approved by the FDA that treats this disease and the first one that can be used by all who suffer from it.
Deflazacort, the drug, is a steroid that improves muscle function in patients with Duchenne muscular dystrophy, thus allowing them to control their wheelchairs better, feed themselves, or even attempt walking.
Duchenne muscular dystrophy affects about 15,000 boys in the U.S. It is a genetic disorder and, apart from movement problems, it causes heart and breathing problems. Those who suffer from the disease usually die in their twenties, although they can live to 30 with intensive care.
The approval of the drug might bring prices of around $89,000 for a year’s supply of the drug. However, Babar Ghias, the chief financial officer at Marathon, reassures the patients that they will not have to pay that much for the drug.
He expects that medical insurances will be able to cover the prices for the drug, which will go under the name Emflaza. Those who do not have a medical insurance will be able to get the drug for free through a special Marathon program.
He further explains why the drug is priced so high. The reasons include the fact that the drug targets a small population, the six years that they have spent trying to bring the drug on the market, and the research that is still needed for possible improvements on the drug.
However, Pat Furlong, the president and CEO of Parent Project Muscular Dystrophy, thinks that this price is much too high, since some people have high deductible insurance plans and they might not cover the expenses.
However, the fact that the FDA approved deflazacort is a big step ahead for the treatment of muscular dystrophy. This drug is already being used in other countries. The first drug approved by the FDA is eteplirsen, developed by Sarepta Therapeutics.
It gained approval last year in September but, unfortunately, is suitable only for 13 percent of those who suffer from Duchenne muscular dystrophy. It can be used only by those who have a specific genetic mutation.
Patients also use prednisone, another steroid, for treatment. However, this is not specifically approved for treatment of this disease. In the end, the medicine world makes visible progresses in the treatment of Duchenne muscular dystrophy.
Image Source: Wikimedia Commons