New Muscular Dystrophy Drug Gains FDA Approval

Just this week, the U.S. Food and Drug Administration (FDA) announced their approval of a new pharmaceutical drug that could significantly change the lives of those affected by Duchenne muscular dystrophy (DMD).

The new drug, named deflazacort, is a steroid-based medication that has been shown to improve muscle function in people with DMD. With consistent use, users may gain back some movement of their limbs, making it easier for them to perform day-to-day tasks. Some people may even regain the ability to walk.

Coming to the Market Soon

Deflazacort was created by Marathon Pharmaceutical and will be released on the market under the brand name Emflaza. The cost for a year’s supply has been set at $89,000, which seems like a high price (for many, it is), but it’s actually right around the average cost for a drug of it’s kind. The good news is that it will be covered by insurers. Users will only be our the cost of copays when receiving their dosage.

Why So Expensive

According to Sci-Tech Today, deflazacort fits into a special class of drugs known as orphan drugs, which are used to treat patients with rare neurological diseases. They are often priced higher than drugs that treat common ailments and diseases. The average cost of an orphan drug in the U.S. in 2014 was $111,820 per patient, making deflazacort on the lower end of the average price for a drug of its kind.

Already Seeing Success

Although the FDA has stopped it from being released in the U.S., deflazacort has already seen success in cases abroad. Until this past year, the FDA hasn’t approved any drugs that treat DMD, so this is a big step in the right direction.

DMD in the US

It’s estimated that 1 in every 7,250 males aged 5 – 24 years in the US suffer from Duchenne and Becker muscular dystrophy, according to the Center for Disease Control. People who are affected often lose muscle control and often use wheelchairs and wheelchair accessible vans to get around.

« Back to Blog