A Cheap Muscular Dystrophy Drug Will Cost U.S. Patients $89,000

The newly-FDA approved drug is available out of the country for far less

Photo Illustration: Diana Quach
Feb 10, 2017 at 4:43 PM ET

Until last year, there had been no approved drugs for Duchenne muscular dystrophy (DMD), a rare genetic condition passed down to men that gradually erodes sufferers’ ability to move and often kills them before the age of 30.

Last September, however, the Food and Drug Administration approved the drug eteplirsen for a subset of DMD patients. And this week, it approved the first steroid for DMD, a generic drug called deflazacort that’s long been imported into the country and used unofficially to slow down the disease. Unlike etelirsen, the drug will be approved for all DMD patients age 5 years and older.

But it seems the mark of approval won’t come cheap. Marathon Pharmaceutical, the Illinois-based company that shepherded the drug through its clinical trials, will sell their version, called Emflaza, for an estimated list price of $89,000 a year, a steep hike up from the brands of deflazacort available elsewhere in the world, some of which can run as low as $30 for a 100-pill bottle. It’s only the latest example to throw into sharp contrast the state of drug prices in America compared to many other countries.

The move, Marathon’s chief financial officer Babar Ghias told the Chicago Tribune Friday, is entirely pragmatic, intended to recoup the costs of a 6-year-long effort to bring the drug to the U.S. market. It also reflects the relatively small patient population it will serve — only an estimated one in every 7,250 men between the ages of 5 to 25 have DMD and a related form of muscular dystrophy, according to the CDC.

In an attempt to head off the sort of controversy that has followed other companies’ unforced price raises, the company has also promised that Emflaza will be made free for poorer patients through its assistance program, and it’s confident that insurance companies will cover the bulk of the drug’s costs. But given that the FDA approval will end the accepted practice of importing deflazacort into the U.S. for DMD treatment, it seems likely that at least some patients’ costs could increase.

The news of deflazacort’s approval, as well as its high sticker price, has parents of DMD children mostly excited. “I think the high cost is beyond ridiculous,” Terry Marlin, the father of two boys with DMD and co-founder of the non-profit charity Fight DMD, told Vocativ in an email. “But if it helps these boys, you can never put a price on [that].”

“Everything we deal with as parents and all the everyday struggles these boys have to go through are beyond what any child or parent should have to bear!!” he added.

Marlin, who has treated his children with imported deflazacort from the UK, is hopeful though that the FDA’s decision will spark future drug approvals.