- The Food and Drug Administration approved a corticosteroid from Marathon Pharmaceuticals for treatment of Duchenne muscular dystrophy (DMD), the agency said Feb. 9.
- The regulator gave a green light to Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with the rare genetic disorder, which causes progressive muscle deterioration and weakness in children, almost always boys.
- Deflazacort has long been used off-label for DMD treatment, but until now hadn't been approved to treat the condition. The drug is expected to cost $89,000 a year. Competitor Sarepta Therapeutics has said its disease-modifying DMD drug, Exondys 51, will cost about $300,000 annually for the average patient in the U.S.
While corticosteroids are the standard treatment commonly used to treat DMD, this is "the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use" in the U.S., the agency said. The agency had granted fast track designation and priority review to Marathon's application.
Marathon said Emflaza is the first widely applicable DMD treatment, since it is indicated for all genetic forms of the condition. Sarepta’s drug works for about 13% of the DMD population with a specific genetic mutation.
Marathon said it was committed to "acceptable access" to Emflaza for DMD, and is pricing the drug at $89,000 per year based on its usefulness in a rare disease and the trials that Marathon had to conduct to get the drug approved for the indication.
The drug, which is available for $1 a pill in Canada, is already attracting backlash over its price tag. The price hike of the already-available, low-cost generic is just fuel on the drug pricing fire that has been burning for the last few years. Martin Shkreli's Turing Pharmaceuticals has been the subject of ire in this debate after a similar situation where the company hiked the price of a decades-old AIDS drug.
Yet, Marathon has insisted that patients will not see the cost. The company has patient financial-support services and expects that U.S. patients will have low or limited out-of-pocket costs. For those patients that don't have insurance, the company has assistance programs in place.
Marathon anticipates a smooth transition for patients already getting deflazacort from overseas pharmacies. Marathon, which said it's fully committed to working with payers and providers to establish coverage, "does not expect widespread insurance company requirements to use another generic corticosteroid that is not approved by the FDA for Duchenne prior to using Emflaza for Duchenne, especially for those currently on a version of deflazacort."