Emflaza Release Put on Hold as DMD Community Reacts to $89K Price Tag

Just days after Emflaza (deflazacort) became the first corticosteroid approved in the U.S. to treat Duchenne muscular dystrophy (DMD) regardless of mutation, the drug’s developer, Marathon Pharmaceuticals, announced it is “temporarily pausing” its sale in the U.S. to address concerns among the DMD community, particularly about the therapy’s high list price — $89,000 a year — insurance coverage, and other potential roadblocks to access.

“Since last week’s approval, we have heard both support from the community and concerns about how the pricing and reimbursement details will affect individual patients and caregivers,  such as how it affects coverage of other components of Duchenne treatment,” Jeff Aronin, chairman and CEO of Marathon, said in a press release posted on the CureDuchenne website.

“Based on these questions, today we are announcing” a pause in the treatment’s sale “to meet with Duchenne community leaders and … review their concerns, discuss all options, and move forward with commercialization based on the resulting plan of action,” Aronin said.

Without giving details, the letter states that Marathon officials expect that insurance coverage would  lower the drug’s cost for patients to “a standard co-pay of typically $20 or less per prescription.”

Those patients in the company’s pre-approval Expanded Access Program will also continue to receive the drug, and access to deflazacort from outside sources, like foreign countries, will continue to be allowed until a new marketing start date for Emflaza is set.

At a Feb. 10 informational webinar sponsored by Parent Project Muscular Dystrophy, Marathon executives said Emflaza was expected to enter the U.S. market in March, and deflazacort prescriptions from outside sources would no longer be honored after that time.

Deflazacort is reported to cost about $1,200 a year when ordered from overseas sources. It and other corticosteroids, which work by reducing inflammation and suppressing the immune system,  are widely used in many countries to slow the decline in muscle strength in boys with Duchenne MD.

Marathon justified the treatment’s high relative price tag in its open letter — and in the webinar — by noting the long years of preclinical and clinical testing that were necessarily conducted on the treatment to get U.S. Food and Drug Administration approval. It was not being used by about 90 percent of the DMD community due to the lack of regulatory approval, the company said, and only an estimated 7 percent to 9 percent of eligible patients were “importing it from abroad.”

“The resources we invested were substantial and we don’t expect to recoup our investment for several years and we have only 7 years of market exclusivity,” Aronin wrote.

The company said in its letter that it will continue to discuss  issues of pricing and accessibility with Parent Project MD and other community leaders “until there is a full understanding of how pricing decisions directly impact the revenues needed to fund past, current and future research as well as opportunities to purchase new therapeutic treatments.”

Emflaza’s price, officials said in both the webinar and letter, was a measure of the extensive cost of testing the drug for FDA approval to make it accessible to more patients, and was low relative to prices placed on other treatments for rare diseases such as Duchenne.

Support programs are in place to help patients and families with financial need, the company added, stating in the letter: “Anyone who needs this medicine will get this medicine.”