Looks like the CEO of Marathon got some hate mail this weekend from many people, including Senators Bernie Sanders and Elijah Cummings
As we reported
last week, Marathon’s newly approved corticosteroid Emflaza (deflazacort) was going to cost $89,000 a year for patients with Duchenne muscular dystrophy. Given that families could purchase the drug overseas for a fraction of that price, the CEO, Jeffrey Aronin issued a statement
that they are pausing the launch of the drug (even though a launch date had never been set).
More specifically, the CEO stated:
We are pausing our launch which has not taken place yet. We have not sold any new product, and we will pause that process
We will maintain our Expanded Access Program, through which 800 patients currently receive Emflaza free of charge. If new patients seek to be included in our EAP, we will make that possible for free.
People currently receiving deflazacort from other sources will continue to have that option
We will meet with caregivers and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization based on an agreed plan of action
The communication went on to note that “Please know we sought FDA approval of Emflaza to improve access to this treatment. We have and always will support you in that endeavor. We hope this brings clarity and comfort to you and reassures you that your
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.
Corticosteroids are a staple for boys with Duchenne and until the approval of deflazacort, families would you use off label corticosteroids or buy deflazacort from overseas pharmacies. And until the approval of deflazacort, the price for it overseas was much lower ($1600 per year).