Today, the FDA approved the use of EMFLAZA (deflazacort) for Duchenne patients over the age of five years old. It is routine for the FDA to designate specific patient populations on the “label” that are approved to receive a drug. The reason the FDA excluded younger patients for EMFLAZA is because there has not been a clinical trial for deflazacort which has included patients younger than five years old.
Having said that, the decision to prescribe a corticosteroid to a patient under the age of five is made at the discretion of the individual prescribing physician in consultation with the patient’s caregivers. If your health care provider already has prescribed deflazacort for treatment of Duchenne to a patient under the age of five, we don’t anticipate that the label will change that decision. For patients under age five, especially those already on deflazacort, we anticipate that insurers would provide coverage for EMFLAZA, although a physician may be required to document the medical need for the insurer. If for some reason your insurance company will not cover EMFLAZA, Marathon will work with the patients and caregivers on a case by case basis.
It’s worth noting that Marathon will be conducting a clinical trial in patients less than age five in an effort to expand the label to include younger patients. While that study is under way, Marathon will offer study participants access to EMFLAZA for free. When the trial is over and the FDA is evaluating the data, Marathon will also offer expanded access to non-study participants upon agreement with FDA as they did over the past 18 months, while the company was working on the New Drug Applications for EMFLAZA and awaiting the agency’s review.