Less than one month after our exploration of the recent colossal price markup of a simple drug used by a vulnerable and desperate group of children and their families, a dramatic turn of events occurred which may make things better or worse. In February, the pharmaceutical company Marathon announced its marketing plans for Emflaza, its brand name for deflazacort, a simple corticosteroid widely available in other countries but not in the United States. Emflaza had recently been approved by the FDA for the treatment of symptoms of Duchenne muscular dystrophy (Duchenne MD) in the United States. Duchenne MD is a usually fatal genetic muscular disorder of young boys for which no curative therapy is currently available, but for which corticosteroids such as deflazacort and prednisone slow the progression of weakness. Deflazacort– the most commonly prescribed all-purpose steroid in some countries– has been used in Canada and elsewhere for the treatment of Duchenne MD for some time. The wrinkle in Marathon’s release was that it declared an annual charge of $89,000 per child for a drug sold in many other countries for a price a measured in pennies instead of dollars.
I have already expressed my puzzlement that the FDA gave its approval to Marathon based on clinical data collected many years old by another drug company that for undisclosed reasons walked away from FDA approval. I am still looking for reliable studies that confirm that deflazacort is superior to, or safer than prednisone to treat Duchenne MD. [Prednisone is the most prescribed corticosteroid in the United States. To say that it is inexpensive would be an exaggeration!] Perhaps a clinician without ties to the pharmaceutical industry will provide us with evidence-based data that Emflaza is an essential or even a better drug for Duchenne MD, or worth the monumental cost for any incremental benefit.