The U.S. Food and Drug Administration on Thursday approved a drug made by Marathon Pharmaceuticals to treat Duchenne muscular dystrophy (DMD), a devastating muscle-wasting disease that mainly affects young boys.
The drug, Emflaza, known generically as deflazacort, belongs to a class of anti-inflammatory drugs known as corticosteroids that are frequently used to treat DMD and other conditions.
Emflaza is the first steroid to win formal FDA approval to treat DMD. Prednisone, another steroid, is frequently prescribed for the disease though it does not have official FDA approval. Emflaza is thought to have fewer side effects than Prednisone.
Deflazacort has been available outside the United States for decades.
The big issue facing closely held Marathon will be how to price the drug. Marathon did not invent deflazacort and patients have been able to import it cheaply for personal use for years because there was not an approved U.S. treatment.
That will now change. Patients will no longer be allowed to import the drug now an FDA-approved product is available. Marathon, based in Northbrook, Illinois, will set its own price and does so at a time of great public and political hostility towards companies who repurpose old drugs and push up the price.
The company did not immediately respond to a request for comment.
Duchenne’s is a rare genetic disorder affecting some 15,000 U.S. patients that causes progressive muscle deterioration and kills most sufferers by the age of 30. Last year Dr. Janet Woodcock, head of the FDA’s pharmaceuticals division, ordered the approval of a Duchenne drug made by Sarepta Therapeutics Inc despite opposition from the agency’s top scientists and a negative recommendation from a panel of outside advisors.
The FDA approved Emflaza for all DMD patients five years and older. Sarepta’s drug treats a subset of DMD patients representing about 13 percent of the total.
Sarepta’s shares were little changed at $29.26 in afternoon trading on Nasdaq.