The U.S. Food and Drug Administration on Thursday approved a Northbrook pharmaceutical company's drug to treat a deadly form of childhood-onset muscular dystrophy -- only the second FDA-approved drug for the disease and the first for everyone with the disorder.
Marathon Pharmaceutical's deflazacort, a steroid, aims to improve the lives of patients with Duchenne muscular dystrophy by improving muscle function, potentially allowing them to walk, feed themselves and control their wheelchairs longer.
The approval of the drug, which will carry a list price of $89,000 for a year's supply, comes as pharmaceutical companies face growing pressure to hold down medication prices.
But Babar Ghias, Marathon chief financial officer, said patients will pay far less than that for the medication, which will go by the brand name Emflaza. Ghias expects the drug will be covered by insurers, so patients only have to cover copays. Patients without insurance can get the drug for free through a Marathon program, he said.
He said Marathon is pricing the drug at $89,000 because of its usefulness for a small population, because of the six years Marathon worked to bring the drug to market and to perform further research on it. Marathon is a 6-year-old company that focuses on rare neurological diseases.
Prices for medications such as deflazacort, which are used to treat rare diseases and called orphan drugs, are often much higher than drugs for more common ailments. The top 100 orphan drugs in the U.S. cost an average of $111,820 a year per patient in 2014, according to a report by Evaluate, a market research firm.
Pat Furlong, president and CEO of Parent Project Muscular Dystrophy, called $89,000 "expensive," saying questions remain about how much patients actually will have to pay out of pocket for the drug, especially among those with high-deductible insurance plans.
"That's a significant cost and patients should not have to bear that cost," Furlong said.
Still, she called the FDA's approval of the drug an important step forward for those with the disease. Parent Project Muscular Dystrophy has received money from Marathon to support its conferences.
The muscle-wasting disease, a genetic disorder, primarily affects boys and can cause movement, heart and breathing problems. Those with the disease typically lose the ability to walk, and die in their 20s, though they can live until their 30s with certain types of care.
Deflazacort already is used to treat Duchenne outside the U.S., where it's approved for other uses. But until now it hasn't been widely available in the U.S. because it wasn't approved by the FDA. Duchenne affects about 15,000 boys in the U.S.
Kerry McNicholas, whose 12-year-old son Liam has the disorder, hopes the drug helps more patients stay active longer. The Lindenhurst mother wonders whether Liam, who's now in a wheelchair, would have walked longer if he had access to deflazacort earlier. Liam lost the ability to walk at 10. He started taking deflazacort in recent years, first as part of a study and then through a Marathon program, she said.
"He stopped walking fairly early, and I kind of attribute that to him not taking deflazacort as his main steroid when he was young," McNicholas said.
She said such drugs allow kids like Liam -- an animal-loving, Nintendo-playing seventh-grader -- to live their lives to the fullest.
"It's really important to the families of kids with Duchenne to get drugs approved," McNicholas said.
Until last year, no drugs had been approved by the FDA to treat Duchenne.
The first drug, eteplirsen, developed by Sarepta Therapeutics, gained FDA approval in September. That drug, however, only helps about 13 percent of Duchenne patients, those with a specific genetic mutation, said Tim Cunniff, Marathon executive vice president for research and development.
Many patients also have been using a different steroid called prednisone, though prednisone is not specifically approved for treating the disease.
Insurers will pay varying amounts for deflazacort depending on individual negotiations, Ghias said. The price also will remain the same regardless of the dose needed by a patient, he said.
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