The U.S. Food and Drug Administration is now approved Emflaza (deflazacort), an old steroid treatment, long accessible outside the United States.
A drug to treat muscular dystrophy will hit the US market with a price tag of $89,000 a year despite being available for decades in Europe at a fraction of that cost. Now, they will be able to get Emflaza tablets and oral suspensions to treat DMD.
Northbrook, Illinois-based Marathon Pharmaceuticals announced on Thursday that it had snagged an FDA green light for corticosteroid Emflaza, known generically as deflazacort. He says the net price will be closer to $54,000 per year once rebates and discounts kick in.
Four-year-old boy with DMD.
Duchenne muscular dystrophy, a rare form of the disease that affects young boys, can be treated with a drug named deflazacort. The disorder is caused when there is an absence of the protein dystrophin, which helps to keep the muscles intact, according to the FDA. The primary symptoms are generally observed in between 3 and 5 years of age and worsen after some time. Individuals with this condition lose little by little their ability to perform activities on their own, and they usually require the use of a wheelchair before their 20s. As the disease progresses, life-threatening heart and respiratory conditions can occur. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. But until now it hasn't been widely available in the US because it wasn't approved by the FDA.
Big-club target Romelu Lukaku focussed on getting Everton into Europe
We did it for 45 minutes today and then in the last 15 minutes as well, but we did not show it for most of the second half. The buzz notched up as well it might given that it is their Stanley Park neighbours who sit those six points ahead.
DMD typically affects boys; however, girls can be mildly affected and be carriers of the gene, according to the Muscular Dystrophy Association.
"Now the hope is at least all children will have access to an actual FDA-approved therapy for Duchenne", said Craig McDonalds, a professor at the University of California. Its content is produced independently of USA TODAY.
Marathon anticipates a smooth transition for patients ready getting deflazacort from overseas pharmacies. The drug was never sold in the USA because companies didn't think it would be profitable enough, The Wall Street Journal reported, but some families have obtained the drug from online pharmacies overseas to help improve muscle strength for boys at least for a while. Competitor Sarepta Therapeutics has said its disease-modifying DMD drug, Exondys 51, will cost about $300,000 annually for the average patient in the U.S.
The new, American, list price for this drug is $89,000 a year. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began. In general, the muscle strength was maintained among those taking the drug until the end of the 52-week study.
The most common adverse effects with Emflaza included Cushingoid appearance, weight gain, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, and central obesity. Other side effects include elevated blood pressure, mood changes, infection, and serious skin rashes. It can also lead to a reduction in the density of the bones.