Dr. Billy Dunn of the Division of Neurology Products with the Center for Drug Evaluation and Research at the Food and Drug Administration said the FDA is hopeful that Emflaza will help a wide range of patients with DMD.

DMD is the most well-known kind of muscular dystrophy that caused by an absence of dystrophin, a protein that keeps muscle cells in place. DMD is the most common type of muscular dystrophy. It can be administered to patients 5-years-old and above.

According to CDA News, people with DMD eventually lose ability to carry out activities on their own and are wheelchair bound by the time they become teenagers. The disease frequently happens in individuals without a known family history of the condition and essentially influences young men, yet rarely happen in girls. It affects one in every 3600 male kids worldwide. But until now it hasn't been widely available in the USA because it wasn't approved by the FDA. Marathon said that as of now just around seven to nine percent of those afflicted with Duchenne have access to deflazacort. Image credit: USA Today.$89,000 a year to treat the disease in the U.S.

Emflaza is marketed by Marathon Pharmaceuticals. That pharmacy recently informed customers that it would stop shipping the drug to the United States after Marathon received FDA approval for its version. The corticosteroid had not been sold in this country in part because no company thought it would be profitable enough to warrant the effort of seeking FDA clearance. Patients have been importing the drug since the 90s, but now they will have to pay 50 to 70 times what they used to pay if they want to get it in the U.S. Prednisone, another steroid, is frequently prescribed for the disease though it does not have official FDA approval.

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The company also invested $5.1 billion in capital expenditures in 2015, making it the U.S.'s largest technology investor. Intel spent $12.1 billion on R&D in 2015, making it the third largest R&D investor in the U.S.

However, some companies have used these incentives to their advantage and created business models based on procuring older drugs and substantially raising their prices. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began. The drug improves muscle strength but doesn't offer cure for the rare condition.

Clinical studies of the drug deflazacort showed improvement in muscle strength compare to those who took a placebo. By week 52, patients in the Emflaza group maintained an overall stability in average muscle strength.

Emflaza side effects are similar to other corticosteroids, such as puffiness of the face, weight gain, increased appetite, infection of the upper respiratory tract, fequent urination, stomach fat, cough, and unwanted hair growth. Other side effects include elevated blood pressure, mood changes, infection, and serious skin rashes.


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