FDA approves $89000 drug to treat Duchenne's Muscular dystrophy

Corticosteroids are a staple for boys with Duchenne and until the approval of deflazacort, families would you use off label corticosteroids or buy deflazacort from overseas pharmacies.

"As a physician, I participated in a clinical trial of Emflaza and have a keen awareness about what the approval of this drug means for patients and families".

Privately held Marathon Pharmaceuticals LLC appears totally unconcerned about the potential negative publicity surrounding its planned $89K price tag for deflazacort, a decades-old steroid drug recently OK'd by the U.S. FDA for the treatment of Duchenne muscular dystrophy (DMD).

Until a year ago, there had been no approved drugs for Duchenne muscular dystrophy (DMD), a rare genetic condition passed down to men that gradually erodes sufferers' ability to move and often kills them before the age of 30. Emflaza is thought to have fewer side effects than Prednisone. While CEO of KaloBios, Shkreli had told investors that he planned to price a drug called benznidazole that's used to treat the neglected parasitic infection Chagas disease somewhere in the $60,000-to-$100,000 per-treatment range.

Complicating the picture is that deflazacort has been available outside the United States for decades and patients have been able to import it for personal use. The drug, which Ghias tells the Journal will be sold under the brand Emflaza starting in March, is a steroid that helps mitigate muscle weakness and cut inflammation, per an FDA release.

Common side effects of the drug are similar to those of other corticosteroids and include: facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, haveig to urinate often during the daytime, unwanted hair growth, and excessive fat around the stomach, according to the FDA. Last year Dr. Janet Woodcock, head of the FDA's pharmaceuticals division, ordered the approval of a Duchenne drug made by Sarepta Therapeutic despite opposition from the agency's top scientists and a negative recommendation from a panel of outside advisors. The drug is indicated to treat patients aged 5 years and older with the rare genetic disorder. With increasing number of DMD patients expected to go for a treatment, drug sales for 2017 were estimated at $138 million.



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