Marathon Pharmaceuticals' Duchenne muscular dystrophy drug wins FDA approval

The FDA has cleared a second drug for Duchenne muscular dystrophy, giving the green light to Marathon Pharma's corticosteroid Emflaza for patients aged five or more with the disease. Following rebates and other discounts, the price is $54,000 per year, said Marathon Pharmaceuticals CFO Babar Ghias.

The practice has prompted congressional investigations and hearings into companies including Valeant Pharmaceuticals International Inc. and Turing Pharmaceuticals LLC, the firm formerly run by onetime hedge-fund manager Martin Shkreli. The bad news is that it comes with a high price of $89,000 a year.

A recently FDA-approved drug used in steroid treatments, which has been on the worldwide market for years, will be introduced in the United States at the inflated price of $89,000 a year. 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.

The vouchers, in theory, exist for positive reasons as regulators try to encourage investment by companies in the development of drugs to use against rare diseases that are afflicting children.

For the last two decades, patients have been buying deflazacort from other countries ever since clinical trials demonstrated its effectiveness for treating Duchenne muscular dystrophy. It has been available for years in other countries, and the family of the individuals with Duchenne Muscular Dystrophy.

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Since so few Americans are affected by the disease, only around 15,000, the drug was approved under an "orphan drug" status, which is a special status given to drugs that treat rare diseases or conditions.

The drug wasn't sold in the USA mainly because no company thought it would be profitable enough to warrant the effort of seeking FDA approval.

At week 12, those on deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared with those on placebo. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated subjects. This is the 9th time that the FDA grants the pediatric disease priority voucher.

Common side effects include facial puffiness (Cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism), and excessive fat around the stomach (central obesity).

Other less-common AEs include problems with endocrine function, increased susceptibility to infection, hypertension, the risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decreased bone density, and vision problems, such as cataracts.

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