A Cheap Muscular Dystrophy Drug Will Cost US Patients $89000

In a Thursday press release, the Food and Drug Administration (FDA) granted approval for a drug by Marathon Pharmaceuticals, a United States company focused exclusively on the development of new treatments for rare diseases.

The efficacy of Emflaza was demonstrated in a clinical study (n=196) of male patients aged 5-15 years at the start of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5.

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. In Canada and other countries around the world, deflazacort has been used for years to treat DMD and the pills or oral suspensions range from about $1,000 to $2,000 a year for patients. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated subjects.

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).

Less common side effects include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk for gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones, and vision problems.

Patients who get immunosuppressive doses of corticosteroids should not be given live or live attenuated vaccines, the FDA warned.

FDA officials today announced the approval of Emflaza (deflazacort) tablets and oral suspension for the treatment of Duchenne muscular dystrophy (DMD). Unlike etelirsen, the drug will be approved for all DMD patients age 5 years and older.

"We now have a treatment option for kids and adults with Duchenne, which is a major advance for the community", said MDA chief medical and scientific officer Valerie Cwik. 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.

A voucher known as a priority review was given to the company, which essentially is equal to a ticket that can be used to get a drug in the future reviewed faster by regulators, or can be sold for hundreds of millions of dollars to another pharmaceutical company.

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