On February 9, the FDA approved Marathon Pharmaceuticals’ Emflaza (deflazacort) for Duchenne muscular dystrophy (DMD) patients ages five years and older.

However, Marathon has received a lot of criticism over the last couple of weeks for planning to charge DMD patients $89,000 a year for Emflaza. As a result, on February 13, the company announced that they are halting the launch of their drug until further notice.

Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.

The FDA approval of Emflaza marks the first corticosteroid treatment approved for DMD in the US, and the first deflazacort treatment for any use in the US.

According to US Key Opinion Leaders (KOLs) interviewed by GlobalData, corticosteroids have been imported from other countries and prescribed off-label for DMD patients for many decades due to their ability to prolong independent ambulation and lessen the likelihood of surgically mandatory progressive scoliosis.

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Multiple branded and generic deflazacort treatments for DMD have already been approved in the EU, where they were first launched in 2001. According to GlobalData, the annual cost of therapy for corticosteroids indicated for DMD in the EU range from $300 to $1100 per year.

DMD is a fatal, recessive, X-linked genetic disorder predominately affecting males. It is caused by mutations in the dystrophin gene, the largest gene in the human genome. DMD manifests in delayed motor function development and severe muscle weakness, resulting in wheelchair use by many DMD patients. Without ventilator intervention and steroid therapy, death occurs by 20–25 years of age.

Although corticosteroids are known to be beneficial to DMD patients and there have been numerous studies that report an increase in muscle strength and a prolongation in the ability to walk associated with their use, their exact mechanism in DMD is unknown.

The approval of Emflaza was supposed to make it easier for DMD patients to access deflazacort in the US. Patients who already accessed this drug from other countries will most likely continue to import them. 

Emflaza was granted FDA orphan designation for treating DMD on August 16, 2013. However, Vermont US Senator Bernie Sanders, in a February 13th letter to the CEO of Marathon Pharmaceuticals, stressed that Marathon is taking advantage of the FDA orphan drug program, which was intended to foster research for rare disease treatments, not to provide pharmaceutical companies with lucrative market exclusivity rights for drugs that have been available worldwide for years.

In their February 13th open letter to the DMD community, Marathon indicated that the new price “should not be a barrier” for patients and that they plan on investing their earnings from Emflaza into developing and funding additional therapies.