The launch of Marathon Pharmaceuticals’ steroid drug, Emflaza (deflazacort), has been delayed following outrage over the drug’s price. On February 9, 2017, the FDA announced the approval of the new drug to treat a rare genetic disorder, Duchenne muscular dystrophy (DMD), which causes progressive muscle deterioration and weakness. Marathon set the price for the drug at $89,000 for a year’s treatment. Patients and lawmakers expressed indignation, noting that such a price is unfair, given the fact that U.S. patients have, for many years, imported deflazacort, the generic version of Emflaza, at the cost of about $1,200 a year. Sen. Bernie Sanders (I-Vt) and Rep. Elijah Cummings (D-Md) sent a letter to Marathon, calling the pricing “unconscionable” and asking for answers regarding the company’s “opportunistic pricing behavior.”
Approval
The drug, Emflaza is a corticosteroid. Corticosteroids function by reducing inflammation and decreasing immune system response. Corticosteroids are commonly used around the world as a treatment for DMD, however, the FDA approval of Emflaza represents the first approval of a corticosteroid for that purpose.
DMD
DMD is caused by an absence of a protein that helps keep muscle cells intact. Early symptoms are usually seen between three and five years of age and the disease predominantly affects males. Specifically, DMD occurs in about one of every 3,600 male infants worldwide. Individuals with DMD progressively lose the ability to perform activities, require use of a wheelchair by their early teens, and have a limited life expectancy of around 20 to 30 years.
Effectiveness
The drug was tested in a clinical study of 196 male patients who were five to 15 years old at the beginning of the trial. All of the patients had documented mutation of the dystrophin gene and weakness before age five. Patients taking Emflaza demonstrated increased muscle strength and possibly lost the ability to walk later than patients taking the placebo.
Orphan drug
Emflaza is designated as an orphan drug, which means it is intended to treat a rare disorder. As part of that designation, Marathon is granted seven years of marketing exclusivity for thedrug, an incentive designed to encourage pharmaceutical companies to develop new treatments for rare diseases. The Sanders/Cummings letter accused Marathon of taking advantage of the orphan drug designation, which the lawmakers said, is designed “encourage research into new treatments for rare diseases—not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades.”
Other benefits
Marathon is also receiving a rare pediatric disease priority review voucher, which is part of another program designed to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. The voucher can be redeemed by Marathon to receive priority review of a subsequent marketing application for a different product. The FDA has granted only nine pediatric disease priority review voucher to date.