Duchenne Drug Launch Delayed Amid Price Outcry

Generic steroid has been around for decades

In an “open letter” posted on the patient-advocacy website Cure Duchenne, the CEO of Marathon Pharmaceuticals has announced that the company is “pausing” the launch of its Duchenne muscular dystrophy drug deflazacort (Emflaza) after U.S. lawmakers questioned why the medication is priced at $89,000 a year when patients have been able to import it for as little as $1,000.

Jeffrey Aronin acknowledged “concerns about how the pricing and reimbursement details will affect individual patients and caregivers.”

“I also want [the Duchenne community] to know that we hear and understand your concerns around the price and recognize it is tied to fears you have about reimbursement for other components of Duchenne treatment,” he wrote.

Aronin further stated that Marathon would maintain its “expanded access program,” under which 800 patients currently receive the drug at no cost. He said that patients currently importing the drug from overseas could continue to do so.

Aronin added: “The resources we invested were substantial and we don’t expect to recoup our investment for several years, and we have only seven years of market exclusivity. If we are profitable, we are committed to reinvesting our earnings from Emflaza into additional research into Duchenne. We are in regular contact with leaders of the Duchenne community about our plans and look forward to continuing that engagement.”

The deflazacort controversy is the latest in a series related to U.S. drug prices, including Mylan’ emergency EpiPen autoinjector, that have drawn the ire of both patients and lawmakers, according to a Reuters report.

Senator Bernie Sanders (I-Vermont) and Representative Elijah Cummings (D-Maryland) have called on Marathon to justify the price of the drug by documenting the cost involved in bringing it to market.

Marathon, after all, did not develop the product, which has been around for decades, the lawmakers wrote in a letter to Aronin. The company acquired rights to two clinical trials that had already been completed. It only conducted further toxicology and other tests to bring the product through the U.S. regulatory process.

“Marathon will have a monopoly on deflazacort for years to come, preventing less-expensive generic competitors from entering the market, despite the fact that this drug is already available in generic form in other countries,” Sanders and Cummings wrote.

The FDA approved deflazacort, a steroid, on February 9 under its orphan drug program, which provides incentives to drug makers to develop treatments for diseases with small patient populations. The incentives include seven years of market exclusivity.

Sources: CureDuchenne; February 13, 2017; and Reuters; February 13, 2017.