COVID VaccinesReturn to WorkMental Health

Drug Company Hits Pause on Launching an $89,000 Muscular Dystrophy Drug After Backlash

February 13, 2017, 10:29 PM UTC

Marathon Pharmaceuticals stared down some serious heat last week when it announced that it would sell a decades-old, cheap anti-inflammatory steroid for $89,000 to treat a heart wrenching rare muscle disorder in the U.S. The firm is having some second thoughts, pausing the drug’s market launch after massive public outrage and an investigation into the $88,000 price hike announced by Vermont Sen. Bernie Sanders and House Oversight Committee ranking Democrat Rep. Elijah Cummings on Monday.

The backstory: Illinois-based Marathon Pharmaceuticals won U.S. FDA approval of deflazacort, a common steroid that you can order for less than $1 per pill from abroad through online pharmacies, under the brand name Emflaza for a topline price of $89,000 for a year’s treatment. Why? The therapy wasn’t already approved in the U.S. as a steroid, and now it’s been cleared to help treat the symptoms of Duchenne muscular dystrophy (DMD) – a rare, muscle-wasting disease that leads to movement loss and eventual death in its young victims. Marathon had free reign over its pricing thanks to the lack of available Duchenne drug competitors.

The company took advantage of a number of U.S. programs meant to spur “orphan drugs,” or treatments for the kinds of disorders that afflict less than 200,000 Americans each year. For one, Emflaza’s approval to bolster muscle strength in DMD patients won the firm a coveted “priority review voucher” which it can sell for hundreds of millions of dollars to a pharma giant or use itself to shave four months off of the regulatory review period for another therapy. And Marathon barely did any actual clinical legwork to get the drug cleared for Duchenne – it relied on 1990-era clinical trial data before tacking on just enough study material to win an approval that doesn’t even address the root cause of the disease.

Click here to subscribe to Brainstorm Health Daily, our brand new newsletter about health innovations.

As I noted last week, this is an exact copy of Martin Shkreli’s modus operandi (Shkreli himself has now called out the similarities on his pharma critique/troll passion project Pharma Skeletons. “Bro. These guys invented price increases. I literally learned it from them,” he said of Marathon.)

The issue has inspired the wrath of patient advocates and lawmakers alike.

“Marathon did not develop deflazacort,” wrote Sanders and Cummings in a letter to Marathon CEO Jeffery Aronin, adding that they believe the firm is “abusing our nation’s ‘orphan drug’ program” and demanding answers for what they describe as an “outrageous plan” to gouge prices.

The firestorm appears to have hit home. Although Marathon has insisted that the $89,000 burden won’t be sloughed on to patients after discounts, rebates, and assistance programs, Aronin declared a temporary ceasefire on Monday.

“Since last week’s approval, we have heard both support from the community, and concerns about how the pricing and reimbursement details will affect individual patients and caregivers, such as how it effects coverage of other Duchenne products, such as EXONDYS 51,” wrote Aronin in a blog post for a Duchenne patient advocacy site. “Based on these questions, today we are announcing: We are pausing our launch which has not taken place yet… We have not sold any new product, and we will pause that process.”

The product that Aronin cites, Sarepta Therapeutics’ Exondys 51, is the only cleared treatment to treat the protein deficiency which causes Duchenne in the U.S., and has also enjoyed its fair share of controversy.

Just how long the “pause” will last is anyone’s guess. Pharma companies take pains to note that list prices are only topline numbers untethered from reality, and that they take pains to make sure vulnerable patients aren’t taking on those prices. For now, Marathon says that Duchenne patients and their families who buy deflazacort from overseas pharmacies at bargain prices may continue to do so.