Did Emflaza's DMD approval expose cracks at the FDA? Lawmakers want to know

Sen. Bernie Sanders and Rep. Elijah Cummings want to know whether the FDA relies on 20-year-old efficacy data in pharma's approval apps, as it appears to have done with Marathon's Duchenne muscular dystrophy drug Emflaza.

After lambasting Marathon Pharma for its pricing strategy on a decades-old med recently approved to treat Duchenne muscular dystrophy, Sen. Bernie Sanders and Rep. Elijah Cummings are turning their fire on the FDA.

The congressmen wrote FDA Acting Commissioner Stephen Ostroff to pose some “serious questions” about the agency’s review and approval for Emflaza. They asked whether the agency normally considers 20-year-old efficacy data—as they said the FDA did in this case—and whether any internal concerns about approving the medicine remained.

The Emflaza situation encapsulates several prominent issues in biopharma, including drugmakers' pricing power, high prices on older meds, and potential problems with the United States’ orphan drug laws. Citing possible “misuse” of the Orphan Drug Act of 1983, Sen. Chuck Grassley recently committed to looking into that topic. For about a year and a half, lawmakers have dug into controversial price hikes throughout the drug industry, investigating moves by Martin Shkreli’s Turing Pharma, Valeant Pharmaceuticals, Mylan and others.

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As Sanders and Cummings note in the new letter (PDF), Marathon received a priority review voucher—potentially worth hundreds of millions of dollars—because its drug notched an FDA approval in a rare pediatric disease. The FDA’s “mission includes advancing public health by making medical products ‘more effective, safer, and more affordable,’” the congressmen wrote. But it also has a responsibility to make sure companies aren’t “gaming the system.”

Worth noting is that Congress, not the FDA, sets laws pertaining to the agency. Still, the FDA writes the regulations implementing those laws and makes its own approval decisions. The agency also works with companies on clinical trial data requirements for drug approvals. It also granted a priority review in this case, a decision that rests at the agency.

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Emflaza won FDA approval last month and Marathon quickly touched off a firestorm by attaching an $89,000 price tag to the drug, first developed decades ago and now available in several countries for $1,000 per year.

As the controversy took off, Sanders and Cummings joined in with the company’s critics. A few days after the approval, they contacted Marathon CEO Jeffrey Aronin, and Marathon halted its launch hours later. Trade group PhRMA pledged to reconsider Marathon’s membership.

On Thursday, Marathon offloaded the scandal-ridden med to PTC Therapeutics for $140 million, plus future sales-based payments in a deal worth up to $190 million. The company also won a priority review voucher with the Emflaza approval, which it could sell to another drugmaker. Such vouchers can be worth $200 million-plus, based on previous sales.

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Marathon isn’t alone in attracting unwanted attention alone, though, as Turing CEO Martin Shkreli became a pricing pariah back in 2015 after hiking an old drug by 5,000%. Soon after, lawmakers began spotlighting high-profile increases on heart meds from Valeant and on Mylan’s EpiPen.

Congress hauled execs in for hearings, and federal authorities launched an investigation into Mylan for alleged Medicaid overpayments. Sanders and Cummings have also called for a probe into Novo Nordisk, Sanofi and Eli Lilly for rising insulin costs.