- Congressional scrutiny of Marathon Pharmaceuticals continued to grow last week despite the company's decision to pause commercialization of its controversial new treatment for Duchenne muscular dystrophy (DMD).
- A group of senators penned a letter to Marathon Pharmaceuticals demanding answers on how much Marathon paid to develop Emflaza (deflazacort), which the company priced at $89,000 a year before widespread criticism forced a rethink.
- "We are concerned that marathon's pricing unfairly exploits the DMD patient population and the FDA's orphan drug incentives," the eight senators wrote. Deflazacort was approved as a new drug under the Orphan Drug Act, granting Marathon seven years of marketing exclusivity even though the old steroid has been used for years off-label to treat DMD.
The letter, sent by seven Democrats and the independent Senator from Maine, Angus King, echoes an earlier demand for answers from the prominent Democratic Senators Bernie Sanders, I-VT, and Elijah Cummings, D-MD. Both letters accuse Marathon of abusing the orphan drug program.
Deflazacort, known commercially as Emflaza, has been available overseas as a generic corticosteroid for years, but was never approved in the U.S. or for use in DMD patients in any regulatory jurisdiction.
Even so, the drug was widely used off-label to treat DMD. The Centers for Disease Control and Prevention estimate that roughly 22% of DMD patients treated with steroids in the U.S. took deflazacort alone, with another 14% using deflazacort in conjunction with prednisone, another steroid.
By pushing deflazacort across the regulatory finish line as a new orphan drug, however, Marathon receives substantial benefits. The company will enjoy seven years of marketing exclusivity and has received a priority review voucher from the Food and Drug Administration, which it can either use to expedite approval of one of its own drugs or sell to other companies.
Sales of the vouchers have fetched as much as $350 million, although Gilead's recent purchase of Sarepta's voucher checked in at only $125 million.
Marathon CEO Jeff Aronin has insisted Marathon wouldn't recoup its investment in developing Emflaza for several years, and has emphasized that most patients would pay a low co-pay even before the price revaluation.
The most recent letter and the earlier request from Sanders and Cummings reflect a growing concern drugmakers are taking advantage of the Orphan Drug Act to secure benefits originally designed to reward development of innovative new molecular entities.
Lawmakers on both sides of the aisle have expressed interest in investigating possible misuse of the program, and simmering anger over high drug prices could serve as a powerful motivator for future legislation.
Marathon has not given any update on its plans to restart commercialization, or indicated any new price for the drug. But with pressure from Congress continuing to mount, it will certainly be under the microscope when it does.
Senators signing last week's letter include Patty Murray, D-WA; Tammy Baldwin, D-WI; Cory Booker, D-NJ; Al Franken, D-MN; Maggie Hassan, D-NH; Angus King, I-ME; Elizabeth Warren, D-MA; Sheldon Whitehouse, D-RI.