There’s a new Duchenne muscular dystrophy (DMD) drug on the scene—at least, technically speaking.
Northbrook, Illinois-based Marathon Pharmaceuticals announced on Thursday that it had snagged an FDA green light for corticosteroid Emflaza, known generically as deflazacort. While the compound has been on the market for decades outside the country—cheaply imported to treat the deadly muscle-wasting disease—it hasn’t before been formally approved by U.S. regulators, who granted it orphan drug designation.
Now, with an FDA-approved med on the market—the only one green-lighted to treat all DMD patients age 5 and older—the import option is off the table for patients, and Marathon is slapping an $89,000-per-year list price on the med. To help the pricey med reach DMD sufferers, Marathon says it’s creating “robust” patient support programs.
“Marathon is committed to working closely with payers to meet the patient and payer needs for acceptable access to Emflaza. Additionally, Marathon has invested in Emflaza being accessible to all families and physicians who wish to use it without undue burden on families or the healthcare system,” the company said in a statement.
The approval comes at a time of increased scrutiny surrounding both price increases on older meds and drugs that have been repurposed to gain orphan exclusivity. Last month, a Kaiser Health News investigation found that about one-third of the orphan drug approvals the FDA had doled out since the program began more than 30 years ago had been for repurposed, large-market products or drugs with multiple orphan green lights. And Friday, U.S. Sen. Chuck Grassley (R-Iowa) opened an inquiry into potential abuses of the Orphan Drug Act that may have contributed to sky-high prices on commonly used meds.
Meanwhile, other drugmakers looking to treat the underlying causes of DMD haven't seen nearly the kind of swift regulatory success that Marathon has. In September, the FDA finally won an approval for long-delayed, controversial treatment Exondys 51, but it was limited to a tiny fraction of patients. Before that, two of its rivals suffered rejections: BioMarin sidelined its candidate, Kyndrisa, after the FDA refused to approve it, and PTC failed to win a hearing at the agency at all.