Payer snubs PTC's Emflaza, signaling pricing trouble ahead of launch

Washington state's largest healthcare purchaser will put limits on PTC Therapeutics' Emflaza, according to a new report, instead opting to treat DMD patients with prednisone.

PTC Therapeutics faces an uphill battle to extract brand-level pricing and sales with its new, and controversial, DMD drug Emflaza if a new policy report out of Washington state is any indication of payer sentiments.

Washington State’s Health Care Authority, the largest purchaser in the state, published a review of the newly approved med compared to the much cheaper prednisone, currently the most commonly used steroid by Duchenne muscular disorder patients.

Emflaza, a decades-old steroid itself, recently won FDA approval under Marathon Pharma’s ownership. But that drugmaker’s decision to attach an $89,000 price tag to a med that’s available elsewhere for much cheaper created instant backlash. Marathon eventually offloaded the drug to PTC Therapeutics in a deal potentially worth more than $190 million.

RELATED: Marathon, under heavy fire for Emflaza pricing, makes surprise deal to sell drug for $140M-plus

As Washington State’s HCA noted, PTC Therapeutics has not announced its new price. But the group published a report (PDF) stating that prednisone—at a cost of 5 cents per tablet and $55 per year—will be its preferred corticosteroid for DMD patients. It’s the “lower cost, equally effective” option, according to HCA.

Emflaza will be reviewed on a case-by-case basis and won’t be used off-label, the report stated.

The PTC med’s efficacy has been tested against prednisone in four studies, according to HCA, with all featuring a “very low quality of evidence on outcomes of muscle strength, motor outcomes, weight gain, and cataracts."

RELATED: Marathon halts launch of DMD drug as $89K price attracts congressional heat

Two of three systematic reviews of the data found the meds similarly effective, but noted that Emflaza leads to less weight gain. A third review couldn’t find a difference in effectiveness and found “very low quality evidence” that Emflaza is associated with less weight gain.

Marathon won approval for Emflaza in February but immediately ran into backlash over the price. Under Congressional scrutiny, the drugmaker halted the drug's launch then sold it in March to PTC Therapeutics.

RELATED: Lawmakers want to know PTC's pricing plans for ex-Marathon drug Emflaza

Since that announcement, Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., have pressed PTC for info on its marketing plans. In a letter to PTC CEO Stuart Peltz, they asked the company to “keep the price of this relatively common steroid at its current import cost.”

Peltz responded in a statement that “PTC continues to engage with key stakeholders to understand how to make Emflaza available to patients who need it.”