How low does DMD med Emflaza's price need to go? PTC hopes $35,000 is low enough

PTC Therapeutics shelled out $140 million-plus in March for a newly FDA-approved Duchenne muscular dystrophy drug, Emflaza, amid an outcry over the $89,000 list price set by its previous owner. PTC now says it's planning a $35,000 annual net price.

But is that enough to win over angry parents, politicians and payers? That's open for debate as analysts and payers digest the new figures.

Emflaza is an older steroid, sold in other countries for as low as $1,000 per year, and at least one potential payer has already said the ultracheap generic prednisone is a reasonable alternative to the new branded med. 

After previously estimating a PTC price of $25,000, RBC Capital Markets analyst Simos Simeonidis wrote Monday that he expects the launch at $35,000 "will prove challenging." PTC will likely experience payer pushback and "continued headline noise," the analyst said.

The company had "no choice other than dramatically lowering the price," Simeonidis had said back in March, when PTC snapped up the product in a $140 million-plus deal. "The question," Simeonidis pointed out, "is how low is low enough, especially when patients (and now politicians, given all the noise) know that the drug is available overseas for $1,000/year?"

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

Importantly, the $35,000 price may be after discounts, but that's for a patient weighing 25 kilograms (55 pounds), according to a PTC spokesperson. Treatment would cost more for patients who weigh more. The drugmaker didn't disclose whether it adjusted the list price from Marathon's figure, according to the Wall Street Journal.

On a Monday conference call, PTC CEO Stuart Peltz described the company's net price as “sustainable,” according to Marketwatch. His reasoning was that the figure “balances providing access to all eligible patients in the United States in an ultra-orphan population while maintaining sufficient infrastructure and programs, including continued investment in Duchenne.”

PTC agreed to pick up Emflaza from Marathon back in March as the latter company suffered from plenty of unwanted attention, including from Congress, over its $89,000 list price. Marathon brought the drug to the U.S. market after it had been available elsewhere for decades—and after many DMD parents had grown accustomed to obtaining supplies from overseas markets.

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

Those factors led to a quick backlash, and Marathon sold off the drug in a deal potentially worth more than $190 million, including an up-front cash payment and some sales-related milestones built in. Among Marathon’s critics were Sen. Bernie Sanders and Rep. Elijah Cummings, who later reached out to PTC Therapeutics to learn more about its plans for Emflaza.

Since then, Marathon has resigned from PhRMA after the industry trade group kicked off a membership review. The private company has also discussed shuttering its operations, according to Endpoints News.

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

For its part, PTC is now set to launch Emflaza in the coming weeks. Peltz added in a Monday statement that the company is “focused on providing access to all eligible patients.”

Emflaza’s FDA approval followed Sarepta’s tough but successful campaign to win approval for Exondys 51, now approved for some patients with the debilitating and rare Duchenne muscular dystrophy. The condition affects mostly boys and progressively impacts the ability to independently perform routine activities.

Editor's note: This story was updated with analyst comments and to reflect that PTC's disclosed price for Emflaza is after discounts.