It’s time for Stuart Peltz to take the Congressional hot seat on drug pricing.
The CEO of PTC Therapeutics acquired Marathon’s steroid deflazacort for $140 million up front a few days ago with an eye to sell it to a small population of Duchenne muscular dystrophy patients. And that comes with as much of a glaring spotlight as Senator Bernie Sanders and Rep. Elijah Cummings can bring to it.
In a new letter directed to Peltz, the two activist lawmakers noted in a preemptive strike that the CEO has been in touch with the Parent Project for Muscular Dystrophy to talk price. And they have a retail number in mind that PTC $PTCT won’t likely cotton to: The UK net price of $1,000 to $1,200 a month, which is what many parents are paying today.
“We urge you to keep the price of this relatively common steroid at its current importation cost,” the two lawmakers note. They also are asking what Peltz’s plans are in pursuing another orphan approval for juvenile arthritis.
Peltz wasn’t ready to tell analysts what he thought PTC’s price would be when he announced the deal to acquire the controversial steroid. That was a subject that he said would be revisited in light of the controversy over the $89,000 price. It’s unlikely, though, that he was thinking of a range stretching from $12,000 to $14,400 a year.
Peltz is quite familiar with controversy, though. He managed to persuade the Europeans to approve ataluren for Duchenne muscular dystrophy, even though that therapy has now failed three straight studies, including two for DMD. He was barred at the door by the FDA, but used agency’s regulations to force a review and PDUFA date for the drug.
Marathon and its drug became virtually radioactive after it priced deflazacort at $89,000 after gaining a narrow FDA approval as an orphan therapy, spawning a fresh wave of outrage over price gouging that left PhRMA reviewing its membership rules and whether Marathon CEO’s Jeff Aronin should be ousted from the board. Sanders and Cummings also followed up recently by asking the FDA why it handled a cheap, old steroid as an orphan drug, in a program that will now be reviewed by the GAO.
Aronin notoriously pledged to Duchenne families that his company had done the “heavy lifting” on deflazacort with a development program that required 17 trials. But it turned out the company bought the efficacy data it needed for only a low six figures, according to the Wall Street Journal, and much of its “heavy lifting” included small preclinical and clinical studies that likely were done on the cheap.
Now Peltz can try to find out if you can take a cheap old steroid that’s been available for years from overseas sources at a marginal cost and reprice it for a US market without coming under a full assault from outraged lawmakers. That’s no easy task.
On the other hand, Martin Shkreli practically invented the simmering controversy over price gouging with his decision to up the price of an old generic more than 5000%. Shkreli resigned from his post at Turing — and is now facing unrelated fraud charges — but the biotech never discounted the price, despite Congressional hearings, angry lawmakers and an online mob that shrieked for retribution. There are no laws restricting drug prices, as Peltz is well aware of.
To be continued.
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