Marathon Pharmaceuticals is facing a backlash over plans to launch its just-approved steroid drug Emflaza - available for decades - at the princely sum of $89,000 a year for Duchenne muscular dystrophy.
US politicians including Senator Bernie Sanders and Representative Elijah Cummings have the company firmly in their sights, saying that the pricing is "unconscionable", and suggesting the firm is abusing "government-granted exclusivity periods and incentives" in search of profits.
In a nutshell, they want Marathon to justify its claim that the cost of developing Emflaza for DMD warrants that price tag, given that deflazacort a is "widely available abroad for approximately $1,000 per year".
They want information on the cost of trials as well as licensing rights to the drug, the calculations used for setting its price and estimates of how much revenue Marathon intends to make from it.
A report in Endpoints suggests that the cost of developing the drug is probably somewhere between $50m and $75m, and that if half the DMD patients in the US received Emflaza based on the company's estimated net price of $54,000 per year (after discounts) it would translate to revenues of more than $400m a year.
Sanders and Cummings note that Marathon also received another lucrative award from the government, a priority review voucher, which could cover the cost of developing the drug if sold on to another company.
In response, Marathon has said it is "pausing its commercialisation plans" for Emflaza while it meets with the DMD community. It added that it would continue its expanded access programme for the drug, which it says is "modestly priced" for an orphan medicine.
The company reiterated its position that every patient who needs the drug will have access to it for free or with a minimum co-pay. But that positon - commonly voiced by companies facing scrutiny over pricing - is increasingly falling on deaf ears in the US as high prices affect everyone in the form of rising insurance premiums.
Sanders and Cummings say Marathon's action is "an indictment of a system that allows drug companies to engage in such opportunistic pricing behaviour”.
Marathon chief executive Jeffrey Aronin said the company is "committed to the Duchenne community for the long term and we will re-invest the earnings into developing and funding additional therapies”.