Duchenne drug poised to cost $35,000 but with major catch

For those that have not followed the story, Emflaza was successfully taken through the FDA regulatory process by Marathon as the first new treatment for Duchenne muscular dystrophy. It seemed great news until the price was revealed, at $89,000 per year when patients and carers had previously been paying around $1,000 a year by importing the drug.

The public backlash to another case of price gauging stung Marathon’s fingertips and it passed the hot potato to PTC Therapeutics – who paid $140 million in March for the pleasure of trying to negotiate a price point on the drug that would not have the public up in arms whilst still allowing it to make a profit.

The price it decided on has been revealed to be $35,000 per year. It is a significant drop on the $89,000 that Marathon were planning to sell the drug for. However, the price is dependent on the weight of patient – with those weighing 25 kilograms and under able to purchase the drug for $35,000.

For those that weigh more than this, the price becomes considerably more expensive. For an adult male with the condition, 75 kilograms is a possibility and that means a price of $105,000 per year is also possibility. This price manages to exceed Marathon’s previous price.

PTC plan to move quickly to get the drug onto market, with a launch predicted to happen in the coming weeks. It will be desperate to avoid a repeat of Marathon’s failure to launch the drug, as it became embroiled in a furore over the pricing and delayed its launch.

Analysts have judged the price to be too aggressive and PTC’s share price reflects this, having dipped by 10%.

Ben Hargreaves