PTC comes under fire from Congress for Emflaza
- Backlash to the price of a recently-approved steroid for Duchenne muscular dystrophy (DMD) continued Wednesday, with Senator Bernie Sanders and Representative Elijah Cummings sending a letter to PTC Therapeutics asking what price the company intends to set for the drug.
- In an odd move, PTC bought the rights to the drug last week from Marathon Pharmaceuticals in a deal worth $140 million that included a $75 million upfront and shares of the acquiring company.
- Marathon gained approval for the decades old steroid for DMD from the Food and Drug Administration last month and quickly faced scrutiny when they set the price at $89,000 for a course of treatment. Marathon was then forced to delay the launch of Emflaza (deflazacort).
In a highly anticipated move, Sanders and Cummings asked the biotech company to reveal its intentions for pricing Emflaza.
The drug, which is available outside the U.S. for about $1,000 to $1,200 for a course of treatment, has long been imported and used by patients with the devastating rare disease.
“We urge you to keep the price of this relatively common steroid at its current importation cost,” the congressmen said in the letter, also asking if the company intended to pursue approval of deflazacort as an orphan drug for juvenile arthritis.
PTC CEO Stuart Peltz has until April 3 to provide answers to the inquiry.
The FDA approval shocked many, and gave Marathon (and now PTC) seven years of exclusivity on the marketing of the drug, as well as the power to price it more steeply.
The advantages are part of the Orphan Drug Act of 1983, which gives companies incentives for developing drugs for orphan diseases.
Yet, many industry skeptics feel that the incentive program is no longer working the way it was intended and is instead being abused by drug companies who want to charge steep prices.
The controversy over Emflaza is not a new one; the backlash to drug pricing has reached a fever pitch over the last two years. And companies that raise the price of older drugs have come under particular scrutiny.
- Congress of the United States Letter to PTC Therapeutics
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