PTC Therapeutics has announced that its recently-acquired Duchenne muscular dystrophy (DMD) drug, Emflaza (deflazacort), will be priced at $35,000 for a year’s worth of treatment. The drug was approved earlier this year while it was still owned by Marathon Pharmaceuticals, however the company’s proposed $89,000 list price attracted plenty of negative attention, eventually prompting the pharmaceutical company to sell the rare disease drug to PTC.
“We are thrilled to bring Emflaza to Duchenne muscular dystrophy patients in the United States,” said Dr. Stuart W. Peltz, Chief Executive Officer, PTC Therapeutics, Inc. “The acquisition of Emflaza supports our commitment to providing new treatment options for Duchenne patients. We plan to launch EMFLAZA in the coming weeks and are focused on providing access to all eligible patients.”
While Marathon conducted 17 preclinical and clinical studies of deflazacort in the DMD patient population, the company did not develop the drug. A generic version of the steroid has been sold in other countries for as little as $1,000 per year – a fact that US DMD patients used to take advantage of by importing the medicine from other nations.
According to Peltz, the new list price is “sustainable” for the company and “balances providing access to all eligible patient in the United States in an ultra-orphan population while maintaining sufficient infrastructure and programs, including continued investment in Duchenne. The factors in our decision include a consideration of the resources required to provide access to Emflaza for a small patient population to improve the standard of care and further the Duchenne community’s educational need while enabling continual investment in therapies for rare diseases and providing financial return for our shareholders.”
In March, PTC acquired Emflaza in a deal worth $75 million in cash and $65 million in stock. The drug is set to be launched in the coming weeks, and only time will tell whether the $35,000 list price will be accepted by patients, payers and industry watchdogs.
“With our nearly 20-year commitment to the Duchenne community, it is deeply meaningful for us to bring this critical therapy to US patients,” said Peltz at the time of the acquisition. “We believe Emflaza is a disease-modifying therapy that has been shown to slow disease progression. In keeping with PTC’s mission, we are excited to work with the community to raise the standard of care for DMD patients.”