Emflaza, DMD Treatment, May Soon Be Available in U.S. via PTC Therapeutics

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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PTC Therapeutics

The Duchenne muscular dystrophy (DMD) treatment Emflaza (deflazacort) was successfully acquired by PTC Therapeutics under terms agreed to in March, and may soon become available to patients at, potentially, a new and lower listing price.

The now-completed agreement followed U.S. Food and Drug Administration (FDA) approval of Emflaza to treat all forms of DMD, regardless of mutation, in February. But plans by its developer, Marathon Pharmaceuticals, to charge  $89,000 a year for the treatment was met with outrage and led to a delay in the drug’s release. Emflaza’s key ingredient, deflazacort, is widely used outside the United States to treat DMD and has been available to U.S. patients from outside sources for about $1,200 a year.

Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.

“We are pleased the acquisition was completed ahead of schedule, following early conclusion of the antitrust review period,” Stuart Peltz, PhD, chief executive officer of PTC, said in a recent press release. The company is expected to announce its plans for Emflaza’s relaunch by mid-year.

Financial terms of the acquisition include an upfront payment of $140 million to Marathon, which will also receive payments based on annual net sales of Emflaza beginning in 2018. Marathon can also receive a single $50 million sales-based milestone.

PTC Therapeutics will have all rights to distribute Emflaza in the United States.

The FDA approved Emflaza to treat DMD patients, 5 and older, based on the results a 52-week Phase 3 clinical trial involving 196 boys with DMD between ages 5 and 15. In treated patients, average muscle strength – the trial’s effectiveness measure – was found to be significantly greater at week 12 compared to placebo, and continued to improve through week 52, demonstrating the persistence of the treatment’s benefits.

The participants’ function time – time to stand, time to climb four stairs and time to walk 30 feet – also improved in patients given Emflaza compared to placebo. Participants in the Emflaza group also gained less weight than those taking prednisone.

“We’ve been engaging with key stakeholders in the DMD community to understand their needs and are working to make Emflaza commercially available as soon as possible. PTC is committed to bringing this important therapy to patients with DMD,” Peltz said. “Our goal is to enable access for eligible patients irrespective of insurance status, and we look forward to discussing commercial launch details on our upcoming quarterly earnings call.”