March 16, 2017
By Mark Terry, BioSpace.com Breaking News Staff
South Plainfield, NJ-based PTC Therapeutics announced that it was acquiring rights to Marathon Pharmaceuticals’ Emflaza (deflazacort) to treat Duchenne muscular dystrophy (DMD) patients five years or older.
On February 10, Marathon, based in Northbrook, Ill., announced that the U.S. Food and Drug Administration (FDA) had approved Emflaza. This presented a number of issues. First, deflazacort is a corticosteroid that has been on the market in other countries for decades. It is widely used as an anti-inflammatory agent. A similar anti-inflammatory corticosteroid, Prednisone, is often used to treat DMD. Secondly, Marathon indicated it planned to charge $89,000 a year for the drug. It is available in Canada and other markets, however, for about $1,000 for a year’s supply.
This created a lot of outrage, particularly in light of ongoing criticism of drug pricing by President Trump, Senator Bernie Sanders, and during the U.S. presidential election, Secretary Hillary Clinton.
Marathon appears to have opted out of the controversy by selling its rights to the drug to PTC for $140 million in cash and stock, as well as a potential $50 million sales bonus and royalties that could hit low-to-mid 20s.
“With our nearly 20-year commitment to the Duchenne community, it is deeply meaningful for us to bring this critical therapy to U.S. patients,” said PTC’s chief executive officer, Stuart Peltz, in a statement. “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.”
John Carroll, writing for Endpoints News, says, “Claiming that deflazacort is a disease modifying therapy will surprise many in the Duchenne community. Like any steroid, it strengthens patients at a cost. Many of the parents came to prefer deflazacort over prednisone because it is associated with less weight gain.”
In addition, Christine McSherry, a DMD advocate and head of The Jett Foundation, notes that many DMD families were able to get the steroid without any particular problem. She estimated that 40 percent to 50 percent of DMD were already on deflazacort, although she later dropped that estimate to 25 percent. She also suggested that they will be forced to buy the drug through higher priced U.S. supplier.
PTC has had issues with both the FDA and its own DMD drug, ataluren, which failed back-to-back clinical trials for DMD and on March 2, failed a clinical trial for nonsense mutation cystic fibrosis (nmCF). The company still insists the drug is effective when you look at the “totality” of the data. On March 6, the company indicated the FDA had agreed, over protest, to consider PTC’s New Drug Application (NDA) for Translarna for nonsense mutation DMD, with a PDUFA date of October 24, 2017.
The drug has conditional approval in Europe, contingent upon an additional trial in the next five years.
PTC hasn’t indicated how it will price deflazacort (Emflaza). It’s no doubt that advocates and U.S. politicians will be watching, however. Even the Pharmaceutical Research and Manufacturers of American (PhRMA) released a statement criticizing Marathon’s Emflaza pricing and indicating that it’s practices in this regard could threaten its membership in the organization.
The statement said, in part, “The leadership of the PhRMA Board of Directors has begun a comprehensive review of our membership criteria to ensure we are focused on representing research-based biopharmaceutical companies who take significant risks to bring new treatments and cures to patients.”
