For a simple systemic steroid that is cheap and easily available outside the US, deflazacort has received unusually careful support from the FDA, breezing through a regulatory process that has bedeviled several developers trying to address the root cause of the disease.
Deflazacort won a priority review from the agency as a new therapy for Duchenne muscular dystrophy — cutting four months off the regulatory process — along with an orphan drug designation and rare pediatric disease status. And today, the FDA handed Marathon Pharmaceuticals a green light to start marketing the drug to all boys 5 and older whose lives are being slowly destroyed by the disease.
The corticosteroid will be sold as Emflaza after one study involving 196 DMD patients indicated that it could improve the strength of patients with this disease. In another trial with 29 male patients that lasted 104 weeks, the FDA reported, “deflazacort demonstrated a numerical advantage over placebo on an assessment of average muscle strength.”
The larger of those two studies was completed 21 years ago, according to an abstract posted in Neurology last year.
For that, the company plans to price the drug at $89,000 a year, according to the Chicago Tribune, more than $88,oo0 a year more than what Canadians would pay for the same drug. But like other cases involving price gouging, the company went on to defend the price, saying that insurers will cover most of the price.
In addition to the approval, Marathon wins a rare pediatric disease priority review voucher. These vouchers have fetched hundreds of millions of dollars from drug companies looking for a quick and sure way to slash four months off of one of their own drug reviews.
Deflazacort has been available in countries around the world for decades.
Currently, you can buy deflazacort from online pharmacies in Canada for less than a buck a pill. Until fairly recently it was sold by Shire in the UK, for example, as Calcort in 6 mg tablets and used to treat a variety of chronic autoimmune conditions like rheumatoid arthritis. (A spokesperson for Shire says that the drug changed hands.) But because it’s never been approved in America, where other steroids have been available for those same conditions, it gets the full regulatory treatment in the US.
One Duchenne mom emailed me to say that she felt her family was being held “hostage” by Marathon, after being able to buy the drug for a thousand dollars a year. And more criticism began to surface on Friday, as the Wall Street Journal reported that Christine McSherry, who runs the nonprofit Jett Foundation, said she is “disappointed that Marathon increased my cost for the drug by more than $87,000 a year.” A number of patient advocates said they were worried especially for families with high deductible insurance plans.
Deflazacort now joins a list of controversial meds that have been acquired and then sold at an astronomical markup. Martin Shkreli became the poster child of the pricing debate with his 5,000% price hike on Daraprim, another old generic drug that targets a rare condition, which came without any additional research. And executives at Valeant and Mylan were hauled in front of lawmakers last year and grilled on their own pricing tactics for aging products.
None of it is against the law in the US, though, which is one reason why it’s so controversial. The FDA is required by law to be agnostic on pricing issues.
“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” said Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “We hope that this treatment option will benefit many patients with DMD.”
Up until Marathon, though, Duchenne MD has been a combat zone at the FDA.
Sarepta won a controversial approval for its DMD drug last year after fighting for years to get an OK based on data from a tiny, badly flawed study. Their approval came with a label that tells patients that the company has yet to gain clear evidence of efficacy. PTC was shut out, much to their chagrin. And BioMarin‘s drisapersen was shown the door as well.
Marathon did not respond to my queries. The biotech is run by CEO Jeffrey Aronin, who started Ovation and sold it to Lundbeck for $900 million in 2009.
I asked the FDA why the drug warranted VIP approval. Their response:
Deflazacort has never been approved for any use in the United States. Under U.S. law, it was reviewed as a “new drug” and assessed for safety and efficacy for the specific conditions of use in the labeling (prescribing information). Versions of deflazacort are available in some countries for other indications, but not for DMD. The U.S. approval is the first anywhere for DMD. The FDA-approved product labeling includes safety and clinical information specific to the drug’s use in DMD. If a drug meets the statutory requirements for orphan drug designation, expedited programs, and rare pediatric disease designation, then a sponsor is eligible to receive those benefits.
“We are in a new era in the treatment of Duchenne muscular dystrophy. For the first time, patients in the U.S. with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition. We are pleased that this development will help patients with this disease stay stronger longer,” said Timothy M. Cunniff, Pharm.D., Executive Vice President, Research & Development, Marathon Pharmaceuticals, in a statement.
— John LaMattina (@John_LaMattina) February 10, 2017
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