February 10, 2017
By Mark Terry, BioSpace.com Breaking News Staff
In a move that is sure to raise eyebrows, Northbrook, Ill.-based Marathon Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to treat Duchenne muscular dystrophy (DMD) in patients five years and older.
There are several reasons to wonder about this. 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 used to treat DMD. Secondly, Marathon has indicated the company will charge $89,000 for a year of the drug. It is available in Canada, for example, for about $1,000.
One study examined 196 DMD patients and found that Emflaza improved the patients’ strength. Another trial with 29 male patients ran 104 weeks, and the FDA reported, “deflazacort demonstrated a numerical advantage over placebo on an assessment of average muscle strength.” But the larger study was completed 21 years ago.
John Carroll, writing for Endpoints News, says, “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 U.S.”
DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.
2016 marked a lengthy and dramatic approval process for Sarepta Therapeutics ’s eteplirsen for DMD. After numerous public hearings, letters from Congress, and a great deal of internal conflict at the FDA, eterplirsen was approved in September 2016. It is on the market now as Exondys 51 and has a price of $300,000 for a year’s treatment, although that price is based on the weight of the patient.
In defense of its pricing, and what seems like a jab at Sarepta, Greg Wujek, Marathon’s vice president of Payer Relations, said in a statement, “We have developed models to ensure Emflaza is of great value to payers, and does not create a burden of any sort to the healthcare system. This is important to us, as an American research and development company that has a long-term vision of bringing many new therapies to those living with rare medical conditions such as Duchenne muscular dystrophy. Emflaza will have the same cost for all tablet dosage strengths and nearly all tablet dispensing combinations so there is no distinctive to increasing the dose as the patient weight increases.”
Carroll questioned the FDA as to why Marathon’s drug warranted the VIP approval. They responded: “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 benfits.”
Marathon indicates that it conducted 17 new preclinical and clinical studies, as well as the two additional clinical studies. “Additional post-market research will continue to advance the science of the drug and the care of patients with Duchenne. One planned study will examine various dosage regimens in younger patients with Duchenne to determine if earlier intervention is safe and effective and ultimately impacts the course of the disease. A second planned study will examine various dosage regimens innon-ambulatory patients with Duchenne and characterize the pulmonary and cardiac effects of Emflaza.”
Some investors have expressed concern about the competition to Sarepta, whose dropped slightly at the news of Marathon’s approval, although only by about two percent, then recovered. Emflaza and Exondys 51 have dramatically different mechanisms of activity, so it seems unlikely that they would affect each other’s sales.
