FDA Approves Muscular Dystrophy Drug, Will Cost Patients $89,000

While available for several years outside the United States, a drug used to treat an unusual form of muscular dystrophy was approved by the federal Food and Drug Administration (FDA) earlier this week. However, the cost for the medicine could make you sick.

A USA Today report reveals the drug, a steroid known as deflazacort, will cost patients a whopping $89,000 per year, nearly 70 times higher than buying the drug in other countries. Patients overseas can obtain the treatment for around $1,200 per year.

Deflazacort, sold under the name Emflaza, is an effective treatment for Duchenne muscular dystrophy by helping to improve muscle strength and reducing inflammation minus the health complications associated with other steroids. The incurable disease typically strikes teenage boys, most of whom lose their ability to walk and die before they reach age 40.

deflazacort can treat rare form of muscular dystrophy
Duchenne muscular dystrophy, a rare form of the disease that affects young boys, can be treated with a drug named deflazacort. [Image by Michael Loccisano/Getty Images]

Marathon Pharmaceuticals, the manufacturer of the drug, got approval to classify deflazacort as an “orphan drug,” a category specifically intended to encourage research of drugs for very rare diseases. Even though the drug has been available as a generic in other countries for quite some time, the federal designation also gives Marathon exclusive rights to market the drug in the U.S. for seven years.

The company was also able to obtain a “priority review” voucher. Fundamentally, the voucher is a way to get a future drug reviewed faster by regulators. It also opens the door to selling the treatment to a competing company for millions of dollars. The intent of the vouchers is to inspire drug companies to devote more resources to creating treatments that help children.

However, some companies have used these incentives to their advantage and created business models based on procuring older drugs and substantially raising their prices. In recent years, companies like Valeant Pharmaceuticals International and Turing Pharmaceuticals LLC have come under intense scrutiny from regulators and Congress for price gouging and abusing the rules just for profits.

“It seems like it’s yet another example of gaming the system,” said Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, as quoted by the Washington Post. “How many examples of this do we have to see before we can start to rethink the priority review voucher as a means of incentivizing innovation? This also seems to be another example of gaming the Orphan Drug Act, which was intended to try and encourage research into new therapeutic entities for people who have rare diseases — and it doesn’t seem like this is that.”

Some drug companies use the FDA approval system to take advantage of patients.
Former head of Turing Pharmaceuticals Martin Shkreli was accused of illegally manipulating the price of life-changing drugs. [Image by Drew Angerer/Getty Images]

While the $89,000 price tag is more than most could likely pay out of pocket, Marathon’s Chief Financial Officer, Babar Ghias, provided some optimism to patients. He says the net price will be closer to $54,000 per year once rebates and discounts kick in. Ghias also noted that insurance and financial assistance programs should bring the price down to zero.

This is little reassurance to patients and families who need access to the drug.

“Instead of making the price at a level that is reasonable for patients, they make it a very high price and offer this pathway that patients may not qualify for, they may not know about, there may be limitations on it. So it’s a marketing move and not really a public health solution,” said Kesselheim, per the Washington Post.

For the last two decades, patients have been buying deflazacort from other countries ever since clinical trials demonstrated its effectiveness for treating Duchenne muscular dystrophy. However, the drug was never approved by the FDA as no companies thought it profitable enough to jump through the regulatory hoops necessary to sell it in the U.S.

Even at the cost of $89,000, patient advocacy groups are enthusiastic about the FDA approval of deflazacort. Although many questions, including pricing, still need to be answered, Parent Project Muscular Dystrophy said in a statement that the government’s approval would increase access to more families that desperately need the treatment.

[Featured Image by Andrew Harnik/AP Images]