New gene therapy costs $3.5 million, but Greenville mothers explains why it may be worth it

GREENVILLE, S.C. (FOX Carolina) - The world’s most expensive drug is available here in South Carolina. It was approved by the FDA two months ago to treat a rare blood disorder and costs about $3.5 million. One Greenville mother explains why the treatment may be worth the cost.

Sue Martin has five children, a daughter and four boys who were star athletes in high school.

“They took state championships, all three of them,” Martin said. “It was a rare feat and so this was really exciting.”

Three of her son’s were diagnosed with a rare blood disorder known as hemophilia B. It’s a genetic disorder that hinders their ability to produce a blood clotting protein.

“The three boys were not normal when it came to their injuries,” Martin said. “They were always prolonged and there was always extra bleeding.”

What made matters worse is that their treatments were massively expensive, costing up to $300,000 a year. They paid a portion of that thanks to their insurance, but they had a cap. It’s why her husband often moved and switched jobs.

“We would start another insurance policy in another state and start all over,” Martin said. “They have their treatment and then before we hit our maximum annual cap, we would move again.”

Martin is now the executive director of the Bleeding Disorders Association of South Carolina. Her children have grown into adults, but their blood disorder is a lifelong condition they still have to treat. Currently she says they undergo replacement therapy every few months. They intravenously infuse themselves with factor IX, which is the clotting protein their body struggles to produce.

“Right now their treatment is about $250,000 per person each year,” Martin said.

That’s the cost without insurance and the treatment is continuous. But a new one-time dose gene therapy known as Hemgenix could change all that. The drug company says the cost is $3.5 million.

Dr. Shayla Bergmann is a pediatric hematologist at the Medical University of South Carolina. She says the treatment uses a virus to alter cells.

“It uses a modified adenovirus particle that is able to carry the correct DNA code or sequence into a cell,” Dr. Bergmann said.

Just like viruses mutate genes in a bad way, researchers have learned to engineer them in a good way. The engineered adenovirus makes its way into liver cells and delivers a copy of the functional gene that people with hemophilia b are missing. Once delivered into the cell it corrects the DNA sequence to help the body start producing factor IX.

“You are correcting the sequence so you can make the protein,” Bergmann said.

Bergmann says MUSC is currently the only place in the state offering the treatment and only certain people with hemophilia B qualify. Once patients receive this treatment she said they can’t receive another gene therapy down the road.

“They can get it one time and the hope is that it lasts for a long duration, hopefully for their life,” Bergmann said.

The cost of $3.5 million dollars may sound startling. The drug company, CSL Behring, says this price was based on long-term “cost savings.” In a statement the company said the cost “of treating people with moderate to severe hemophilia B can be significant over a lifetime, by some estimates more than $20 million dollars per person.”

Martin is not sure if her children will try the new treatment, but is happy it is out there as an option.

“If you look at what this costs, if it lasts for a very long time, those patients will be saving a lot of money,” Martin said. “And they will not be bleeding and continuously bleeding.”

CSL Behring also says they will be providing discounts to certain patients through a 340B program and value based agreements with commercial payers.

“The 340B program helps subsidize the cost of critical supportive coordinated care that hemophilia treatment centers (HTCs) provide to people with hemophilia and other bleeding disorders. CSL Behring also will offer value-based agreements as we are confident in the value of HEMGENIX to people with hemophilia B and the healthcare system,” a spokesperson for CSL Behring said.

It’s not clear if health insurance will cover the cost of the treatment. The Centers for Medicare and Medicaid Services faced a similar decision with an expensive gene therapy known as CAR T-Cell therapy in 2019. They decided to cover the cost when administered at healthcare facilities under certain conditions.

Currently Pfizer is also developing a similar gene therapy for hemophilia B. They released their phase three clinical trial results last month and Dr. Bergmann says the effectiveness of that treatment is similar to Hemgenix. But we don’t know the price yet.

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