(Bloomberg) -- Bluebird Bio Inc.’s gene therapy for a rare blood disorder was cleared by US regulators, and the company said it would charge $2.8 million for the first-ever, one-time treatment for patients with beta thalassemia.

Sold under the brand name Zynteglo and also known as beti-cel, the treatment is a game-changer for patients who now depend on regular blood transfusions for most of their lives. Previously, the only available cure was a bone marrow transplant from a sibling. Bluebird’s therapy promises a one-time fix by inserting a functional copy of the faulty gene that causes beta thalassemia, an inherited inability to make sufficient amounts a protein that shuttles oxygen throughout the body. 

The drug’s price is in line with a range considered cost-effective by an independent review group. Insurers would pay a one-time fee and get their money back if the therapy doesn’t work as promised within a set amount of time, the company said in a statement.

Zynteglo’s US approval should help revive Bluebird, a gene-therapy pioneer that in recent years has gone from a biotech darling to the brink of running out of money. It marks a milestone for the industry as much as it does for patients and the company, Chief Executive Officer Andrew Obenshain said in an interview. 

“We’re finally there, and we’re finally able to have that impact” on the lives of people with beta thalassemia, Obenshain said. 

The shares jumped as much as 24% Wednesday in New York after being halted leading up to the approval, and were up about 7% at 3:06 p.m.

The clearance “gives the company a path to meaningful revenue after a failed attempt in Europe and US regulatory delays,” Bloomberg Intelligence analyst Marc Engelsgjerd wrote in a note. 

Read More: Bluebird Sees Road to Revival After Gene-Therapy Misfires 

A panel of FDA advisers voted unanimously in June to back Zynteglo’s clearance, saying the benefits of the treatment outweighed its risks. In late-stage trials, 89% of patients treated with the gene therapy remained transfusion-free, and there haven’t been any notable safety issues. 

Alexis Thompson, who has treated people with Bluebird’s gene therapy in clinical trials, said most of those patients’ lives have changed dramatically, though not everyone has responded. People with beta thalassemia who require blood transfusions can spend four to six hours at a facility every two to four weeks, sometimes even requiring overnight hospital stays, said Thompson, who leads the hematology division at Children’s Hospital of Philadelphia. The grueling schedule can interrupt children’s schooling, adults’ work schedules, or anyone’s life. 

Patients’ Dreams

“It’s only now that patients can tell you what they had always hoped to do, but never dared to really allow themselves to dream about,” said Thompson, who has consulted for Bluebird, “whether it is marrying and having their own families, traveling internationally, attending school without having to take the time off that’s needed to engage in what to most of us feel like would be a more normal family life.”

Zynteglo was authorized in Europe in 2019, but Bluebird withdrew the product in 2021 after payers balked at the $1.8 million price tag. Bluebird had proposed spreading the cost out over five years and giving insurers their money back if the therapy didn’t free patients from blood transfusions. It turned out that insurers preferred paying for the treatment all at once, leading Bluebird to scrap the extended payment plan in the US, Obenshain said.

The setback in Europe delayed Bluebird’s hopes of making money, leaving the company teetering on the brink of going out of business. Once a biotech darling, Bluebird’s shares had fallen to less than $7 as of Tuesday’s close, about 96% from their 2018 peak. 

Bluebird will be in a “very, very different” financial position following the approval of Zynteglo and the potential approval of another gene therapy expected later this year, Obenshain said. Upon each clearance, Bluebird will receive a voucher that grants a speedy regulatory review for a chosen drug. Bluebird plans to sell the vouchers, which could fetch around $110 million each. 

Brain Therapy

“We’re definitely evaluating the next steps to bring more cash in,” Obenshain said. “I’d say the urgency to do that is certainly less than it was.”

Bluebird will introduce the gene therapy through a handful of qualified centers. The company is limiting places that can administer the treatment because it’s so complex. A patient’s blood cells are collected and sent out to be genetically modified, which takes 70 to 90 days. Then, the patient undergoes harsh chemotherapy to wipe out their bone marrow. Finally, the patient is infused with the modified cells. 

Bluebird expects to identify the first prospects by the end of the year and start giving them therapy in the first quarter of next year, Obenshain said. About 50 potentially eligible people are now getting care at the centers that will be the first to administer the gene therapy treatment, Bluebird estimates. Eventually, Bluebird expects about 850 people could become candidates, or about half of the roughly 1,500 people with transfusion-dependent beta thalassemia in the US.

Bluebird is awaiting a decision from regulators on eli-cel, its gene therapy for a brain-wasting disease called cerebral adrenoleukodystrophy. That decision is expected by Sept. 16. Bluebird also plans to seek approval for lovo-cel, its gene therapy for sickle cell disease, early next year. Analysts are expecting that treatment to be the most lucrative of Bluebird’s three therapies. 

(Updates with analyst’s comment in sixth pararaph)

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