UPDATED: Bluebird bio's $2.8M gene therapy Zynteglo wins FDA backing. Will its US launch take flight?

Bluebird bio’s blood disorder gene therapy has flown the nest early in the U.S.

Wednesday, the FDA approved the company’s one-time beta thalassemia treatment Zynteglo, also known as betibeglogene autotemcel (beti-cel), two days ahead of the regulator’s approval action date of Aug. 19.

The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s chief commercial officer, said in an interview. Bluebird has endured its share of “bumps and bruises over the last year-and-a-half or so,” but things have been looking up since a positive FDA advisory committee meeting earlier this summer, he added.

While bluebird’s approval prospects looked hazy at the start of the year, the clouds parted in June after an FDA expert panel unanimously endorsed the gene therapy. At the meeting, one member claimed the med could be “life-changing” for patients currently fettered by blood transfusions.

The approval clears the drug for kids and adults with beta thalassemia who require regular red blood cell transfusions, the FDA said in a release. Beta thalassemia, a type of inherited blood disorder, causes a reduction of normal hemoglobin and red blood cells in the blood. That in turn leads to insufficient oxygen delivery in the body.

The disorder can cause dizziness, weakness, fatigue and bone abnormalities, plus more serious complications, the FDA pointed out. Transfusion-dependent beta thalassemia is the more severe form of the condition and typically forces patients to undergo lifelong red blood cell transfusions. Transfusions can lead to health complications of their own, including problems in the heart, liver and other organs from an excess build-up of iron in the body.

Regarding launch plans, bluebird aims to bring its first wave of qualified treatment centers online by September, Klima said. Meanwhile, bluebird plans to start collecting patients' cells by 2022’s fourth quarter, with the aim to kick off treatment of its first patients in early 2023, he added.

As a one-time bespoke treatment, each Zynteglo dose is created by genetically modifying a patient’s own bone marrow stem cells. Specifically, the patient’s cells are modified to produce functional beta-globin, which is a component of hemoglobin.

Across two clinical studies looking at adults and kids with transfusion-dependent beta thalassemia, 89% of the 41 patients on Zynteglo achieved transfusion independence, the FDA said.  

Bluebird’s med won’t come cheap. Zynteglo will roll out at a price of $2.8 million per patient, Klima told Fierce Pharma, adding that the company feels that price tag “reflects the value of a one-time treatment in the U.S.” The list price doesn’t include the cost of hospitalization required for patients receiving the therapy, Stat News noted.

While bluebird celebrates the approval, insurers are no doubt taking notice. In launching the drug stateside, bluebird will have to do what it couldn't in Europe: Convince payers of Zynteglo's value.

Bluebird is finalizing contracts with commercial payers now, Klima said. The company's access strategy revolves around a "one-time upfront payment," with the option of a "rebate" for patients who don't achieve transfusion independence, which could help patients recoup up to 80% of the one-time treatment cost, the CCO explained.

The upfront payment and rebate scheme is a "clear positive for [bluebird] and the broader field of gene therapy," RBC Capital Markets analysts wrote to clients Wednesday. However, the RBC team said there's still uncertainty surrounding bluebird's stock, given the "relatively small commercial opportunity" for Zynteglo, especially considering the turbulent experience the drug's had in Europe.

Zynteglo won European approval back in 2019, where it was priced at almost 1.58 million euros (about $1.8 million at the time). However, the company later pulled the drug off the market because it couldn’t reach reimbursement deals with officials in various markets. In April, bluebird clipped 30% of its staff.

With its U.S. approval, meanwhile, Zynteglo is now looking at a potential market of roughly 1,000 to 1,500 transfusion-dependent beta thalassemia patients.

Editor's note: This story has been updated with comments from Tom Klima, bluebird bio's chief commercial officer, as well as commentary from RBC Capital Markets analysts.