Dive Insight:

Bluebird’s financial problems seemed unthinkable just a few years ago, when the company was pioneering the development of gene therapies for rare blood and brain diseases, had a multiple myeloma treatment in advanced testing and a market capitalization in the billions of dollars.

But the company has tumbled since. Trouble selling its gene therapies in Europe led the company to close its operations there, while regulatory submissions in the U.S. were delayed. Multiple setbacks and clinical holds enabled a rival program from CRISPR Therapeutics to move past Bluebird’s.

The company vowed a decision to split in two last year and spin off its oncology business would help right the ship and allow it to focus on its coming drug launches. Instead, its financial runway has gotten shorter and shorter, to the point that the U.S. review of those gene therapies, beti-cel and eli-cel, will likely determine Bluebird’s future.

Should they gain approval on schedule, beti-cel and eli-cel would be only the third and fourth gene therapies for inherited diseases to reach market in the U.S., following Roche’s Luxturna and Novartis’ Zolgensma. Both would represent new options for patients with two inherited conditions, the blood disorder beta thalassemia and the lethal brain disease cerebral adrenoleukodystrophy. They’d also lead to the FDA to award Bluebird with two priority review vouchers, which speed up drug reviews and can be sold to other companies. Bluebird has specifically identified selling those vouchers as crucial to its financial future.

Bluebird should be able to command high prices for each of its treatments, too, given they’re intended to be one-time treatments meant to stabilize patients with conditions that can require numerous costly healthcare interventions. Novartis sells Zolgensma for more than $2 million, for example, and Roche charges $850,000 for Luxturna.

But the U.S. market for Bluebird’s drugs is small. Around 1,500 beta thalassemia patients and 50 adrenoleukodystrophy patients would be eligible for the gene therapies following approval, wrote Luca Issi, an analyst at RBC Capital Markets. Demand for beta thalassemia treatments is higher in Europe, but payers there balked at its $1.8 million price tag.

Issi characterized the U.S. market in beta thalassemia as “modest,” while the stock implications of an adrenoleukodystrophy approval were “limited” because of the number of patients. The FDA has scheduled a two-day advisory panel meeting on both treatments on June 9 and June 10. The agency will make an approval decision on beti-cel by Aug. 19 and eli-cel by Sept. 16.

Before that, however, Bluebird needs to get the FDA’s blessing to restart testing of eli-cel. The FDA stopped testing after the occurrence of a bone marrow cancer known as myelodysplastic syndrome in patients in the clinical trial. Issi wrote that the “large unmet medical need” may spur FDA reviewers to approve its use more widely in spite of the risk of that adverse effect.

The pressure is on, though, as Bluebird’s cash reserves are dwindling. Beyond selling the vouchers, the company is currently “exploring multiple financing opportunities” while also “focusing on further cost efficiencies.” The effort will now be led by Jason Cole, who will take over for Consylman as CFO after her departure on April. He has been serving as chief business officer since last November.