Last month’s news that Bluebird Bio had been hit with another hold on its sickle cell gene therapy was a deflating moment. But the firm, which has been dealt a number of setbacks, is pushing forward with the commercial launch of two other one-and-done treatments for rare, childhood diseases.

The two gene therapies, both of which have been submitted to the FDA, are beti-cel for transfusion-dependent beta-thalassemia (TDT) and eli-cel for cerebral adrenoleukodystrophy (CALD). The FDA is set to decide on the drugs in May and June of this year, respectively.

At the JPMorgan Healthcare Conference Wednesday, the biotech’s CEO and president, Andrew Obenshain, offered an update on its launch preparations. 

“We’re engaging with payers on the value of our therapies and putting in place wrap-around patient services,” said Obenshain, who moved into his current role in November, just prior to the company’s split into two separate companies. Obenshain, formerly Bluebird’s European head, took over for Nick Leschly, who now runs the oncology spin-off company 2seventy bio.

The commercial moves for the beti-cel launch include setting up treatment centers at hospitals, educating a referral network, providing patient support and engaging early to ensure access. Its field force is talking with KOLs on a regular basis, said Obenshain, adding that Bluebird is also drawing on its experience of launching the TDT therapy in the EU. 

That experience has been a decidedly mixed one, however. Bluebird ceased commercial operations in Europe last summer, after what it said were difficulties convincing EU governments to pay steep up-front prices for its treatment, then sold under the brand name Zyntelgo, with the promise of reducing downstream health system costs. 

Bluebird had sought $1.8 million per treatment. Negotiations with the German government broke down in the spring after authorities there reportedly offered to pay $790,000. They later increased the reimbursement to $950,000 pending proof of a reduction in the need for blood transfusions. 

Tom Klima, chief commercial officer, said the European experience was otherwise “quite successful. We had a bolus of patients and demand. Pricing and reimbursement were the only challenge.”

In the US, Klima added, payers “recognize the value of a one-time therapy. We’re confident in our ability to price it and secure reimbursement.”

Both TDT (1,500 patients) and CALD (50 patients) are extremely rare diseases, but sickle cell disease (SCD) presents a much bigger commercial opportunity: 20,000 addressable patients out of about 100,000 who have the disease in the US, Obenshain noted.

“Within that 100,000, there’s pervasive dissatisfaction with current treatments,” the CEO observed.

Bluebird saw the first clinical hold on its SCD gene therapy last summer. It’s assessing the impact of the current hold and, pending receipt of feedback from the FDA, the company plans to submit a BLA for the treatment, called lovi-cel, early next year.

As the biotech anticipates a mid-2022 US debut of beti-cel and eli-cel, its approach will underpin the potential launch of lovi-cel for SCD in 2023, Obenshain said. 

That’s because most (67%) of beta-thalassemia prescribers also treat SCD patients, 100% of the beta-thalassemia treatment centers will become SCD treatment centers, and 90% of SCD patients live within a 90-mile radius of a treatment center, he said.