Bluebird bio lays gene therapy launch groundwork as sickle cell application nears

While commercial revenue has yet to materialize in bluebird bio’s earnings report, the company is flying high on the launches of its two new gene therapies Zynteglo and Skysona. 

After last year’s back-to-back approvals, Zynteglo for beta thalassemia and Skysona for cerebral adrenoleukodystrophy are carving a commercial path in the U.S., bluebird executives said on a conference call.

So far, five patients have started the cell collection process for Zynteglo, versus two for Skysona. March 16, the first commercial infusion of Skysona took place at Boston Children’s Hospital, bluebird’s chief commercial officer Tom Klima said Wednesday.

Thanks to those launches, plus the recent sale of a pair of FDA priority review vouchers, the company holds the “strongest financial position bluebird has had since we emerged as a dedicated gene therapy company in November of 2021,” new Chief Financial Officer Chris Krawtschuk added.

Still, it’s “premature” to provide revenue and profit guidance around the company’s current launches, Krawtschuk said. Bluebird expects to report its first Zynteglo and Skysona sales when it posts first-quarter results in May, he noted.

Bluebird’s commercial strategy around its pricey gene therapies rests on three pillars, Klima explained. Those are: education for families and patients; the company’s network of treatment centers; and lastly, access and reimbursement.

Skysona costs $3 million before discounts, while Zynteglo has been priced slightly lower at $2.8 million. Both therapies are one-time treatments.

Meanwhile, since launching Zynteglo and Skysona, bluebird has seen success across all three areas, with the “momentum continuing to build,” Klima said.

The commercial chief flagged “significant patient demand and uptake” for Zynteglo specifically. To meet that growing demand, bluebird is forging ahead on plans to expand manufacturing capacity, Klima added.

As for reimbursement, bluebird says prior authorization for its gene therapies has been taking only two weeks, and to date, the company hasn’t seen any coverage denials.

On treatment centers—which both form part of the bluebird supply chain and serve as “ambassadors” for the company’s therapies—bluebird has activated 12 so far, including both adult and pediatric centers, Klima said.

Some 30 additional treatment centers are now in the onboarding stage, the CCO said. That puts the company on track to scale to between 40 and 50 by the end of the year.

Aside from Skysona and Zynteglo, bluebird is plotting the prospective launch of a third gene therapy, lovo-cel, in sickle cell disease. The company figures its current gene therapy rollouts will help lay the groundwork ahead of time.

For one, the treating physicians would be the same, as would bluebird’s network of treatment centers. Finally, Klima contends bluebird is “leading the way” on pricing and access for commercial gene therapies.

With lovo-cel, the company will “likely miss” its first-quarter submission goal at the U.S. Food and Drug Administration, bluebird’s CEO Andrew Obenshain said.

Still, the application delay shouldn’t affect bluebird’s launch plans for lovo-cel, the CEO stressed.

“At this time, we are not forecasting any change to our commercial plans for lovo-cel and are continuing to anticipate an early 2024 launch,” Obenshain explained.

The application delay comes as bluebird awaits FDA feedback around updated chemistry, manufacturing and controls data.

Back in December, bluebird handed the FDA a “snapshot of comparability data,” Obenshain said. The regulator came back in February with feedback and questions, requesting responses prior to the submission of bluebird’s application for approval.

Bluebird provided that updated information in March.