Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month. Vertex and CRISPR Therapeutics are up first with a Dec. 8 decision deadline on their exa-cel gene therapy, followed by bluebird bio's lovo-cel on Dec. 20.
One potential advantage for bluebird, giving this timing, could be a chance to view its rival's pricing before deciding itself. But, on Tuesday, bluebird said Vertex’s price tag will not factor into how the Massachusetts company will price its treatment.
“We’re gonna run the same process on our price for lovo-cel that we ran with Zynteglo and Skysona, obviously different dynamics in sickle cell disease,” Tom Klima, bluebird’s chief commercial and operating officer, said during the company’s third-quarter earnings call. “We believe that we are leaders in establishing a value and setting price in these therapies and we’ll continue to price the way that we price.”
While Zynteglo, bluebird’s treatment for beta thalassemia, costs $2.8 million, Skysona, its therapy for cerebral adrenoleukodystrophy, is priced at $3 million. The gene therapies each became the most expensive drug in U.S. history when they were approved a month apart in 2022. A few months later, CSL took the throne with Hemgenix, its $3.5 million gene therapy for hemophilia B.
Earlier this year, cost watchdog the Institute for Clinical and Economic Review (ICER) assessed that both Vertex and bluebird’s gene therapies would meet cost-effectiveness thresholds if priced between $1.35 million and $2.05 million for a single dose.
In its report, ICER said that both gene therapies had “important uncertainties” with respect to their long-term safety and efficacy, and especially so with exa-cel, which would be the first approved gene therapy using CRISPR’s gene editing technology, which captured a Nobel Prize in 2020.
Both companies have been quick to point out that transfusions needed to get SCD patients through life can run between $4 million and $6 million.
Vertex is on track for an approval of exa-cel in Europe and has taken steps to avoid bluebird’s experience there. In 2021, the company decided to close its business in Europe after failing to come to reimbursement agreements with local governments.
As for potential competition with Vertex in the U.S., bluebird CEO Andrew Obenshain pointed out Tuesday that the FDA did not require lovo-cel to pass the muster with an advisory committee assessment. Exa-cel passed an advisory committee examination last week.
“We believe the fact that an FDA advisory committee wasn’t requested for lovo-cel is a testament to the depth and breadth of the lovo-cel data, significantly more than any other gene therapy program for sickle cell disease,” Obenshain said.
The CEO also stressed the experience bluebird has had with its previous gene therapies. He added that the company will present nine- and five-year follow-up data next month on patients who have received Zynteglo and lovo-cel.
As for the third quarter, Bluebird reported a net loss of $71.7 million. Its revenue reached $12.3 million for the quarter versus $6.8 million in the previous quarter.
The company said that in early 2024 it will provide an update on its cash runway guidance, which will be enhanced by a potential $103 million sale of a priority review voucher (PRV) to an undisclosed company. Bluebird revealed the agreement at the end of last month. The PRV will be gained upon the approval of lovo-cel.
The company also touted its launch of Zynteglo and Skysona, saying that 22 patients have undergone cell collections, the first step in receiving the treatments. Sixteen beta thalassemia patients have begun the process versus 11 at the end of the second quarter. The 22 patient starts will add up to $63 million in revenue, bluebird said.