Bluebird signs major coverage deal for sickle cell gene therapy Lyfgenia, easing some price concerns

Despite the high list price of Lyfgenia, bluebird has signed a large reimbursement deal for the sickle cell disease (SCD) gene therapy less than a week after its FDA approval.

The outcomes-based agreement bluebird reached for Lyfgenia is with an organization that covers about 100 million people in the U.S., the gene therapy specialist said in a securities filing (PDF) Thursday. That’s nearly a third of the country’s entire population, which is estimated at 335 million.

For Lyfgenia, bluebird is offering an outcomes-based framework that tracks treatment results for three years and offers rebates if a patient is hospitalized because of a painful episode of vaso-occlusion, which is a common complication of SCD.

The deal marks Lyfgenia’s first coverage agreement. A bluebird spokesperson declined to disclose more details of the contract for competitive reasons.

Meanwhile, the speed of this deal underscores that payers recognize the value of the therapy and that they’re ready to support access, bluebird’s spokesperson said.

The deal could ease some concerns over the $3.1 million wholesale acquisition cost bluebird placed on Lyfgenia. That list price is about 40% higher than Vertex and CRISPR Therapeutics’ rival SCD gene therapy Casgevy. The two treatments were simultaneously approved by the FDA on Friday, Dec. 8.

When those price tags came out last week, analysts suggested that bluebird would face payer pushback, leading to lower adoption compared with its competitor.

In response, bluebird’s chief commercial & operating officer, Tom Klima, said the company was confident in its value-based approach in deciding Lyfgenia’s price. The company considered the positive impact of a potentially curative therapy for a devastating disease on the healthcare system and patients, he said.

“We took a very heavy feedback approach when it comes to our outcomes-based agreement with payers,” Klima said during an investor call Friday. “And we expect that the outcomes-based agreement would be a key part of the strategy to ensure rapid access and access consistent with our label.”

Since then, researchers offered an update of Lyfgenia’s clinical data at the American Society of Hematology annual meeting over the weekend. Among 34 evaluable patients, 32 (94) had complete resolution of severe vaso-occlusive events and 30 (88.2%) had no events whatsoever after a median follow-up of 35.8 months.

Bluebird is in advanced discussions with other large commercial payers and more than 15 Medicaid agencies, which together cover 80% of the U.S.’ SCD population, the company said Thursday.

The bluebird spokesperson couldn’t offer a timeline as to when the company could finish those talks, pointing out that each payer has its internal pace and that medical exceptions are common practice before a formal agreement.

Despite the gap in list prices, a J.P. Morgan team led by Eric Joseph said it expects that bluebird and Vertex will offer similar post-discount net prices through contracting with treatment centers.

Bluebird expects cell collection for the first Lyfgenia patients to begin in the first quarter of 2024. The company expects 85 to 105 cell collections in 2024 across its three commercial products, including beta thalassemia therapy Zynteglo and cerebral adrenoleukodystrophy treatment Skysona, it said Thursday. All three gene therapies carry list prices of around $3 million.