Bluebird’s Newly Approved SCD Gene Therapy Faces Rough Road with Hefty Price Tag, Boxed Warning

2023-12-11

上市批准优先审批并购基因疗法临床结果

Pictured: Illustration of sickle cells in the bloodstream/iStock, Artur Plawgo Despite winning FDA approval on Friday for its sickle cell disease gene therapy Lyfgenia (lovotibeglogene autotemcel), bluebird bio’s investors were less than bullish on the company. The Massachusetts-based biotech closed the day down nearly 35%. Much of the negative share reaction could have been driven by Lyfgenia’s list price. Shortly after its regulatory win, bluebird announced that the gene therapy would have a wholesale acquisition cost of $3.1 million. In its news announcement, bluebird said it arrived at this price “in recognition of the value the therapy may deliver through robust and sustained clinical benefits and the estimated lifetime impact,” including savings from lower healthcare utilization, patients’ future earnings and their life opportunities. By comparison, Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), which was approved on the same day as Lyfgenia, will go for $2.2 million. Casgevy is a CRISPR-based gene edited therapy, is also indicated for sickle cell disease (SCD) and will likely be a strong competitor for Lyfgenia. Over the weekend, at the 65th Annual Meeting & Exposition of the American Society of Hematology, experts were cautiously optimistic about what the dual approvals of Casgevy and Lyfgenia might mean for patients, STAT News reported. However, many expressed their confusion, if not shock, at bluebird’s pricing. “For two therapies which do pretty much the same thing and are targeting exactly the same population, a price tag that’s $1 million apart, I think, is a major deciding factor for many patients and providers and even insurance programs,” Akshay Sharma, who treats pediatric SCD patients at St. Jude Children’s Research Hospital, told STAT. “It’s baffling to me why Bluebird Bio priced their therapy so high — or maybe Vertex priced theirs low, I don’t know,” Sharma said. Also potentially contributing to bluebird’s drop on Friday is Lyfgenia’s boxed warning, which flags instances of hematologic malignancies in patients who had been treated with the gene therapy. Additionally, bluebird failed to secure an FDA priority review voucher, which it had planned to sell to Novartis for $103 million in a bid to improve its cash position. In its third-quarter financial report, bluebird said that it had $227 million in cash, cash equivalents, marketable securities and restricted cash, which would be enough to support its operations only through the second quarter of 2024. Seemingly lost in the discussion about Lyfgenia’s pricing are new long-term follow-up data, which bluebird posted on Saturday. Over five years of follow-up, 94% patients treated with the gene therapy achieved complete resolution of severe vaso-occlusive events, while 88.2% saw complete resolution of all vaso-occlusive events. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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