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Menu BioSpace logo Show Search Bluebird’s Newly Approved SCD Gene Therapy Faces Rough Road with Hefty Price Tag, Boxed Warning December 11, 2023 | 2 min read | Tristan Manalac Twitter LinkedIn Facebook Email Print Pictured: Illustration showing sickle cells in the Pictured: Illustration showing sickle cells in the These factors, along with bluebird bio’s failure to receive a priority review voucher, all hurt the company’s stock price following Lyfgenia’s approval. 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.

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When I was a kid, I used to watch other kids play video games more than I ever played them. I wasn’t very skilled or cool under pressure. I didn’t own a Sega console, so I never got high scores on Sonic The Hedgehog. I did, however, admire the steadfastness of the premise. He speeds through obstacles, collecting seemingly arbitrary rings. At its best, the game encouraged us to become pure momentum, which in microcosm is the joy of video games in general — total escape from reality at the speed of sound with the pleasing melody of his collecting rings the only reward you needed. Fittingly, I didn’t get invited to see the new film adaptation ahead of time as press. I had to walk into a theatre looking hungover and unwashed at 11:00 a.m. to stare at a man who’d been awake for hours and say, “one for the 10:50 a.m. Sonic The Hedgehog.” Hearing yourself say it, in your own 30-year-old voice … it stuns you a little. The man behind the glass smiled through his evident discomfort in helping me achieve this Sisyphean task. “Just to let you know, there’s a school group in there …

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