More than a year after launching the first-ever gene therapy for hemophilia B, CSL Behring revealed that it’s treated more than 12 patients so far and that it’s taken longer than expected to make inroads in the US.
The update, which came as part of CSL’s latest earnings report, adds another case study for the slow uptake of gene therapies for hemophilia, which have been attributed to hefty price tags, existing treatment options and patient hesitation. It’s still unclear if Pfizer has dosed any patient with its second-to-market hemophilia B treatment, Beqvez. And BioMarin, which markets a gene therapy for hemophilia A, has had to reel back its ambitions.
CSL’s therapy, Hemgenix, carries a US list price of $3.5 million.
When asked by an analyst about CSL’s expectations for patient numbers in 2025, CEO Paul McKenzie said, “We had 12 in 2024, handful or so in July.” The company’s fiscal year runs from July to June.
“But again, the US healthcare system is quite fragmented, and it’s taken us longer than we had planned to penetrate that,” he said on the call, according to an AlphaSense transcript. Specifically, the transition from referrals to administration has been “slower than we anticipated,” he said.
The Australian conglomerate secured FDA approval in late 2022 and dosed the first commercial patient in June 2023.
In Europe, McKenzie said, reimbursement contract negotiations “have progressed well.” While he declined to provide a forecast for patient numbers in 2025, the company is hoping to increase uptake.
CSL did not break down sales numbers of Hemgenix in its report. But earlier in the month, uniQure — the biotech from which CSL had licensed Hemgenix — reported that it received $3.1 million of royalty revenue in the first six months of 2024. UniQure CEO Matt Kapusta previously told Endpoints News that royalty tiers range from 16% to low 20% (the biotech has also sold a chunk of its royalty rights).