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
