Pfizer confirms a patient death in a long-term Hympavzi trial but sees no expected impact on overall drug safety.

Five years after treatment, patients in the HOPE-B study largely still enjoy the benefit they obtained from gene therapy for hemophilia B: bleeding reduction with little to no need for factor replacement. The end-of-study analysis, reported at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition1 and published in Blood,2 closes out follow-up for the phase 3 trial of etranacogene dezaparvovec in adults with severe or moderately severe hemophilia B. The study was led by Steven Pipe, MD, at the University of Michigan and was designed to answer a question that has hovered over the field since the earliest gene therapy readouts: Does the effect last?

Market Overview and Insights The Hemophilia Gene Therapy Market is experiencing unprecedented growth driven by rising prevalence of hemophilia, technological advancements in gene therapy, and increased investments in rare disease research. Valued at USD 2.62 billion in 2024, the market is projected to reach USD 3.07 billion in 2025 and expand significantly to USD 15.22 billion by 2035. The surge reflects growing adoption of gene-based treatments and enhanced awareness of hemophilia management strategies among healthcare providers and patients.

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