Exton, PA, Aug. 13, 2026 (GLOBE NEWSWIRE) -- The hemophilia gene therapy market is contracting commercially, but physician and patient interest in the modality has not disappeared. Recent commercial developments, including the discontinuation of BEQVEZ® (Pfizer), withdrawal of ROCTAVIAN® (BioMarin), and supply challenges for HEMGENIX® (CSL Behring), have narrowed the available gene therapy landscape. Yet new research from Spherix Global Insights suggests expectations for what gene therapy must deliver are becoming considerably more demanding.
According to the latest Market Dynamix™: Hemophilia (EU5) 2026 study, based on a survey of 160 hematologists across France, Germany, Italy, Spain, and the United Kingdom, gene therapy continues to dominate physicians’ expectations for what will shape hemophilia care over the next year. In an open-ended assessment of the developments most likely to affect how they treat or manage patients, the four most frequently cited responses were all gene therapy-related, spanning gene therapy success in patients (22%), withdrawal of ROCTAVIAN (17%), long-term safety of gene therapy (11%), and advancements in gene therapy (11%).
At the same time, the evidence threshold for referral has risen sharply. In 2025, 66% of EU4+UK physicians reported needing longer-term efficacy data before referring a patient with hemophilia A for gene therapy. That figure has climbed 23 percentage points to 89% in 2026, suggesting that as experience with the first generation of commercial gene therapies accumulates, physicians are placing greater emphasis on evidence of durable benefit before acting on their interest. Patient-level research reveals a similar tension between continued potential and immediate pursuit. In Spherix’s Patient Chart Dynamix™: Hemophilia (US) 2026 study, 37% of 169 patients with hemophilia A or B were considered good candidates for gene therapy by their treating physicians but were not currently considering pursuing it, suggesting that candidacy does not necessarily translate into immediate uptake and leaving a notable population for whom gene therapy could remain a future consideration. Spherix’s Patient Voice Dynamix™: Hemophilia research further suggests that despite lingering concerns, gene therapy continues to hold meaningful appeal for some patients. Among participating patients, 36% of those with hemophilia A (n=53) and 24% of those with hemophilia B (n=29) expressed strong interest in gene therapy. Qualitatively, patients described a cure as potentially “life-changing” and continued to express a desire for a “hemophilia-free mind,” reflecting patient aspirations for a treatment that could reduce the physical and psychological burden of the disease. At the same time, potential side effects and limited real-world experience remain important barriers to pursuing gene therapy.
Taken together, the findings suggest that recent commercial setbacks have not extinguished interest in gene therapy. Rather, they appear to be reshaping expectations for what a successful gene therapy must demonstrate. For physicians, longer-term evidence of efficacy and safety is increasingly important, while patients continue to weigh the potential for transformative benefit against uncertainty surrounding side effects and real-world experience. Those expectations may become even more consequential as the broader hemophilia landscape evolves. With highly effective and increasingly convenient alternatives expanding the options available to patients, gene therapy must demonstrate not simply that it can work, but that the durability and magnitude of its long-term benefits warrant choosing it over established and emerging alternatives.
The findings point to a hemophilia gene therapy market at an inflection point: physician and patient interest persists, but increasingly, the future of the modality may depend on whether gene therapy can deliver the durable efficacy, safety, and real-world evidence needed to meet a rising bar for adoption.
Spherix will continue monitoring these dynamics through its integrated hemophilia research portfolio, including Patient Voice Dynamix™, Patient Chart Dynamix™, and Market Dynamix™. The forthcoming Market Dynamix™: Hemophilia (US) 2026 study will provide an additional view into whether similar attitudes are emerging among U.S. hematologists and how the evolving treatment landscape is influencing expectations for gene therapy.
About Market Dynamix™
Market Dynamix™ is an independent, data-driven service focused on understanding the evolving dynamics of specialty markets poised for disruption. Leveraging quantitative and qualitative research, the service evaluates current treatment approaches, unmet needs, and likely impact of pipeline agents over a three-to-five-year horizon.
About Patient Chart Dynamix™
Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the “why” behind treatment decisions, include year-over-year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal and demographic data to highlight differences between stated and actual treatment patterns.
About Patient Voice Dynamix™
Patient Voice Dynamix™ is an independent, data-driven service designed to capture patients lived experiences as they navigate diagnosis, treatment, and long-term disease management. By integrating quantitative surveys, qualitative interviews, and validated patient-reported outcome measures, the service provides a comprehensive view of the burden of illness and unmet need from the patient perspective.
About Spherix Global Insights
Spherix Global Insights is a leading independent provider of market intelligence and advisory services for specialty pharmaceutical and biotech markets. By combining physician, patient, and payer perspectives into a single integrated view, Spherix helps brand teams, market access stakeholders, and investors understand how a specialty market actually behaves, not how any one stakeholder describes it. The firm operates eight dedicated therapeutic franchises – Central Nervous System, Dermatology, Gastroenterology, Hematology, Nephrology, Oncology, Ophthalmology, and Rheumatology – alongside a fully dedicated Market Access team. Each franchise is led by hyper-focused analysts and researchers who track these specialty areas full-time, drawing on independently curated communities of vetted practicing specialists, KOLs, patients recruited through treating physicians and advocacy partners, and medical and pharmacy directors at commercial and government payers. Spherix delivers quarterly trending, launch tracking, chart audits, KOL synthesis, patient experience research, and payer and market access intelligence that support confident, strategic decision-making across the pharma lifecycle. Trusted by 19 of the top 20 pharmaceutical and biotech organizations, Spherix has been a go-to resource for more than a decade for leading brands, market access stakeholders, industry media outlets, financial analysts, professional organizations, and patient advocacy groups seeking an unbiased, holistic view of specialty markets.
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NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insights’ analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.
Sarah Hendry, Hematology/Oncology Franchise Head Spherix Global Insights 484-879-4284 sarah.hendry@spherixglobalinsights.com
