Incorporation of gene therapies, such as Beqvez (Pfizer),
Hemgenix (CSL Behring) and Roctavian (BioMarin), and potential future approvals
of fitusiran (Sanofi) and Mim8/denecimig (Novo Nordisk) will further drive the
increasingly dynamic hemophilia markets.
Exton, PA – October 14, 2024 – Hemophilia, a genetic disorder that impairs blood clotting, affects thousands of people worldwide. The treatment landscape is rapidly evolving, fueled by groundbreaking therapies and a shift toward personalized medicine. Recent research from Spherix Global Insights highlights these developments, exploring market dynamics amid the arrival of new genetic therapies as well as last week’s approval of Pfizer’s Hympavzi.
This summer, Spherix Global Insights conducted research with 50 US hematologists as part of the recently published Market Dynamix™: Hemophilia service, uncovering the current dynamics and future outlook of both the Hemophilia A and B markets. The study’s findings indicate that hematologists consider hemophilia A and B among the most challenging conditions to manage, with an estimated one-third of their patients not receiving optimal care.
In hemophilia A, many hematologists reported changes in patient management over the past year, with the most notable being an increased use of both Hemlibra and Altuviiio. Current market dynamics show that Hemlibra holds a dominant position, with high levels of satisfaction among prescribers. The therapy is expected to see continued growth in usage over the next six months, significantly outperforming traditional factor VIII treatments and solidifying its status as the leading choice for hemophilia A management.
Amid the current and growing use of Hemlibra, Altuviiio – approved in early 2023 – is rapidly emerging as another key treatment option for hemophilia A patients. While current satisfaction levels are slightly lower compared to Hemlibra, Altuviiio significantly surpasses satisfaction with all types of factor VIII products and physicians expect its use to continue to grow. As one hematologist shared, “I recently started using Altuviiio. I like the weekly dosing. I like the low bleeding rate. I love their patient support program. I think that out of all the factors that I have used, Hemlibra included, Altuviiio is the best.”
Although Roctavian (BioMarin) received approval in 2023 for hemophilia A, prescribers have been notably slow to embrace the gene therapy. Several factors have hindered adoption, led by limited perceived patient eligibility and prescriber comfort. Additionally, a lack of patient interest and concerns about the high cost, as well as hesitations around the therapy’s mechanism of action, insufficient data, and safety profile, contribute to the cautious approach.
In contrast, Spherix also explores the potential of gene therapy for hemophilia B patients, where prescriber sentiment appears more favorable. As factor therapy has historically been the standard of care and only option for these patients, physicians appear more open to considering or even referring patients for CSL Behring’s Hemgenix or Pfizer’s recently approved Beqvez. Although hematologists acknowledge that gene therapy adoption has been gradual, nearly all believe it will ultimately transform treatment paradigms for hemophilia B patients.
Spherix is monitoring the launch of Beqvez through its Launch Dynamix™: Beqvez series, which began its 18-months of quarterly tracking in June. Insights include physician awareness, expected patient candidacy, and perceptions of access. While over half of physicians believe the potential benefits of gene therapies outweigh long-term risks, concerns about cost remain a significant barrier to widespread adoption.
The future of hemophilia A and B treatments are likely continue to change with the recent approval of Pfizer’s Hympavzi (marstacimab) on October 11, 2024. Hympavzi was approved for patients 12 years and older with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors.
Pre-approval sentiment among hematologists as part of the recent Spherix report indicates that this new addition is particularly meaningful for hemophilia B patients, where factor therapy has traditionally been the standard of care. Notably, reactions to marstacimab information as part of the survey highlighted interest in its once-weekly subcutaneous administration. As one hematologist remarked, “I’m very interested in the autoinjector (marstacimab). I think from the standpoint of ease of administration, reduced frequency of administration, if it really holds up in terms of safety and efficacy. I think that would absolutely be a step forward and meeting an unmet need for the hemophilia B patients.”
Spherix will be tracking the impact of Hympavzi on both hemophilia markets for the first eighteen months of commercial availability via the Launch Dynamix™ service. As the industry moves toward improved efficacy and patient experiences, it stands on the brink of a new era in hemophilia management. Spherix remains committed to delivering independent, up-to-date data that helps healthcare providers navigate the evolving landscape of hemophilia treatments.
Market Dynamix™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years. Insights highlight market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents.
Launch Dynamix™ is an independent service providing monthly benchmarking of newly launched products for the first eighteen months of commercial availability, augmented by a quarterly deep dive into promotional activity, barriers to uptake, and patient types gravitating to the launch brand.
About Spherix Global Insights
Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle.
The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.
As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth.
To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn.
For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com
Spherix Global Insights Contacts
Tucker Hurtado, Hematology Franchise Launch Head
Tucker.Hurtado@spherixglobalinsights.com
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 Insight’s analysis and do not imply a relationship with or endorse.
