How recombinant therapies are reshaping bleeding disorder treatment for children in 2025

In a pivotal regulatory milestone for pediatric hematology, the U.S. Food and Drug Administration (FDA) approved the expanded use of Takeda Pharmaceutical Company Limited’s recombinant therapy Vonvendi for children with von Willebrand disease (VWD) in September 2025. This decision allows Vonvendi to be used not only in adult patients across all VWD types but also in pediatric populations for on-demand treatment of bleeding episodes and perioperative management.

This moment matters far beyond von Willebrand disease alone. It underscores a broader transformation in bleeding disorder management—one where recombinant therapies are rapidly supplanting traditional plasma-derived treatments. Pediatric hematology, long constrained by limited treatment options and safety trade-offs, is now entering a new era of safer, longer-acting, and biologically engineered clotting factor replacements.

Vonvendi, already notable as the only FDA-approved recombinant von Willebrand factor (VWF) product in the U.S., now becomes the first recombinant VWF therapy approved for use in pediatric patients. This breaks a decades-long dependency on plasma-based VWF therapies for children and firmly establishes recombinant platforms as the new standard of care.

How are recombinant clotting factor therapies disrupting traditional plasma-derived models in 2025?

Recombinant therapies like Vonvendi offer a major clinical and commercial advantage over their plasma-derived counterparts: safety, reliability, and the ability to manufacture at scale without dependence on human donors. In pediatric settings where viral transmission risk, immunogenicity, and supply variability can jeopardize outcomes, this matters profoundly.

Vonvendi’s expanded pediatric indication is part of a broader trend where recombinant biologics are taking over frontline roles in treating hemophilia A, hemophilia B, and von Willebrand disease. In February 2023, Sanofi’s Altuviiio (efanesoctocog alfa), a next-generation recombinant Factor VIII with extended half-life, was approved for both adults and children with hemophilia A. With once-weekly dosing and strong bleed prevention outcomes, Altuviiio is now considered a top-tier option for pediatric patients who previously required more frequent infusions.

Meanwhile, Takeda continues to invest in its rare disease portfolio, acquired during its merger with Shire, with a focus on biologics that can scale globally. CSL Behring, another major player, is pursuing innovation in Factor IX and other recombinant products, while expanding its reach through strategic acquisitions and late-stage development programs in North America, Europe, and Asia-Pacific.

These shifts are no longer incremental—they represent a structural change in how bleeding disorders are managed, particularly in younger populations that demand high safety and low treatment burden.

What are the most promising recombinant and non-replacement therapies now shaping pediatric hemophilia care?

Beyond traditional recombinant clotting factors, the pipeline for bleeding disorder therapeutics now includes rebalancing agents, RNA interference therapies, and even gene therapies. Each targets different patient segments, but many converge around pediatric care as the next frontier of innovation.

Sanofi’s Qfitlia (fitusiran), approved in early 2025, represents a major leap in this space. Indicated for individuals aged 12 and up with hemophilia A or B—with or without inhibitors—Qfitlia works by lowering antithrombin levels, effectively rebalancing clotting. Administered subcutaneously every two months, it has been shown to reduce annual bleeding rates by up to 90%. For adolescent and pediatric patients transitioning from infusion-based care, this represents a radical improvement in convenience and quality of life.

CSL Behring’s Hemgenix, a gene therapy for hemophilia B, and Pfizer’s Beqvez (fidanacogene elaparvovec) are pushing the boundaries even further by offering one-time treatments that may provide years of bleed control. However, their use in pediatric populations remains limited due to safety, durability, and ethical considerations. Regulatory guidance is still evolving on the appropriateness of gene therapy in children, especially where long-term data is lacking.

In hemophilia A, the continued dominance of recombinant Factor VIII products—particularly those with extended half-lives like Altuviiio—reinforces their importance as bridge therapies. They provide a clinically proven, scalable option while newer platforms mature and earn broader reimbursement coverage.

Which patient segments remain underserved despite these advancements in biologic and recombinant therapies?

Despite clinical progress, access to recombinant and biologic therapies for bleeding disorders remains highly uneven—especially for pediatric populations in underfunded healthcare systems or emerging markets.

Many families still face insurance-related delays, prior authorization hurdles, or state Medicaid exclusions for newer biologics. In low- and middle-income countries, plasma-derived products remain the only accessible option due to lower costs and established supply chains. This has created a two-tiered system where the safety and convenience of recombinant therapies are available primarily to patients in high-income settings.

Inhibitor-positive patients also represent a challenging subsegment. While non-replacement therapies such as emicizumab (Hemlibra) and fitusiran offer pathways to reduce bleed frequency in these individuals, not all are eligible or responsive. Moreover, for the youngest patients—those under two years of age—limited data still restricts widespread adoption of biologic therapies.

Rural patients in both developed and developing countries often lack access to comprehensive hemophilia treatment centers, which limits their ability to receive newer therapies even if prescribed. Telemedicine and home-infusion services have helped bridge some of these gaps but are not universally available.

Pediatric hematologists and advocacy groups are calling for expanded formulary inclusion, more representative pediatric trials, and updated treatment guidelines to keep pace with the innovation curve. Without policy alignment, these breakthroughs risk deepening rather than closing the access gap.

How is institutional sentiment shaping the future of bleeding disorder innovation and commercialization?

Analysts broadly agree that the pediatric expansion of Vonvendi, combined with the rollout of Qfitlia and Altuviiio, signals strong momentum in the bleeding disorder therapeutics market. However, they caution that payer pressure and margin dilution could test the commercial durability of these biologics, especially as gene therapies attempt to claim long-term share.

In Takeda’s case, the Vonvendi approval is expected to slightly strengthen its rare disease portfolio performance, though the drug remains a niche product in revenue terms. For Sanofi, Altuviiio and Qfitlia are strategically important assets meant to offset maturing franchises in other therapeutic areas. CSL Behring’s position remains strong in hemophilia B and may gain further edge as Hemgenix gains reimbursement traction.

The broader investor outlook suggests cautious optimism. Institutional investors are closely watching payer adoption patterns and real-world evidence studies. Long-duration biologics and subcutaneous delivery platforms are attracting the most attention due to their favorable cost-of-care profiles over time.

From a regulatory perspective, the FDA’s Center for Biologics Evaluation and Research (CBER) has signaled its intention to reduce pediatric-adult approval gaps. The recent Vonvendi approval reflects this mindset, suggesting that other pediatric label expansions may arrive sooner than expected—particularly for recombinant and non-replacement biologics with proven safety profiles.

What does the next decade of pediatric bleeding disorder treatment look like?

As biologic and recombinant innovations reshape the pediatric treatment landscape, several trends are poised to define the coming years. First, the global push toward eliminating viral risk from donor plasma will further elevate recombinant therapy adoption. Second, subcutaneous and extended half-life options will gain preference in home care and early childhood settings. Third, payer models will evolve—slowly—to accommodate newer high-cost therapies if long-term value can be demonstrated.

Most importantly, pediatric patients and their families stand to benefit from a care model that prioritizes safety, adherence, and flexibility. The days of rigid infusion schedules, frequent bleeds, and donor plasma shortages may finally be numbered.

While gaps remain, especially in global access and inhibitor management, the bleeding disorder space is now firmly in its biologic era—and the pace of innovation is only accelerating.