Global Hemophilia Market Size, Share & Forecast 2026-2033

Overview

The global hemophilia market size was valued at USD 13.80 billion in 2025 and is projected to reach USD 22.84 billion by 2033, growing at a CAGR of 6.5% during the forecast period 2026-2033. Hemophilia is a rare, typically inherited bleeding disorder in which the blood lacks sufficient quantities of a clotting protein, most commonly factor VIII in hemophilia A or factor IX in hemophilia B, leading to prolonged or spontaneous bleeding, including repeated bleeding into joints that can cause chronic joint disease if left inadequately managed.

The World Federation of Hemophilia's global survey work has identified more than 347,000 people with hemophilia across over 120 countries, while researchers estimate the true global population living with the disorder, including undiagnosed cases, may exceed one million, leaving substantial room for diagnosis-driven demand growth, particularly across Asia, Africa, and Latin America where access to confirmatory testing remains uneven.

Demand in established markets is increasingly shaped by a product cycle that has accelerated sharply since 2024, with new approvals and label expansions arriving across factor, non-factor, and gene therapy classes. North America accounted for an estimated 47% of global hemophilia market revenue in 2025, the largest of any region, supported by an established hemophilia treatment center network and favorable reimbursement, while Asia Pacific is the fastest-growing region at an estimated CAGR of approximately 7% as diagnosis rates rise across China and India.

Drivers

Unusually Active Product Approval Cycle Across Factor, Non-Factor, and Gene Therapies

The single most direct driver of hemophilia market growth has been the sheer density of new product approvals and label expansions across multiple treatment classes. The FDA approved Pfizer's BEQVEZ (fidanacogene elaparvovec), the second approved gene therapy for hemophilia B, in April 2024 based on Phase 3 BENEGENE-2 data, and followed in October 2024 with approval of Pfizer's Hympavzi (marstacimab), a once-weekly subcutaneous anti-tissue factor pathway inhibitor therapy for patients without inhibitors. Novo Nordisk's Alhemo (concizumab), a monoclonal antibody therapy for patients with inhibitors, was approved in December 2024, and Sanofi's Qfitlia (fitusiran), which works by lowering antithrombin rather than replacing a clotting factor, followed in March 2025. Each approval has expanded the addressable patient population and given physicians more dosing and administration options, sustaining replacement and switching demand across the existing patient base.

Label expansions are reinforcing this pattern as much as new approvals. Sanofi secured an updated U.S. label for ALTUVIIIO in May 2024 incorporating full results from the Phase 3 XTEND-Kids pediatric study, followed by European Commission approval of the same therapy, marketed as ALTUVOCT, in June 2024. Most recently, in June 2026, the FDA approved an expanded indication for Hympavzi covering patients aged 12 and older with inhibitors and pediatric patients aged 6 to 11, regardless of inhibitor status — broadening a single product's addressable population well beyond its original 2024 approval and illustrating how incremental label expansion, not just new molecule approval, is sustaining commercial momentum across this market.

Long-Term Gene Therapy Outcomes Data is Building Physician and Payer Confidence

Sustained, multi-year clinical follow-up data is becoming an important driver in its own right, particularly for gene therapies whose commercial uptake depends on confidence in durability. CSL Behring presented four-year HOPE-B trial data for HEMGENIX, its one-time gene therapy for adults with hemophilia B, at the European Association for Haemophilia and Allied Disorders congress in February 2025, and followed with five-year, end-of-study HOPE-B results presented at the American Society of Hematology annual meeting and published in the New England Journal of Medicine in December 2025. As gene therapy durability data accumulates across additional years of follow-up, it directly addresses one of the central commercial questions facing curative-intent hemophilia treatments and is likely to influence both prescribing confidence and payer willingness to cover high upfront treatment costs.

Restraint

Gene Therapy Commercial Uptake Has Lagged Expectations Despite Strong Clinical Data

Despite a wave of regulatory approvals, real-world commercial uptake of hemophilia gene therapies has been markedly slower than initially anticipated, representing a meaningful restraint on the segment's near-term revenue contribution. Pfizer terminated its gene therapy partnership with Sangamo Therapeutics, which had been developing a hemophilia A gene therapy candidate, in December 2024, and in February 2025 the company announced it would discontinue commercialization of its own hemophilia gene therapy.

BioMarin's Roctavian illustrates the scale of the shortfall: the company recorded just USD 3.5 million in sales in 2023 against an initial guidance range of USD 50-150 million, followed by only USD 26 million in 2024, and announced plans to divest the product in 2025 after sales failed to recover. CSL Behring's HEMGENIX has fared somewhat better but has likewise remained below initial expectations since its approval, reflecting a combination of high one-time treatment costs, patient and physician caution around a still-young curative modality, payer reimbursement complexity, and the continued strong real-world performance of established prophylactic options such as factor concentrates and non-factor therapies. This dynamic illustrates that regulatory approval alone does not guarantee commercial translation in a market where patients have safe, effective, and increasingly convenient alternatives already available.

High Treatment Cost and Diagnostic Infrastructure Gaps Limit Access in Price-Sensitive Markets

The lifelong nature of hemophilia treatment, combined with the high cost of recombinant factor concentrates, extended half-life products, and especially one-time gene therapies priced in the low millions of dollars per patient, continues to restrain market penetration across price-sensitive healthcare systems in parts of Africa, South Asia, and Latin America. Many patients in these regions still rely on on-demand, plasma-derived factor concentrates rather than routine prophylaxis, and in the most resource-constrained settings, on cryoprecipitate or fresh frozen plasma, reflecting persistent gaps in diagnostic infrastructure and specialist hematology capacity rather than a lack of clinical need. These access gaps mean that a substantial share of the global hemophilia population — plausibly numbering in the hundreds of thousands once underdiagnosis is accounted for — remains largely outside the addressable commercial market for newer, higher-cost therapies.

Market Trends & Opportunities in Hemophilia

Non-Factor and Subcutaneous Therapies are Reducing Treatment Burden and Expanding the Addressable Population

A clear trend across recent approvals is the shift toward subcutaneous, less frequently dosed therapies that do not require intravenous factor replacement. Pfizer's Hympavzi, Novo Nordisk's Alhemo, and Sanofi's Qfitlia all work through non-factor mechanisms and subcutaneous administration, reducing the infusion burden that has historically limited adherence, particularly in pediatric patients and those with difficult venous access. This shift is also commercially significant because non-factor therapies can be prescribed regardless of inhibitor status in some cases, widening the prescribing population beyond patients eligible for traditional factor replacement, and creating an opportunity for continued share gains by companies with strong non-factor pipelines.

Pediatric Label Expansion Represents a Distinct, Underserved Commercial Opportunity

Regulatory activity shows a consistent pattern of approvals expanding from adult populations into progressively younger pediatric age bands, visible across both Hympavzi's pediatric label expansion and ALTUVIIIO's 2024 update incorporating full pediatric XTEND-Kids data. Pediatric patients represent a distinct commercial opportunity because earlier initiation of effective prophylaxis is associated with better long-term joint health outcomes, giving manufacturers a clinical rationale to pursue pediatric indications as a deliberate lifecycle-extension strategy rather than a secondary afterthought, while also addressing a segment of the patient population that has historically had fewer subcutaneous or extended-dosing options available to it.

Real-World Gene Therapy Durability Data Will Determine the Pace of Reimbursement and Adoption

The ongoing accumulation of multi-year follow-up data represents a distinct opportunity for gene therapy developers willing to invest in long-term evidence generation, rather than resting on initial trial results alone. Companies that can demonstrate sustained, multi-year efficacy and an absence of late-emerging safety signals are positioned to differentiate on durability confidence, a dimension of growing importance to physicians and payers evaluating curative-intent therapies, rather than competing solely on price or initial efficacy data.

Diagnosis-Driven Demand in Asia and Latin America Remains a Substantial Underexploited Opportunity

Given that the diagnosed global hemophilia population is estimated at little more than a third of the population believed to actually have the disorder, expanding diagnostic capacity in underdiagnosed regions represents a long-term volume opportunity distinct from the innovation-driven growth occurring in North America and Europe. Government-backed rare-disease programs in markets such as China and India are gradually narrowing this diagnosis gap, and companies that invest early in diagnostic partnerships, patient registries, and physician education in these markets are positioned to capture a disproportionate share of newly diagnosed patients as testing capacity expands, ahead of the more innovation-led competition that characterizes the U.S. and European markets.

Segment Analysis

The global hemophilia industry is segmented based on type, treatment type, therapy, distribution channel, and region.

Hemophilia A Leads on the Strength of Substantially Higher Disease Prevalence

Hemophilia A held the largest share of the type segment in 2025, at an estimated 74%, a position rooted directly in epidemiology: hemophilia A, caused by a deficiency of clotting factor VIII, occurs in roughly one in every 5,000 male births, compared with approximately one in 30,000 for hemophilia B's factor IX deficiency. That prevalence gap is reinforced commercially by a deeper roster of approved products for hemophilia A, including recombinant and extended half-life factor VIII concentrates, the bispecific antibody Hemlibra (emicizumab), and the gene therapy Roctavian, giving prescribers and patients more treatment options than are currently available for hemophilia B.

Prophylaxis is the Fastest-Growing Treatment Approach

Prophylaxis is the fastest-growing treatment-type segment, expanding at an estimated CAGR of approximately 6%, as more patients with severe hemophilia transition from reactive, on-demand bleed management to routine preventive dosing. The growing convenience of newer prophylactic options, including Hemlibra's subcutaneous dosing for hemophilia A and the once-weekly or less-frequent subcutaneous regimens offered by Hympavzi, Alhemo, and Qfitlia, is lowering the practical burden historically associated with routine prophylaxis and supporting its continued shift from a specialist-driven recommendation to the default treatment pattern for severe disease.

Factor Replacement Therapy Remains the Leading Therapy Segment

Factor replacement therapy held the largest share of the therapy segment in 2025, at an estimated 61%, remaining the clinical and commercial backbone of hemophilia management. Factor VIII concentrates are used for hemophilia A and factor IX concentrates for hemophilia B, with both plasma-derived and recombinant DNA-based products available; recombinant and extended half-life recombinant concentrates have steadily gained share from older plasma-derived and standard half-life products in markets with established reimbursement, while plasma-derived concentrates remain clinically relevant, particularly in markets with constrained access to recombinant alternatives or for specific patient populations.

Gene Therapy and Monoclonal Antibodies Represent the Fastest-Growing Therapy Category

The gene therapy and monoclonal antibodies category is the fastest-growing segment within the therapy classification, driven by the combination of an active late-stage clinical pipeline and the continued commercial expansion of Roche's Hemlibra (emicizumab), a bispecific antibody that mimics the function of factor VIII. Pfizer's marstacimab (Hympavzi) and Novo Nordisk's Mim8 (denecimig), the latter under FDA review following a September 2025 Biologics License Application submission, extend this category's pipeline depth. Growth in this category is therefore being driven primarily by monoclonal antibody and other non-factor mechanisms rather than gene therapy alone.

Geographical Penetration

North America Hemophilia Market: Treatment Center Infrastructure and Early Product Access Anchor Regional Leadership

An established hemophilia treatment center network and consistently early access to newly approved therapies gave North America the leading regional share of the global hemophilia market in 2025, at an estimated 47% of global revenue. The CDC estimated that approximately 33,000 males were living with diagnosed hemophilia in the United States in 2024, a relatively small but intensively managed patient population that has nonetheless attracted an unusually dense wave of product approvals: the FDA cleared four new or expanded hemophilia therapies between April 2024 and March 2025 alone, namely Pfizer's BEQVEZ and Hympavzi, Novo Nordisk's Alhemo, and Sanofi's Qfitlia.

Multidisciplinary hemophilia treatment centers, which coordinate most U.S. hemophilia care and source the bulk of their drug supply through specialty pharmacies equipped for cold-chain handling and prior-authorization complexity, continue to anchor the United States hemophilia market, reinforced by the steady cadence of new product approvals and label expansions.

A universal healthcare framework underpins hemophilia care just north of the border, where national blood and plasma authorities, including Canadian Blood Services and Héma-Québec, coordinate the collection and fractionation of plasma-derived products alongside publicly funded access to recombinant factor concentrates and newer non-factor therapies. The Canadian Hemophilia Society and a network of comprehensive care clinics support diagnosis and long-term management, giving Canada a smaller but well-organized market that generally follows U.S. and European approval timelines with a modest lag.

A pattern common across middle-income healthcare systems plays out south of the U.S. border as well: public insurance programs provide a foundation of access to factor replacement therapy, but reimbursement for newer non-factor and gene therapies typically lags behind the United States and Western Europe. As in much of Latin America, demand growth in Mexico is closely tied to continued investment in diagnostic capacity and specialist hematology services, which determine how much of the country's hemophilia population can be identified and brought into structured treatment in the first place.

Europe Hemophilia Market: Mature Recombinant Adoption with Growing Gene Therapy Interest

Europe represents a mature hemophilia market characterized by high adoption of recombinant factor concentrates, near-universal public health system coverage of routine prophylaxis, and growing clinical interest in gene therapy, even as commercial uptake of curative-intent products has been gradual across the region just as it has in the United States.

Germany holds the largest national hemophilia market in Europe, a position reinforced by the country's large, well-funded statutory health insurance system and an emphasis on recombinant therapies; German hospital pharmacies dominate distribution, and patients with inhibitors are increasingly managed through non-factor therapies as they reach the market. NHS coverage ensures broad access to prophylactic factor VIII and IX therapy across the United Kingdom, where the market is expected to grow at one of the fastest rates in Europe, supported by a steady shift toward biosimilars and extended half-life products; specialty centers manage severe and inhibitor cases using monoclonal antibody therapies such as emicizumab.

Comprehensive public healthcare coverage and a long-established network of academic hospital-affiliated treatment centers support hemophilia care in France, where recombinant factor VIII dominates treatment of severe hemophilia A and desmopressin remains a routine option for milder cases. Italy and Spain both operate within public national health systems that provide broad access to factor concentrates and an expanding range of non-factor therapies, with hemophilia treatment centers concentrated in major academic hospitals coordinating most specialist care in each country.

Asia Pacific Hemophilia Market: Fastest-Growing Region as Diagnosis Rates and Treatment Access Expand

Expanding diagnostic access, rising disease awareness, and improving affordability of modern factor and non-factor therapies are together making Asia Pacific the fastest-growing regional hemophilia market, at an estimated CAGR of approximately 7% through the forecast period.

Japan's advanced healthcare system supports broad use of recombinant factor VIII and IX therapies and has seen comparatively early adoption of non-factor options such as emicizumab, with hospital pharmacies and specialty distribution channels ensuring consistent access for the country's hemophilia population. Just across the sea, a national rare-disease policy framework that has formally classified hemophilia as a covered condition since the publication of China's first National List of Rare Diseases in 2018 is channeling growing government and insurance support toward diagnosis and treatment; plasma-derived factor concentrates remain widely used there even as recombinant options expand and as the country's own biotechnology sector matures, exemplified by the April 2025 NMPA approval of BBM-H901, developed by Belief BioMed and commercialized by Takeda China as the country's first domestically developed hemophilia B gene therapy.

A distinctive marker of clinical credibility arrived for the broader region in December 2024, when researchers at the Centre for Stem Cell Research at Christian Medical College, Vellore, working with India's Department of Biotechnology and Emory University, published results in the New England Journal of Medicine from the country's first human gene therapy trial for severe hemophilia A, using a lentiviral vector to deliver a functioning factor VIII gene into patients' own stem cells. Beyond this milestone, India's broader hemophilia care landscape continues to rely predominantly on plasma-derived and standard factor concentrates, with the country's 2021 National Policy for Rare Diseases gradually expanding government support for diagnosis and treatment of conditions including hemophilia.

A comprehensive national health insurance system and well-developed specialist hematology infrastructure support relatively rapid adoption of new recombinant and non-factor therapies in South Korea, typically following close behind their approval in larger markets. Further south, the National Blood Authority and Australian Red Cross Lifeblood coordinate most of the activity in this market, managing centralized procurement and supply of plasma-derived and recombinant clotting factor products to patients through the public Medicare-funded health system. A near-universal health coverage scheme, meanwhile, provides the foundation for continued development in Thailand, even as the country, like much of Southeast Asia, keeps building out specialist diagnostic and hematology capacity.

South America Hemophilia Market: Brazil's Public Program Anchors a Developing Region

South America’s hemophilia market remains in a comparatively early stage of development, with increasing diagnosis of hemophilia A and B and gradually broadening access to prophylactic therapy, though over-the-counter or unstructured treatment remains rare and most care continues to be administered through hospital pharmacies.

Brazil leads the region by a wide margin, anchored by a dedicated national hemophilia care program operated through the country's public Sistema Único de Saúde, which supplies plasma-derived factor VIII and IX concentrates to the public system while private treatment centers provide access to recombinant therapies and monoclonal antibody options for patients with private coverage. Recurring macroeconomic volatility, by contrast, continues to shape the market next door in Argentina, raising the effective cost of imported biologic therapies and complicating consistent procurement planning, even as the country's hemophilia treatment centers and patient organizations maintain structured care for diagnosed patients.

Middle East & Africa Hemophilia Market: Healthcare Modernization Driving Gradual Access Expansion

Rising investment in specialist care infrastructure and gradually improving diagnosis rates for hemophilia A and B are the defining forces behind the Middle East & Africa hemophilia market, an emerging but increasingly visible market for global manufacturers.

The broader healthcare modernization agenda tied to the Kingdom's Vision 2030 reform program is expanding specialist hematology capacity and supporting growing demand for recombinant factor VIII and IX replacement in Saudi Arabia, alongside rising interest in gene therapy trial participation through partnerships with international manufacturers. Substantial healthcare infrastructure investment concentrated in Dubai and Abu Dhabi is similarly lifting demand in the United Arab Emirates, while Kuwait's smaller market operates within the country's free, state-funded healthcare system for citizens, which provides a structural foundation for consistent factor replacement access. A private hospital and specialist hematology sector leads activity at the southern end of the region, in South Africa, supported by the Hemophilia Foundation of South Africa's patient advocacy and education work.

Regulatory & Reimbursement Landscape in Hemophilia Treatment

Hemophilia therapies are regulated and reimbursed through four broadly distinct frameworks across the United States, the European Union, Japan, and China, each combining orphan drug incentives with a different approach to pricing and health technology assessment that materially shapes how quickly, and at what price, new products reach patients.

United States: Orphan Incentives, Warranty-Based Contracting, and a Fragmented Payer Landscape

Nearly all approved hemophilia therapies, including BEQVEZ, Hympavzi, Alhemo, Qfitlia, HEMGENIX, and Roctavian, have qualified for FDA orphan drug designation, which confers seven years of market exclusivity, tax credits for qualified clinical trial costs, and waived prescription drug user fees; several have additionally received Breakthrough Therapy or Priority Review designations that compressed their review timelines. Because the U.S. market lacks a centralized cost-effectiveness gatekeeper, reimbursement instead plays out through negotiation with individual commercial payers, Medicare, Medicaid, and the 340B Drug Pricing Program, with the nonprofit Institute for Clinical and Economic Review acting as an influential but non-binding voice on value: ICER's 2022 assessment proposed fair-value price benchmarks of approximately USD 1.9 million for Roctavian and USD 2.9 million for HEMGENIX, both below the wholesale acquisition cost ultimately set by their manufacturers.

 In response, all three approved hemophilia gene therapies, BEQVEZ, HEMGENIX, and Roctavian, are sold with outcomes-based warranty agreements that refund payers a portion of the purchase price if a patient fails to respond or loses durable benefit within a defined window, typically three-and-a-half to four years; a January 2023 Avalere Health survey of 46 U.S. health plans found that 58% were already using outcomes-based contracts of this kind somewhere in their specialty drug portfolios, underscoring that hemophilia gene therapy warranties fit into a broader, accelerating payer shift toward value-based specialty contracting rather than standing apart as a one-off response.

European Union: Centralized Authorization Meets a New Joint Clinical Assessment Regime

EMA orphan drug designation grants ten years of market exclusivity, together with protocol assistance and fee reductions, and most approved hemophilia therapies hold this status across the bloc. Historically, EMA centralized marketing authorization has been followed by entirely separate, uncoordinated health technology assessments and price negotiations in each member state. That changed on January 12, 2025, when the EU's Health Technology Assessment Regulation became operational, introducing a mandatory Joint Clinical Assessment (JCA) for all new cancer medicines and Advanced Therapy Medicinal Products, a category that includes gene therapies such as HEMGENIX and Roctavian; orphan medicinal products more broadly are scheduled to enter the JCA framework in 2028, with all other centrally authorized products following in 2030.

The JCA produces a single, EU-level clinical-benefit assessment that individual member states then use as an input into their own, still-separate pricing and reimbursement decisions, with the European Commission reporting that ten Joint Clinical Assessments and seven Joint Scientific Consultations were completed in the framework's first year of operation. National pricing patterns continue to vary widely even under this new framework: when BioMarin priced Roctavian at a wholesale acquisition cost of USD 2.9 million in the U.S., the company had already negotiated a price of approximately USD 900,000 for an early patient treated in Germany in 2023, illustrating how U.S. list pricing and European national price negotiations can produce very different outcomes for the same product.

China: A Maturing Rare Disease Framework and a New Commercial Insurance Channel for High-Cost Therapies

China's regulatory and reimbursement framework for hemophilia has matured substantially since the National Health Commission published the country's first National List of Rare Diseases in 2018, which formally includes hemophilia and triggered eligibility for priority review at the National Medical Products Administration alongside province-level patient assistance mechanisms. China's National Reimbursement Drug List (NRDL) runs an annual negotiation round that delivered an average price reduction of 63% across the 89 drugs successfully listed in the 2024 cycle.

China's National Healthcare Security Administration has complemented this with the Commercial Health Insurance Innovative Drug List (CHIIDL), published alongside the renewed NRDL on December 7, 2025 and effective January 1, 2026, as a parallel reimbursement channel specifically designed for high-cost innovative and rare-disease therapies. China's own domestic hemophilia B gene therapy, BBM-H901 (dalnacogene ponparvovec), developed by Belief BioMed and commercialized by Takeda China following its NMPA approval in April 2025, is among the rare-disease candidates positioned for this new commercial-insurance pathway, alongside continued City-level commercial insurance coverage gains for Roche's Hemlibra.

Epidemiology in Hemophilia: Diagnosed, Treated, and Addressable Population

Sizing the hemophilia market accurately requires distinguishing between the population believed to have the disorder, the population formally diagnosed and registered with patient organizations, and the smaller population currently treated with modern, reimbursed therapies, since the gap between these layers represents both an access challenge and a long-term volume opportunity. The World Federation of Hemophilia's global survey work has formally identified more than 347,000 people with hemophilia across more than 120 countries, while researchers studying underdiagnosis estimate the true global population living with the disorder, including those never formally diagnosed, may exceed 1.1 million, implying that less than a third of the people who actually have hemophilia are currently captured in formal registries and care pathways.

This diagnosis gap is uneven across geographies, which is precisely why epidemiology rather than pure economic development explains much of this report's regional growth pattern: the CDC's relatively precise estimate of approximately 33,000 diagnosed males living with hemophilia in the United States reflects a mature, comprehensive surveillance system built around hemophilia treatment centers, while China's rare-disease policy framework and expanding insurance coverage have been narrowing the country's historical diagnosis gap. The practical implication for market sizing is that Asia Pacific's status as the fastest-growing region, discussed earlier in this report, is driven as much by newly diagnosed patients entering treatment for the first time as by switching among already-diagnosed patients to newer therapy classes, a dynamic that differs meaningfully from the more innovation- and switching-driven growth pattern in North America and Europe, where the diagnosed population is comparatively stable and growth instead comes from therapy-class migration and price.

Hemophilia Pipeline Overview

The hemophilia pipeline behind the products already covered in this report's Key Developments section remains active across both factor and non-factor mechanisms. Novo Nordisk filed a U.S. Biologics License Application in September 2025 for Mim8 (denecimig), a once-weekly, once-every-two-weeks, or once-monthly bispecific antibody designed to mimic factor VIIIa for hemophilia A regardless of inhibitor status, positioning it as a direct successor-mechanism challenger to Roche's Hemlibra. In China, Belief BioMed's BBM-H901 (dalnacogene ponparvovec), approved by the NMPA in April 2025 and commercialized through a partnership with Takeda China, gives the country its first domestically developed hemophilia B gene therapy, while global developers including uniQure, the original architect of the HEMGENIX vector technology now commercialized by CSL Behring, continue long-term post-marketing follow-up work that will shape how subsequent gene therapy candidates are evaluated by regulators and payers alike.

Key Developments

In June 2026, the FDA approved an expanded indication for Pfizer's Hympavzi (marstacimab-hncq), broadening its use to people aged 12 and older with hemophilia A or B who have inhibitors, and to pediatric patients aged 6 to 11 with or without inhibitors.

In December 2025, CSL Behring presented five-year, end-of-study results from the pivotal HOPE-B trial for HEMGENIX (etranacogene dezaparvovec) at the American Society of Hematology annual meeting, with the data published simultaneously in the New England Journal of Medicine.

In September 2025, Novo Nordisk submitted a Biologics License Application to the FDA for Mim8 (denecimig), a once-weekly bispecific antibody for hemophilia A with or without inhibitors, based on the Phase 3 FRONTIER program.

In April 2025, China's National Medical Products Administration approved BBM-H901 (dalnacogene ponparvovec), developed by Belief BioMed and commercialized by Takeda China, as the country's first domestically developed gene therapy for hemophilia B.

In March 2025, the FDA approved Sanofi's Qfitlia (fitusiran) for routine prophylaxis in adults and pediatric patients aged 12 and older with hemophilia A or B, with or without factor VIII or IX inhibitors.

In December 2024, researchers at the Centre for Stem Cell Research, Christian Medical College, Vellore, published results in the New England Journal of Medicine from India's first human gene therapy trial for severe hemophilia A, using a lentiviral vector to deliver a factor VIII transgene into patients' own hematopoietic stem cells.

In December 2024, the FDA approved Novo Nordisk's Alhemo (concizumab) for routine prophylaxis in patients with hemophilia A or B with inhibitors.

In October 2024, the FDA approved Pfizer's Hympavzi (marstacimab) for routine prophylaxis in adult and pediatric patients aged 12 and older with hemophilia A or B without inhibitors.

In June 2024, the European Commission approved Sanofi's ALTUVIIIO, marketed in the EU as ALTUVOCT, for the treatment and prevention of bleeding episodes and perioperative management in hemophilia A.

In May 2024, the FDA approved an updated label for Sanofi's ALTUVIIIO incorporating full results from the Phase 3 XTEND-Kids study, confirming effective bleed protection with once-weekly dosing in children with hemophilia A.

In April 2024, the FDA approved Pfizer's BEQVEZ (fidanacogene elaparvovec), the second approved gene therapy for hemophilia B, based on Phase 3 BENEGENE-2 trial data.