Haemophilia Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Type (Haemophilia A, Haemophilia B, Haemophilia C), By Product (Recombinant Coagulation Factor Concentrates, Plasma Derived Coagulation Factor Concentrate, Desmopressin, Antifibrinolytics Agents), By Treatment (On-demand and Prophylaxis), By Therapy (Replacement Therapy, ITI therapy and Gene Therapy), By Region and Competition, 2021-2031F

Forecast Period	2027-2031
Market Size (2025)	USD 12.03 Billion
CAGR (2026-2031)	5.75%
Fastest Growing Segment	Haemophilia A
Largest Market	North America
Market Size (2031)	USD 16.82 Billion

Market Overview

The Global Haemophilia Treatment Market will grow from USD 12.03 Billion in 2025 to USD 16.82 Billion by 2031 at a 5.75% CAGR. Haemophilia treatment comprises replacement therapies, including recombinant and plasma-derived clotting factors, alongside non-factor agents designed to correct deficiencies in Factor VIII or IX. The market is primarily driven by the increasing global identification of patients and the shift toward prophylactic care regimens which necessitate consistent product usage. According to the World Federation of Hemophilia, in 2023, the total number of people identified with haemophilia globally was reported to be 218,804. This rising diagnosis rate provides a tangible basis for sustained demand as healthcare systems prioritize long-term management of bleeding disorders.

However, a significant challenge impeding market expansion is the prohibitive cost associated with advanced therapeutic options. The high price of recombinant factors and novel therapies creates severe accessibility barriers, particularly in low- and middle-income countries where reimbursement frameworks are often insufficient. This economic disparity restricts the addressable market in developing regions and remains a critical hurdle for manufacturers aiming to broaden their global reach.

Key Market Drivers

The commercialization of novel gene therapies and curative solutions is a primary driver reshaping the global haemophilia treatment landscape, moving the market trajectory from chronic disease management toward one-time potential cures. This transition is characterized by the approval of high-value treatments designed to provide long-term bleed protection through a single administration, thereby altering payer models and long-term revenue projections. A pivotal market development highlights this value shift; according to Fierce Pharma, April 2024, in the 'Pfizer scores FDA nod for hemophilia B gene therapy' article, the newly approved gene therapy Beqvez entered the market with a list price of $3.5 million. Such therapies attract substantial interest from healthcare systems aiming to mitigate the lifetime economic burden of severe bleeding disorders, despite the significant upfront investment required for these sophisticated biologic interventions.

Simultaneously, the rapid uptake of non-factor replacement therapies is fundamentally changing standard prophylactic care by reducing the treatment burden for patients. The market is witnessing a decisive shift from intravenous clotting factors to subcutaneous bispecific antibodies that offer reduced dosing frequency and consistent efficacy. This trend is substantiated by the financial performance of leading non-factor agents; according to Roche, February 2024, in the 'Finance Report 2023', full-year sales of the non-factor therapy Hemlibra reached CHF 4,147 million, reflecting its dominance in the prophylaxis setting. While innovative non-factor agents expand their reach, the overall volume of therapeutic demand remains immense across the sector. According to the World Federation of Hemophilia, October 2024, in the 'Report on the Annual Global Survey 2023', the total global consumption of Factor VIII concentrates was reported to be approximately 9.4 billion IU, underscoring the sustained scale of therapeutic requirements worldwide.

Download Free Sample Report

Key Market Challenges

The prohibitive cost associated with advanced therapeutic options stands as a formidable barrier to the growth of the global haemophilia treatment market. High prices for recombinant clotting factors and novel gene therapies create a steep financial threshold that many healthcare systems in developing regions cannot surmount. In countries where public reimbursement frameworks are insufficient or nonexistent, these life-saving treatments remain largely inaccessible to the majority of the patient population. This economic disconnect severely restricts the commercial reach of pharmaceutical manufacturers, forcing them to rely heavily on saturated high-income markets while leaving substantial demand in emerging economies unaddressed due to affordability constraints.

The impact of this economic disparity is clearly illustrated by the uneven distribution of treatment products worldwide. According to the World Federation of Hemophilia, in 2023, high-income countries accounted for 61% of the total global Factor VIII usage, whereas low-income countries contributed a mere 0.3% to the global consumption. This stark imbalance demonstrates that despite rising diagnosis rates, the market’s potential is artificially capped by the inability of lower-income nations to purchase premium therapeutics. Consequently, the industry struggles to convert the growing global patient pool into tangible revenue, hampering overall market expansion.

Key Market Trends

The Widespread Adoption of Extended Half-Life Factor Concentrates is actively reshaping the competitive dynamics of the haemophilia sector by reinvigorating the relevance of factor replacement therapies against the rise of non-factor alternatives. This trend is characterized by the uptake of next-generation fusion proteins that significantly extend the circulating half-life of Factor VIII, thereby allowing patients to maintain high sustained factor activity with reduced injection frequency. The immediate commercial impact of this shift is evident in the rapid market penetration of recently approved agents that offer once-weekly dosing while preserving peak protection levels. According to Sanofi, October 2024, in the 'Press Release: Sanofi Q3 2024', sales of the extended half-life therapy Altuviiio reached €172 million in the third quarter alone, driven primarily by patients switching from both standard factor regimens and other therapeutics.

Parallel to the evolution of factor replacement, the Advancement in Novel RNA Interference Therapeutics is introducing a distinct mechanism of action that targets the coagulation cascade at a fundamental level. Unlike replacement factors or bispecific antibodies, these investigational therapies utilize small interfering RNA to suppress antithrombin production, effectively rebalancing hemostasis for patients with either Haemophilia A or B, regardless of inhibitor status. This approach aims to provide consistent bleed protection through a substrate-independent pathway, marking a departure from traditional protein replacement strategies. New clinical data supports this mechanism's precision; according to Sanofi, June 2024, in the 'Press Release: ISTH: Sanofi advances leadership in hemophilia', presentations from the phase 3 study demonstrated that maintaining antithrombin activity levels between 15% and 35% via this RNAi therapy resulted in clinically meaningful bleed control for patients.

Segmental Insights

The Haemophilia A segment represents the fastest-growing category within the Global Haemophilia Treatment Market, primarily driven by its significantly higher prevalence compared to other forms of the disorder. This expansion is further accelerated by a paradigm shift in therapeutic protocols, characterized by the rising adoption of innovative non-factor replacement therapies and extended half-life products. Furthermore, consistent approvals from major regulatory bodies, such as the FDA and EMA, for breakthrough gene therapies have revolutionized disease management. These advancements address critical unmet needs by reducing infusion frequency and improving patient outcomes, thereby fueling rapid market uptake for Haemophilia A treatments.

Regional Insights

North America maintains a leading position in the global haemophilia treatment market driven by the high rate of diagnosis and the presence of established pharmaceutical manufacturers. The region benefits from a robust healthcare infrastructure that enables early disease detection and consistent management. Additionally, favorable reimbursement policies and strong regulatory support from the US Food and Drug Administration improve patient access to essential therapies. These elements create a supportive environment for market expansion and ensure the region continues to command a significant share of the global industry.

Recent Developments

• In December 2024, Novo Nordisk presented key clinical data from its haemophilia portfolio at the 66th American Society of Hematology (ASH) Annual Meeting. The company highlighted interim results from the Phase 3 Frontier4 extension study of Mim8, a next-generation bispecific antibody designed for prophylaxis in people with haemophilia A, regardless of inhibitor status. Additionally, findings from the Explorer7 trial evaluating concizumab in patients with haemophilia A or B were shared. These presentations underscored the company's progress in developing subcutaneous treatment options that aim to improve clinical outcomes and reduce the treatment burden for individuals with rare bleeding disorders.
• In October 2024, Pfizer Inc. received approval from the US FDA for HYMPAVZI (marstacimab-hncq), a novel treatment for routine prophylaxis in adults and pediatric patients aged 12 years and older with haemophilia A or B without inhibitors. This approval introduced the first anti-tissue factor pathway inhibitor to the US market, offering a once-weekly subcutaneous injection option that targets a protein in the blood clotting process to prevent bleeding. The authorization was supported by data from the Phase 3 BASIS trial, which showed that the therapy significantly reduced the annualized bleeding rate compared to standard factor replacement regimens.
• In June 2024, Swedish Orphan Biovitrum AB (Sobi) announced that the European Commission granted Marketing Authorisation for ALTUVOCT (efanesoctocog alfa) for the treatment and prevention of bleeds in patients with haemophilia A. Developed in collaboration with Sanofi, this high-sustained factor VIII replacement therapy was approved for use in all age groups and disease severities. The approval was based on results from the pivotal Phase 3 XTEND-1 and XTEND-Kids studies, which demonstrated that once-weekly prophylaxis provided significant bleed protection and improved joint health. This regulatory milestone marked a significant expansion of the product's availability following its earlier approvals in the United States and Japan.
• In April 2024, Pfizer Inc. announced that the US Food and Drug Administration (FDA) granted approval for its gene therapy, BEQVEZ (fidanacogene elaparvovec-dzkt), for the treatment of adults with moderate to severe haemophilia B. This one-time treatment was indicated for patients who currently use factor IX prophylaxis therapy, have a history of life-threatening hemorrhage, or experience repeated, serious spontaneous bleeding episodes. The therapy was designed to enable patients to produce factor IX themselves, significantly reducing the burden of regular infusions. The company set the list price for this transformative therapy at $3.5 million, matching the cost of other gene therapies in the market.

Key Market Players

• Bayer AG
• BioMarin Pharmaceutical, Inc.
• CSL Behring LLC
• Kedrion S.p.A
• Novo Nordisk A/S
• Pfizer, Inc.
• Octapharma AG
• Sanofi SA
• Takeda Pharmaceutical Company Limited
• Grifols S.A.

By Type	By Product	By Treatment	By Therapy	By Region
Haemophilia A Haemophilia B Haemophilia C	Recombinant Coagulation Factor Concentrates Plasma Derived Coagulation Factor Concentrate Desmopressin Antifibrinolytics Agents	On-demand and Prophylaxis	Replacement Therapy ITI therapy and Gene Therapy	North America Europe Asia Pacific South America Middle East & Africa

Report Scope:

In this report, the Global Haemophilia Treatment Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• Haemophilia Treatment Market, By Type:

• Haemophilia A
• Haemophilia B
• Haemophilia C

• Haemophilia Treatment Market, By Product:

• Recombinant Coagulation Factor Concentrates
• Plasma Derived Coagulation Factor Concentrate
• Desmopressin
• Antifibrinolytics Agents

• Haemophilia Treatment Market, By Treatment:

• On-demand and Prophylaxis

• Haemophilia Treatment Market, By Therapy:

• Replacement Therapy
• ITI therapy and Gene Therapy

• Haemophilia Treatment Market, By Region:

• North America
• United States
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Italy
• Germany
• Spain
• Asia Pacific
• China
• India
• Japan
• Australia
• South Korea
• South America
• Brazil
• Argentina
• Colombia
• Middle East & Africa
• South Africa
• Saudi Arabia
• UAE