GLOBAL HEMOPHILIA MARKET ANALYSIS (2024 - 2030)

The estimated global market size for Hemophilia is projected to increase from USD 11.49 Billion in 2023 to approximately USD 16.06 billion by 2030, indicating a robust Compound Annual Growth Rate (CAGR) of 4.9% during the period from 2024 to 2030. This growth is primarily propelled by factors such as the rising prevalence of hemophilia and the introduction of new products and medications aimed at its treatment.

Industry Overview:

Hemophilia, a hereditary disorder characterized by inadequate blood coagulation, results in spontaneous bleeding, joint pain, swelling, and hemorrhages in various parts of the body. Effective management of hemophilia entails high-quality medical care delivered by healthcare professionals knowledgeable about the condition, thereby helping patients prevent severe complications. Comprehensive Hemophilia Treatment Centers (HTCs) often represent the optimal treatment approach. Techniques such as hemophoresis, involving blood circulation or tissue flushing, along with the use of desmopressin to enhance endogenous factor VIII levels in hemophilia A, are commonly employed.

The response to desmopressin can vary significantly among individuals. Based on expert recommendations, prophylaxis with SHL-FVIII concentrate is typically the standard treatment for severe hemophilia A patients, with potential consideration for certain individuals with moderate disease. Hemophilia manifests across a spectrum of severity, categorized as severe, moderate, or mild, based on plasma clotting factor activity levels.

According to a World Federation of Hemophilia report from October 2021, hemophilia is more prevalent in males due to its genetic association. It affects individuals worldwide across all ethnic groups. As per the Global Hemophilia Federation's Global Report published in October 2021, there are 165,379 patients diagnosed with hemophilia A, 33,076 with hemophilia B, and 11,159 with unspecified types globally, totaling 209,614 diagnosed cases. This substantial global prevalence underscores the increasing adoption of hemophilia treatments, thereby driving market growth.

Impact of COVID-19 on the Hemophilia Market:

Individuals with chronic conditions like hemophilia face heightened susceptibility to infections and must undertake special precautions to mitigate the risk of contracting COVID-19, leading to potential impacts on the market. A study published in the Journal of Dental and Medical Sciences in August 2020, titled "The Impact of the Covid19 Pandemic on the Prevention of Hemophilia in Children: The Experience of the Hemophilia Treatment Center in West Bengal, India," reported a significant increase in the average annual bleeding rate during the pandemic-induced lockdown. Factors such as transportation challenges, fear of viral transmission, and the financial burden of alternate transportation contributed to hindrances in accessing hemophilia treatment centers, thereby impeding market growth during the pandemic period.

Market Drivers:

The increasing predominance of Hemophilia is estimated to drive the growth of the market

The escalating prevalence of hemophilia is anticipated to be a key driver for market expansion. Techniques like hemophoresis, as well as advancements in medical research leading to the introduction of new therapeutic options, are further contributing to market growth.

New products and medication launches for the treatment of hemophilia are also driving the growth of the market

The launch of novel products and medications designed to address hemophilia is also fueling market expansion. For instance, in January 2022, Biomarin reported positive results from a phase III trial evaluating the gene therapy valoctocogeneroxaparvovec for severe hemophilia A.

Market Restraints:

However, challenges related to treatment accessibility and the high cost of therapy are restraining market growth.

Despite advancements in hemophilia treatment, administrative hurdles and escalating treatment expenses persist. Issues such as the development of inhibitors and the need for frequent infusions due to short half-lives of therapeutic molecules pose challenges for patients and healthcare providers alike, thereby hindering market growth. Additionally, the complexity and costs associated with immune regulation and tolerance induction in the presence of inhibitors further impede effective hemophilia management. Insufficient treatment guidelines and variability in patient responses to therapy complicate clinical decision-making, presenting further obstacles to market expansion.

Hemophilia Market - By Type:

• Hemophilia A
• Hemophilia B
• Hemophilia C
• Others

Categorized by type, the market encompasses various forms of hemophilia such as types A, B, C, among others. This classification hinges upon the deficiency of coagulation factors within individual patients. Notably, type A stands as the most prevalent, surpassing type B hemophilia by a factor of four.

WebMDLLC statistics from 2019 indicate a varying prevalence of type A across different countries, ranging from 5,414.5 cases per 100,000 men. Approximately 50.60% of patients grapple with severe hemophilia A, characterized by pronounced bleeding symptoms. Moreover, the majority of available treatments are tailored for hemophilia A.

However, Type B is projected to experience the most rapid growth throughout the forecast period, buoyed by robust pipeline products. Type B constitutes around 20% of global hemophilia cases, with half of those presenting factor IX levels exceeding 1%.

Hemophilia Market - By Application:

• On-Demand
• Prophylaxis

Regarding treatment modalities, the hemophilia market distinguishes between preventative measures and on-demand treatment. On-demand treatment occupied the largest market share in 2021, with prophylaxis expected to witness the swiftest expansion in the foreseeable future.

Prophylaxis entails the regular administration of coagulation factor concentrate, a widely accepted regimen among severe hemophilia patients, contributing to its growth trajectory. The duration of prophylactic treatment is contingent upon the severity of the patient's condition, with anticipated benefits including minimized productivity losses and enhanced quality of life.

Hemophilia Market - By Therapy:

• Replacement therapy
• ITI therapy
• Gene therapy

Therapeutic interventions available in the market encompass immune tolerance induction therapy (ITI), replacement therapy, and gene therapy. Replacement therapy dominated as the largest segment in 2021, while gene therapy is poised to be the fastest-growing sector in the forecast period. Replacement therapy serves as the standard treatment approach, facilitating the replenishment of deficient clotting factors within patients. Gene therapy represents an innovative frontier, targeting defective DNA bases to restore functional integrity. UniQure's AMT060 emerges as a promising candidate for type B treatment, currently undergoing clinical trials. Additionally, corporate endeavors by entities such as Spark Therapeutics Inc. and BioMarin further stimulate advancements in gene therapy for hemophilia.

Hemophilia Market - By Region:

• North America
• Europe
• Asia-Pacific
• Latin America
• The Middle East
• Africa

Geographically, North America commands market dominance, contributing over 44.4% to total sales. Regional growth is propelled by increasing disease prevalence and a shifting trend towards preventive measures. Notably, hemophilia diagnosis in the United States typically occurs during infancy.

Conversely, the Asia Pacific region is anticipated to exhibit the most accelerated growth in the forecast period. Early disease detection in countries like Japan, India, Indonesia, and Malaysia, coupled with heightened per capita usage of factor VIII and factor IX treatments, underpin this growth trajectory.

Hemophilia Market Share by Company:

1. Bayer AG
2. BioMarin Pharmaceutical, Inc.
3. CSL Behring
4. Kedrion
5. Novo Nordisk
6. Pfizer, Inc.
7. Roche (Chugai Pharmaceutical Co.)
8. Sanofi (Genzyme Corporation)
9. Takeda Pharmaceutical (Shire Plc.).

Pfizer Inc. and Sangamo Therapeutics, Inc., a genomic pharmaceuticals company, announced updated data from the Phase 1/2 Alta study of giroctocogenefitelparvovec, an investigational gene therapy for moderately severe to severe hemophilia A patients. Results from the Alta study in severe hemophilia A patients were slated for presentation at the 63rd American Society for Hematology Annual Meeting and Exposition, held virtually and in Atlanta, GA, from December 11-14. Detailed oral presentation slides, including follow-up data spanning up to 195 weeks for the longest-treated patient, were accessible on Sangamo's website under Events and Presentations in the Investors and Media section.

NOTABLE DEVELOPMENTS IN THE HEMOPHILIA MARKET RECENTLY:

• Clinical Milestone - In April 2022, Freeline Therapeutics Holdings plc administered the initial dose to the first patient in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for hemophilia B treatment, a debilitating genetic bleeding disorder stemming from a deficiency in clotting factor IX protein.
• Clinical Updates - At the 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders in April 2022, BioMarin Pharmaceutical Inc. unveiled positive findings from a two-year analysis of the Phase 3 GENEr8-1 study, along with a comprehensive safety update on valoctocogeneroxaparvovec, an investigational gene therapy targeting severe hemophilia A in adults.