Hemophilia B Market Outlook

Hemophilia B companies are Centessa Pharmaceuticals, Sanofi (Genzyme)/Alnylam Pharmaceuticals, Pfizer/ Spark Therapeutics, Novo Nordisk.

LAS VEGAS, NEVADA, UNITED STATES, May 27, 2024 /EINPresswire.com/ -- DelveInsight’s “Hemophilia B Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Hemophilia B, historical and forecasted epidemiology as well as the Hemophilia B market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

To Know in detail about the Hemophilia B market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Hemophilia B Market Forecast

Some of the key facts of the Hemophilia B Market Report:

The Hemophilia B market size is anticipated to grow with a significant CAGR during the study period (2020-2034).
The market size of hemophilia B in 2023, in the 7MM was around USD 3,400 million. Among the 7MM, the United States accounted for the highest market size.
The most common treatment for hemophilia B is replacement therapy. BeneFIX, RIXUBIS, IXINITY, ALPROLIX, IDELVION, and REBINYN are the some available recombinant factor IX products in the US. Apart from these, bypassing agents such as Feiba, Novoseven, and SEVENFACT and human plasma-derived coagulation factor IX – AlphaNine SD and Mononine have bagged the FDA approval as well.
The advent of gene therapies brings the potential for curing patients with Hemophilia B. HEMGENIX, an approved gene therapy for Hemophilia B in November 2022, carries an astonishing price tag of USD 3.5 million per dosage, making it the most expensive medication worldwide and marking the first gene therapy to be approved for this rare disease.
The most recent development in hemophilia B is the addition of Pfizer’s BEQVEZ, another gene therapy approved in April 2024 for the treatment of hemophilia B.
Pfizer is the second company in the indication, trailing CSL and uniQure's HEMGENIX for hemophilia B treatment. Despite having the opportunity to offer a lower price than its competitor, Pfizer opted to price its drug, BEQVEZ, at the same USD 3.5 million.
Hemophilia B market has diverse pipeline such as extended half-life therapies, siRNA, antibodies and gene therapy. These therapies are expected to provide the patient convenient route of administration and longer treatment duration.
Recently, in December 2023, the US FDA has accepted BLA for Pfizer’s Marstacimab treating hemophilia A or B without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). The European MAA for marstacimab also passed validation and is currently under review by the EMA. The Prescription Drug User Fee Act (PDUFA) action date is in the fourth quarter of 2024, and the decision from the European Commission is anticipated by the first quarter of 2025.
Expected launch of potential therapies, FITUSIRAN (Sanofi), Marstacimab (Pfizer), SerpinPC (Centessa Pharmaceuticals and others may increase the market size in the coming years, assisted by an increase in the prevalent population of hemophilia B. In addition to this, gaining popularity of gene therapies and increased focus on prophylactic treatment will boast the market 7MM during the forecast period (2024─2034).
Key Hemophilia B Companies: Centessa Pharmaceuticals, Sanofi (Genzyme)/Alnylam Pharmaceuticals, Pfizer/ Spark Therapeutics, Novo Nordisk, and others
Key Hemophilia B Therapies: BEQVEZ (fidanacogene elaparvovec-dzkt), HEMGENIX (etranacogene dezaparvovec-drlb), Fitusiran, PF-06741086 (Marstacimab), and others
The Hemophilia B market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Hemophilia B pipeline products will significantly revolutionize the Hemophilia B market dynamics.

Hemophilia B Overview

Hemophilia B, also known as Christmas disease, is a genetic bleeding disorder caused by a deficiency in clotting factor IX, a protein crucial for blood clotting. This condition is inherited in an X-linked recessive pattern, primarily affecting males while females typically carry the genetic mutation without displaying symptoms. Individuals with hemophilia B are prone to prolonged bleeding episodes, particularly after injury or surgery, due to the impaired ability of their blood to clot properly.

Symptoms of hemophilia B may include frequent nosebleeds, easy bruising, prolonged bleeding from cuts or injuries, and in severe cases, spontaneous bleeding into joints and muscles. The severity of symptoms varies depending on the level of factor IX deficiency, with those severely affected experiencing more frequent and severe bleeding episodes.

Treatment for hemophilia B typically involves replacement therapy with clotting factor IX concentrates to restore the missing or deficient clotting factor in the blood. This can be administered preventively to reduce the risk of bleeding (prophylactic therapy) or on-demand to manage bleeding episodes. In recent years, gene therapy has emerged as a promising approach for treating hemophilia B by introducing functional factor IX genes into the body to produce the missing clotting factor.

While there is currently no cure for hemophilia B, ongoing advancements in treatment options and management strategies have significantly improved the quality of life for individuals living with this condition. Additionally, preventive measures such as avoiding activities with a high risk of injury and maintaining regular medical follow-ups can help minimize complications associated with hemophilia B.

Hemophilia B Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Hemophilia B Epidemiology Segmentation:

The Hemophilia B market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Total Prevalence of Hemophilia B
Prevalent Cases of Hemophilia B by severity
Gender-specific Prevalence of Hemophilia B
Diagnosed Cases of Episodic and Chronic Hemophilia B

Download the report to understand which factors are driving Hemophilia B epidemiology trends @ Hemophilia B Epidemiology Forecast

Hemophilia B Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Hemophilia B market or expected to get launched during the study period. The analysis covers Hemophilia B market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Hemophilia B Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Hemophilia B Key Companies

Centessa Pharmaceuticals, Sanofi (Genzyme)/Alnylam Pharmaceuticals, Pfizer/ Spark Therapeutics, Novo Nordisk

Hemophilia B Therapies

BEQVEZ (fidanacogene elaparvovec-dzkt), HEMGENIX (etranacogene dezaparvovec-drlb), Fitusiran, PF-06741086 (Marstacimab)
Discover more about therapies set to grab major Hemophilia B market share @ Hemophilia B Treatment Landscape

Hemophilia B Market

At present, hemophilia B remains without a cure, with no existing treatments capable of halting or reversing the disease's progression. While a handful of drugs approved by the FDA can modestly slow its course, complete eradication remains elusive. Consequently, management strategies for hemophilia B primarily revolve around providing supportive care and addressing symptoms as they arise. Recent years have witnessed a surge in research efforts exploring novel treatment modalities, including gene therapy, cellular therapy, and immune tolerance induction agents. While a few approved drugs, such as IDELVION, IXINITY, REBINYN, and ALPROLIX, offer some reprieve by extending periods of autonomy and enhancing survival rates, their efficacy remains limited.

Despite significant strides in hemophilia treatment over the past two decades, the emergence of inhibitors poses a formidable challenge, complicating efforts to manage bleeding episodes and perform surgical procedures effectively. The absence of a curative treatment option significantly impacts patients' quality of life and hampers their daily activities. Gene therapy presents a beacon of hope for a definitive cure, offering the potential for lasting benefits with a single administration, unlike the diverse array of treatments currently in use. Notably, HEMGENIX and BEQVEZ have secured FDA approval as gene therapies for treating hemophilia B.

The pipeline of potential therapies for hemophilia B is brimming with promise, with numerous candidates in various stages of development. Notable contenders such as fitusiran, Marstacimab, SerpinPC, among others, are undergoing clinical trials, showcasing potential as disease-modifying agents, symptomatic treatments, and targeted therapies addressing specific pathways. These advancements hold the potential to address the unmet treatment needs of hemophilia B patients, offering renewed hope for improved outcomes and enhanced quality of life.

Scope of the Hemophilia B Market Report

Study Period: 2020–2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Hemophilia B Companies: Centessa Pharmaceuticals, Sanofi (Genzyme)/Alnylam Pharmaceuticals, Pfizer/ Spark Therapeutics, Novo Nordisk, and others
Key Hemophilia B Therapies: BEQVEZ (fidanacogene elaparvovec-dzkt), HEMGENIX (etranacogene dezaparvovec-drlb), Fitusiran, PF-06741086 (Marstacimab), and others
Hemophilia B Therapeutic Assessment: Hemophilia B current marketed and Hemophilia B emerging therapies
Hemophilia B Market Dynamics: Hemophilia B market drivers and Hemophilia B market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
Hemophilia B Unmet Needs, KOL’s views, Analyst’s views, Hemophilia B Market Access and Reimbursement

To know more about Hemophilia B companies working in the treatment market, visit @ Hemophilia B Clinical Trials and Therapeutic Assessment

Table of Contents

1. Hemophilia B Market Report Introduction
2. Executive Summary for Hemophilia B
3. SWOT analysis of Hemophilia B
4. Hemophilia B Patient Share (%) Overview at a Glance
5. Hemophilia B Market Overview at a Glance
6. Hemophilia B Disease Background and Overview
7. Hemophilia B Epidemiology and Patient Population
8. Country-Specific Patient Population of Hemophilia B
9. Hemophilia B Current Treatment and Medical Practices
10. Hemophilia B Unmet Needs
11. Hemophilia B Emerging Therapies
12. Hemophilia B Market Outlook
13. Country-Wise Hemophilia B Market Analysis (2020–2034)
14. Hemophilia B Market Access and Reimbursement of Therapies
15. Hemophilia B Market Drivers
16. Hemophilia B Market Barriers
17. Hemophilia B Appendix
18. Hemophilia B Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

Related Reports:

Hemophilia B Pipeline

"Hemophilia B Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Hemophilia B market. A detailed picture of the Hemophilia B pipeline landscape is provided, which includes the disease overview and Hemophilia B treatment guidelines.

Hemophilia B Epidemiology

DelveInsight's 'Hemophilia B Epidemiology Forecast to 2034' report delivers an in-depth understanding of the disease, historical and forecasted Hemophilia B epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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