The Evolving Market Dynamics of Factor-based Bleeding Disorders: Hemophilia A, Hemophilia B, and Von Willebrand Disease | Key Analysis by DelveInsight

Bleeding and clotting disorders are conditions that can impact both men and women, but women face unique issues related to these disorders because of the potential effects on their reproductive health and quality of life. Current data indicates that as many as 1% of women in the United States may be affected by a bleeding disorder, with many remaining unaware of their condition.

LAS VEGAS, Oct. 9, 2023 /PRNewswire/ — Bleeding disorders are typically hereditary, with common symptoms encompassing easy bruising, frequent nosebleeds, heavy menstrual bleeding, and excessive bleeding from minor cuts. Treatment approaches are tailored to the specific disorder and may involve factor replacement therapy, iron supplementation, topical applications, or blood transfusions.

DelveInsight has recently released a series of epidemiology-based market reports focusing on factor-based bleeding disorders including Hemophilia A, Hemophilia B, and Von Willebrand Disease. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032  segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Additionally, the reports feature an examination of prominent companies working with their lead candidates in different stages of clinical development. Let’s deep dive into the assessment of these factor-based bleeding disorders markets individually.

Hemophilia A Market

Hemophilia A is a hereditary bleeding disorder characterized by a deficiency or low levels of clotting factor VIII proteins in the bloodstream. Inadequate factor VIII inhibits proper blood clotting, leading to difficulty in controlling bleeding. While the disorder is typically inherited from parents, approximately one-third of cases occur without any prior family history. It results from an X-linked recessive genetic trait, with the malfunctioning gene located on the X chromosome. In females, who possess two X chromosomes, the unaffected gene on the other chromosome can compensate for the defective one, ensuring sufficient factor VIII production. According to DelveInsight’s recently published Hemophilia A market report, the total prevalent population of Hemophilia A in the 7MM comprised of 45K cases in 2021 and are projected to increase by 2032.

Approximately 30% of severe Hemophilia A patients develop neutralizing anti-FVIII alloantibodies (inhibitors), which make FVIII replacement therapy ineffective. The standard treatment approach for patients with inhibitors involves inducing immune tolerance with high-dose, high-frequency FVIII and using bypassing agents, such as recombinant activated factor VII (e.g., NOVOSEVEN) or FEIBA. Hemophilia A management includes various approved therapies, such as HEMLIBRA (for both inhibitor and non-inhibitor cases), SEVENFACT, KOGENATE FS, FEIBA, KOVALTRY, ELOCTATE, NUWIQ, AFSTYLA, and others. 

Recently in February 2023, the US FDA approved ALTUVIIIO (efanesoctocog alfa) factor VIII replacement therapy for adults and children with hemophilia A. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. Last year in September 2022, the company filed for approval in Japan. In August 2022, EC granted conditional marketing authorization to Roctavian gene therapy for treating severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VII inhibitors and without detectable antibodies to adeno-associated virus serotype 5. Whereas, in June 2023, BioMarin made a significant announcement revealing the FDA’s approval of Roctavian as a one-time remedy for individuals suffering from severe hemophilia A. 

Learn more about the FDA-approved drugs for hemophilia A @ Drugs for Hemophilia A Treatment

DelveInsight estimated that the hemophilia A market size is projected to grow from USD 8.5 billion in 2021 at a significant CAGR BY 2032. Currently, the treatment hemisphere is mainly driven by non-inhibitor drug candidates. Emerging potential candidates pose an adverse threat to current hemophilia A pharmaceuticals giants, by means of their potential to transform the standard of care for hemophilia A patients. The hemophilia treatment landscape continues to evolve, and several companies are furiously working toward the development of new treatments that could potentially cure and change the treatment landscape of hemophilia A. Companies are focused on the development of gene therapies that bispecific antibodies for the treatment of hemophilia A, this shall create a positive impact on the hemophilia A market size in coming years.

Hemophilia A Pipeline Therapies and Key Companies

• RG6357 (SPK-8011): Roche (Spark Therapeutics)
• RG6358 (SPK-8016): Roche (Spark Therapeutics)
• SerpinPC: ApcinteX
• Concizumab (NN7415): Novo Nordisk
• Fitusiran (ALN-AT3, SAR-439774): Sanofi (Genzyme)/Alnylam Pharmaceuticals
• BIVV001 (Efanesoctocog alfa) (rFVIIIFc-VWF-XTEN): Sanofi
• Marstacimab (PF-06741086): Pfizer
• NNC0365-3769 A (Mim8): Novo Nordisk A/S
• Giroctocogene fitelparvovec (SB-525 or PF-07055480): Pfizer/Sangamo Therapeutics
• BAY2599023 (DTX201 AAV FVIII): Bayer/Ultragenyx Pharmaceutical

Dive deeper for rich insights into the Hemophilia A Clinical Trials

Hemophilia B Market

Hemophilia B, a rare genetic bleeding disorder, is characterized by a deficiency of a blood protein known as factor IX. The level of factor IX in an affected individual’s blood dictates the severity of their hemophilia. Approximately 3 out of every 100 people with hemophilia B develop an antibody, referred to as an inhibitor, against the factor IX replacement therapy utilized to manage or prevent bleeding episodes. This inhibitor impedes the effectiveness of the therapy, making it more challenging to prevent episodes of bleeding. As per DelveInsight’s analysis, in 2022, the total treated prevalent population of hemophilia B in the US was approximately 4K. As per the estimates it can be observed that moderate hemophilia B cases are higher in number than mild and severe cases in the US.

At present, there is no known cure for hemophilia, but researchers are actively dedicated to developing a cure through gene therapy. One promising approach involves introducing a healthy version of the faulty blood factor gene, allowing individuals with hemophilia to produce adequate levels of the factor themselves. Treatment for hemophilia B typically includes two main approaches: “On-Demand and “Prophylaxis.” In recent years, the importance of prophylaxis treatment has been increasingly recognized over on-demand options. The primary treatment options for hemophilia B currently include Recombinant Factor IX Concentrates, Plasma-Derived Factor IX Concentrates, and Fresh Frozen Plasma.

In November 2022, the US FDA approved HEMGENIX (etranacogene dezaparvovec) developed by uniQure Pharmaceuticals. It is an adeno-associated virus vector-based gene therapy to treat adults with hemophilia B (congenital Factor IX deficiency) who currently use factor IX prophylaxis therapy or have a current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.

As per DelveInsight analysis, in 2022, the total hemophilia B market size in the 7MM was approximately USD 3.2 billion. HEMGENIX and fidanacogene elaparvovec work by delivering a functional copy of the defective gene coding for factor IX. There is tough competition between these two gene therapies as both aim to provide a cure for hemophilia B by addressing the underlying genetic defect. HEMGENIX is already approved in the US in 2023 whereas the FDA has set a Prescription Drug User Fee Act (PDUFA) goal date for fidanacogene elaparvovec in the second quarter of 2024. In the US and Japan, hemophilia B patients have shifted to Extended Half-Life (EHL) recombinant FIX treatment options compared to EU4 and the UK. Latest data from Europe suggests that still more than 50% of patients are receiving the recombinant FIX options with Standard Half-life (SHL).

The dynamics of the hemophilia B therapeutic market are anticipated to change in the coming years owing to the improvement in the rise in the number of healthcare spending across the world. Major Pharma giants are thoroughly working toward the development of new treatment therapies for this indication, in order to provide better relief for the symptoms and hence improve the Quality of life (QoL) of patients with hemophilia B.

Hemophilia B Pipeline Therapies and Key Companies

• Marstacimab (PF-06741086): Pfizer
• Fidanacogene elaparvovec (SPK-9001/PF-06838435): Pfizer/Spark Therapeutics
• Fitusiran (ALN-AT3, SAR-439774): Sanofi (Genzyme)/Alnylam Pharmaceuticals
• Concizumab (NN-7415): Novo Nordisk
• Verbrinacogene setparvovec (FLT-180a): Freeline Therapeutics
• SerpinPC (AP-0101): ApcinteX

For a comprehensive view of the hemophilia B market, check out the Hemophilia B Market Assessment

Von Willebrand Disease Market

Von Willebrand disease is the most prevalent hereditary bleeding disorder in the United States, impacting an estimated 1 in every 1,000 individuals. The unfortunate reality is that there is a substantial dearth of awareness within the patient community, their close-knit families, healthcare professionals, and the wider community, leading to detrimental effects on patients’ health and their overall quality of life. Von Willebrand disease is frequently likened to hemophilia, though it presents distinct characteristics and bleeding tendencies. It has an equal incidence in both males and females, unlike hemophilia, which predominantly affects males. A parent carrying the VWD gene has a 50% likelihood of transmitting it to their offspring. As per DelveInsight estimates, the total diagnosed von Willebrand disease prevalence was 12.5K in the US in 2020, which is further expected to increase with a significant CAGR by 2032.

The current approach to treating Von Willebrand disease in the United States encompasses three primary categories of drugs: Non-replacement Therapy, which involves the use of desmopressin (DDAVP) to boost vWF levels in the bloodstream; Replacement Therapy, utilizing plasma-derived coagulation factor concentrates such as Humate-P, Alphanate, Immunate, and Wilate; and Adjunct Therapy, primarily employing antifibrinolytics like Tranexamic Acid (Cyclokapron). 

In the realm of recombinant coagulation factor concentrates, a novel addition, Vonvendi by Shire, has recently emerged in the field of replacement therapy. Market contributors, including approved (along with their generic counterparts) and off-label products used in the treatment of von Willebrand disease, predominantly fall within these therapeutic categories. For non-replacement therapy options, Desmopressin has traditionally served as the primary treatment choice, particularly for patients with Type 1 variants and select type 2 cases. Notable medications in this category, all featuring desmopressin acetate as the active ingredient, encompass Stimate (CSL Behring GmbH), DDAVP (Sanofi-Aventis), and Octostim (CSL Behring GmbH).

DelveInsight’s analysts estimate that the von Willebrand disease market is expected to show positive growth and will grow from USD 309 million in 2020 at a significant CAGR by 2032. The dynamics of the von Willebrand disease market are anticipated to change in the coming years owing to the expanded use of Vonvendi (BAX 111) for prophylactic as well as pediatric therapies in the US. Its expanded approval is also expected to raise awareness among a large section of von Willebrand disease patients who have VWF defects and have moderate disease but are currently undertreated. Overall, the increasing diagnosed prevalence of the disease along with the promising expanded recombinant therapies will fuel the von Willebrand disease market during the forecasted period of 2023–2032.

Von Willebrand Disease Pipeline Therapies and Key Companies

• Emicizumab: Genentech, Inc.
• Wilate: Octapharma
• rVWF: Takeda

For a deeper understanding of the VMD market landscape, explore the Von Willebrand Disease Market Outlook

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SOURCE DelveInsight Business Research, LLP