Esophageal Cancer - Market Insight, Epidemiology And Market Forecast - 2034

Esophageal Cancer - Market Insight, Epidemiology And Market Forecast - 2034

Key Highlights

In 2023, the market size of hemophilia A was the largest in the US among the 7MM, accounting for approximately USD 5,000 million, which is further expected to increase by 2034.

In September 2023, concizumab was approved in Japan under the brand name ALHEMO for treating hemophilia A and B with inhibitors. Novo Nordisk expects to launch ALHEMO in Japan during the first half of 2024.

In June 2023, the US FDA approved ROCTAVIAN by BioMarin Pharmaceutical for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

BioMarin Pharmaceutical announced that the first commercial patient with ROCTAVIAN was treated in Germany in the third quarter of 2023, followed by a second commercial patient in November 2023.

Sanofi is anticipating the submission timeline of fitusiran in 2024 for hemophilia A and B and 2025+ for hemophilia A and B pediatric.

The emerging pipeline for hemophilia A includes several potential drugs in the late and early stages (Phase III and I/II) that include dirloctocogene samoparvovec (RG6357 or SPK-8011), fitusiran (ALN-AT3, SAR-439774), marstacimab (PF-06741086), and others, which can bring about significant change in the market during the forecast period.

DelveInsight’s “Hemophilia A – Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of the Hemophilia A, historical and forecasted epidemiology as well as the Hemophilia A market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

Hemophilia A market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Hemophilia A market size from 2020 to 2034. The report also covers current Hemophilia A treatment practice/algorithm and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered

The United States

EU4 (Germany, France, Italy, and Spain), and the United Kingdom

Japan

Study Period: 2020–2034

Hemophilia A Disease Understanding and Treatment Algorithm

Hemophilia A Overview

Hemophilia A is a genetic bleeding disorder in which an individual lacks or has low levels of proteins named clotting factor VIII. The mainstay treatment option has long been FVIII replacement therapy. Initially, FVIII replacement was accomplished by donated whole blood, subsequently by plasma, and currently by recombinant human FVIII (rFVIII) replacement therapies, which revolutionized the treatment of Hemophilia A. Although hemophilia is usually diagnosed at birth, the disorder can also be acquired later in life if the body begins to produce antibodies that attack and destroy clotting factors. However, this acquired type of hemophilia is very rare.

Hemophilia A Diagnosis

The symptoms of Hemophilia A can vary greatly from one person to another; it ranges from mild to moderate to severe. The age of onset and frequency of bleeding episodes depend upon the amount of factor VIII protein and the overall clotting ability of the blood. In most individuals, regardless of severity, bleeding episodes tend to be more frequent in childhood and adolescence than in adulthood.

Additionally, the diagnosis of Hemophilia A depends on the identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation, and a variety of specialized laboratory tests. The identification of a hemizygous F8 pathogenic variant on molecular genetic testing in a male proband confirms the diagnosis.

Hemophilia A Treatment

About 30% of severe Hemophilia A patients develop neutralizing anti-FVIII alloantibodies (inhibitors), which render the FVIII replacement ineffective. The standard of care therapy for patients with inhibitors is to induce immune tolerance with high-dose, high-frequency FVIII and treatment with bypassing agents (e.g., recombinant activated factor VII such as NOVOSEVEN, FEIBA). There are many approved therapies for the management of Hemophilia A, which include ALTUVIIIO, ROCTAVIAN, HEMLIBRA, ALHEMO, and others.

Further details related to diagnosis and treatment are provided in the report…

Hemophilia A Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of hemophilia A, age-specific prevalent cases of hemophilia A, severity-specific prevalent cases of hemophilia A, prevalent cases of hemophilia A with or without inhibitors and the treated patient pool of hemophilia A in the 7MM market covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

The epidemiology segment also provides the Hemophilia A epidemiology data and findings across the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The total prevalent population of Hemophilia A in the 7MM comprised nearly 45,500 cases in 2023 and is projected to increase during the forecast period.

The total prevalent population of Hemophilia A in the United States was nearly 14,300 in 2023.

The United States contributed to the largest prevalent population of Hemophilia A, accounting for ~30% of the 7MM in 2023.

In EU4 and the UK, the diagnosed prevalence of Hemophilia A was found to be maximum in France, followed by the UK. While the least number of cases were found in Spain in 2023.

In Japan, the total prevalent population of Hemophilia A was nearly 5,600 in 2023 and is anticipated to rise during the forecast period.

In 2023, the prevalent cases of mild Hemophilia A in the United States were identified to be ~3,600, moderate as ~2,900, and severe as ~7,900.

In the US, nearly 2,400 cases accounted for inhibitors and ~12,000 cases for non-inhibitors in 2023.

Hemophilia A Drug Chapters

The drug chapter segment of the Hemophilia A report encloses the detailed analysis of Hemophilia A mid and late-stage pipeline drugs. It also helps understand the Hemophilia A clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug, and the latest news and press releases.

Marketed Drugs

ROCTAVIAN (valoctocogene roxaparvovec)

ROCTAVIAN (valoctocogene roxaparvovec) is an AAV5 gene therapy for treating severe hemophilia A. It delivers a functional gene designed to enable the body to produce Factor VIII independently without needing continued hemophilia prophylaxis, thus relieving patients of their treatment burden relative to currently available therapies. People with hemophilia A have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting. The expressed hFVIII-SQ replaces the missing coagulation factor VIII needed for effective hemostasis. Following valoctocogene roxaparvovec infusion, vector DNA is processed in vivo to form full-length, episomal transgenes that persist as the stable DNA forms that support the long-term production of hFVIII-SQ.

OBIZUR (susoctocog alfa): Takeda

OBIZUR treats bleeding episodes in adults with acquired hemophilia (a bleeding disorder caused by a lack of Factor VIII activity due to antibody development). These antibodies have a less neutralizing effect against OBIZUR than against human Factor VIII. OBIZUR contains the active substance susoctocog alfa, antihemophilic Factor VIII, and porcine sequence.

Emerging Drugs

Fitusiran: Sanofi

Fitusiran, currently under development by Sanofi (Genzyme)/Alnylam Pharmaceuticals, is a subcutaneously administered small interfering RNA (siRNA) technology to target antithrombin. In Q2 2022, the company announced data with lower doses expected in the second half of 2023. The company anticipates the first filing by 2024. The Pediatric (age 1–11) study is ongoing, and the company expects the first filing by 2026.

Marstacimab: Pfizer

Marstacimab, which is currently under development by Pfizer, is a Tissue Factor Pathway Inhibitor monoclonal antibody to treat Hemophilia A and B with or without Inhibitors. Currently, it is in Phase III clinical trial for the treatment of severe Hemophilia A and B with or without Inhibitors. The company also projected a pivotal readout in Q2 2023 and projected submission for non-inhibitor indication in Q3 2023.

Note: Detailed therapy assessment will be provided in the final report.

Drug Class Insights

In the market, there are several third-generation products available; ADVATE (Baxter) and XYNTHA (Pfizer) are examples of recombinant factor VIII products. There are several other products approved under this category; however, these products were approved a long time ago, and since then, short and longer-acting recombinant (not derived from human plasma) factor therapies have entered the market, of which ELOCTATE and ADVATE have gained the popularity, in terms of treatment horizon of Hemophilia A. ELOCTATE is a recombinant fusion protein that temporarily replaces the missing Coagulation Factor VIII needed for effective hemostasis. KOGENATE FS is a new formulation of recombinant factor VIII formulated with sucrose, as opposed to human plasma protein (Albumin). Another class of factor concentrate is plasma-derived factor concentrate FVIII products that are produced by utilizing human plasma. But this class of products acquires less market share because of their less usage in comparison to the recombinant-derived factor concentrates. Several drugs are approved under this category, such as FEIBA (Pfizer), MONOCLATEP (CSL Behring), and HEMOFIL-M (Baxter).

Hemophilia A Market Outlook

In the market, several recombinant factor VIII (FVIII) products are available with high-specific activities (the amount of desired clotting factor per mg of total protein). Plasma-derived clotting factors products are also available. However, the current market is mainly dominated by the recombinants of several generations (recombinant third-generation and recombinant second-generation). Although several products are available at present, then again, none of these products might be able to cure or manage this situation, completely. HEMLIBRA—a product of “Genentech/Chugai/Roche,” is a novel bispecific antibody that was first approved in 2017 for patients with Hemophilia A with FVIII inhibitors in the US. However, in 2018, HEMLIBRA was approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with Hemophilia A without factor VIII inhibitors. This therapy is also approved for treatment for people with Hemophilia A without or without inhibitors in Europe as well as in Japan. At the start of 2023, the FDA approved ALTUVIIIO for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management for adults and children with hemophilia A. Later, in June 2023, the FDA approved ROCTAVIAN, a gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. Both the drug’s efficacy and safety are better than HEMLIBRA. Currently, the treatment hemisphere is mainly driven by non-inhibitor drug candidates. However, 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. Key players, like Novo Nordisk, Sanofi, Pfizer/Sangamo, Roche/Spark Therapeutics, and others, are coming up with novel therapeutic approaches that can entirely change the treatment landscape of Hemophilia A.

Key Findings

This section includes a glimpse of the Hemophilia A in the 7MM

The total market size of Hemophilia A in the 7MM is nearly USD ~11,200 USD million in 2023 and is projected to grow during the forecast period (2024–2034).

According to DelveInsight’s estimates, the largest market size of Hemophilia A is from the United States in 2023.

In EU4 and the UK, France has the maximum revenue share, followed by the United Kingdom in 2023 while Spain had the lowest market share.

The market size of Hemophilia A in Japan was USD ~1,200 million in 2023, which is expected to rise during the forecast period (2024–2034).

The upcoming therapies for Hemophilia A are expected to combat the current unmet needs faced by patients with Hemophilia A.

Hemophilia A Drugs Uptake

This section focuses on the rate of uptake of the potential drugs expected to be launched in the market during the study period 2020–2034. The analysis covers Hemophilia A market uptake by drugs, patient uptake by therapies, and sales of each drug. Sanofi/Alnylam is developing fitusiran, an antithrombin inhibitor for the treatment of hemophilia A and B (with/without inhibitors). The advantages of Fitusiran’s approach are mostly the same as anti-TFPI antibodies. With improved convenience to subcutaneous, once monthly dosing and potentially every 2 months, the drug has differentiated efficacy in inhibitor patients; since the drug does not target FVIII/FIX, it can bypass the threat posed by inhibitors. Fitusiran’s demonstrated ability to achieve FVIII levels significantly higher than what is possible on current therapies, notably with subcutaneous monthly dosing, has generated excitement among physicians. The primary question is safety, particularly when patients have breakthrough bleeds and require additional therapies.

Hemophilia A Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II, Phase III, and Phase I stages. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers detailed information on collaborations, acquisitions and mergers, licensing, and patent details for Hemophilia A emerging therapies.

KOL- Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging therapies and treatment patterns or Hemophilia A market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

The analyst analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.

In efficacy, the trial’s primary and secondary outcome measures are evaluated. Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials.

Market Access and Reimbursement

Reimbursement is a crucial factor affecting the drug’s market access. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, payers and other industry insiders are considering many payment models. One can obtain the initial 30-day supply of ALTUVIIIO typically within 24–48 h upon presenting a valid prescription from the healthcare provider. During the collaborative decision-making process between the individual and the doctor regarding suitable treatment, the assigned Sanofi professional will assess health insurance information.

Scope of the Report

The report covers the descriptive overview of Hemophilia A, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.

Comprehensive insight has been provided into the Hemophilia A epidemiology and treatment.

Additionally, an all-inclusive account of both the current and emerging therapies for Hemophilia A is provided, along with the assessment of new therapies that will have an impact on the current treatment landscape.

A detailed review of the Hemophilia A market, historical and forecasted, is included in the report, covering the 7MM drug outreach.

The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM Hemophilia A market.

Report Highlights

In the coming years, the Hemophilia A market is set to change due to emerging therapies in the pipeline and incremental healthcare spending across the world, which would expand the size of the market to enable drug manufacturers to penetrate more into the market.

The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia A R&D. The therapies under development are focused on novel approaches to treat or improve the disease condition.

The report also encompasses other major segments, i.e., age-specific, severity-specific cases of hemophilia A, prevalent pool based on inhibitor and non-inhibitor segment, and treated pool of Hemophilia A.

Expected launch of potential therapies, Fitusiran (Sanofi), SPK-8011 (Spark Therapeutics), and others might change the landscape in the treatment of Hemophilia A.

Currently, there are many approved therapies targeting the non-inhibitor segments. The current emerging pipeline consists of a number of novel candidates targeting both inhibitor and non-inhibitor segments. Many drugs are also being studied with subcutaneous route of administration.

Hemophilia A Report Insights

Patient Population

Therapeutic Approaches

Hemophilia A Pipeline Analysis

Hemophilia A Market Size and Trends

Market Opportunities

Impact of Upcoming Therapies

Hemophilia A Report Key Strengths

11 Year Forecast

7MM Coverage

Hemophilia A Epidemiology Segmentation

Key Cross Competition

Highly Analyzed Market

Drugs Uptake

Hemophilia A Report Assessment

Current Treatment Practices

Unmet Needs

Pipeline Product Profiles

Market Attractiveness

SWOT

Conjoint Analysis

Key Questions

FAQs

What was the Hemophilia A market share (%) distribution in 2020, and what it would look like in 2034?

What would be the Hemophilia A total market size as well as market size by therapies across the 7MM during the study period (2020–2034)?

What are the key findings pertaining to the market across the 7MM, and which country will have the largest Hemophilia A market size during the study period (2020–2034)?

At what CAGR, the Hemophilia A market is expected to grow at the 7MM level during the study period (2020–2034)?

What would be the Hemophilia A market outlook across the 7MM during the study period (2020–2034)?

What would be the Hemophilia A market growth till 2034, and what will be the resultant market size in 2034?

How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

What are the disease risk, burden, and unmet needs of Hemophilia A?

What is the historical Hemophilia A patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the UK, and Japan?

What would be the forecasted patient pool of Hemophilia A at the 7MM level?

What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Hemophilia A?

Out of the above-mentioned countries, which country would have the highest prevalent population of Hemophilia A during the study period (2020–2034)?

At what CAGR is the population expected to grow across the 7MM during the study period (2020–2034)?

Reasons to buy

The report will help in developing business strategies by understanding trends shaping and driving the Hemophilia A market.

To understand the future market competition in the Hemophilia A market and an insightful review of the key market drivers and barriers.

Organize sales and marketing efforts by identifying the best opportunities for Hemophilia A in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.

Organize sales and marketing efforts by identifying the best opportunities for the Hemophilia A market.

To understand the future market competition in the Hemophilia A market.