Desmoid Tumors - Market Insight, Epidemiology, and Market Forecast - 2034

Key Highlights:

• The market size of desmoid tumors in the 7MM was around USD 89 million in 2023 and is expected to increase with a significant CAGR during the forecast period.
• Among the 7MM, the United States accounted for the largest market size of desmoid tumors, i.e., approximately 68% of the overall market in 2023.
• The total incident cases of desmoid tumors in the 7MM were ~3,400 in 2023, out of which the highest incident cases of this disease were in the United States.
• The mutation-specific cases of desmoid tumors in the 7MM were majorly contributed by CTNNB1 gene mutation with almost 28,400 cases, compared to APC gene mutation with ~3,000 cases in 2023.
• Current market is covered by the only US FDA approved drug OGSIVEO (nirogacestat) for the treatment of adult patients with progressing desmoid tumors who require systemic treatment as well as chemotherapy and targeted, systemic and other supportive therapies.
• Medical therapies are the second-line treatments for sporadic desmoid tumors located at all other sites and for all familiar desmoid tumor failing observation.
• Key players such as SpringWorks Therapeutics (OGSIVEO (nirogacestat)), Immunome/Ayala Pharmaceuticals (AL102), Iterion Therapeutics and Apollomics (Tegavivint (BC2059)), Eisai and PRISM BioLab (E7386), and others are evaluating their lead candidates in different stages of clinical development, respectively and will significantly impact the desmoid tumors market during the forecast period (2024-2034).
• The management of desmoid tumors has been an area of significant clinical change as the field has shifted away from aggressive surgical interventions to active surveillance and systemic therapies that are less morbid for patients.
• The dynamics of the desmoid tumors market are anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the disease, and incremental healthcare spending across the world.

DelveInsight's "Desmoid Tumors - Market Insight, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of desmoid tumor, historical and forecasted epidemiology as well as the desmoid tumors market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The desmoid tumors market report provides current treatment practices, emerging drugs, desmoid tumors market share of individual therapies, and current and forecasted desmoid tumors market size from 2020 to 2034, segmented by seven major markets. The report also covers current desmoid tumors treatment practices/algorithms 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

Desmoid Tumors Disease Understanding and Treatment Algorithm

Desmoid Tumors Overview

Desmoid tumors are rare mesenchymal neoplasms, occurring in 2 to 6 cases per million, characterized by local invasiveness but no metastatic potential. They arise from fibroblast cells in connective tissue, playing a key role in wound healing and structural support. Also called "aggressive fibromatosis," these tumors primarily affect younger individuals and are often associated with APC gene mutations, particularly in those with familial adenomatous polyposis (FAP). Although not metastatic, desmoid tumors can cause significant morbidity due to their local invasiveness, pain, and difficulty in surgical removal.

Desmoid tumors are more common in FAP, especially in abdominal cases caused by APC mutations. They often develop at sites of previous surgery, with prior surgery being a known risk factor. In patients undergoing prophylactic colectomy, desmoid tumors contribute more to morbidity and mortality than colon cancer. They are also more frequent in females during or after pregnancy, with abdominal trauma and elevated estrogen levels suggested as contributing factors. Pregnancy-related desmoid tumors generally have better outcomes.

Desmoid Tumors Diagnosis

Desmoid tumors may be misdiagnosed in 30%-40% of cases, with a study showing a 54% delay of over a year in diagnosis. Proper diagnosis is crucial but challenging. A multidisciplinary team, including oncologists, radiologists, and geneticists, is recommended for evaluation. Symptoms vary by location: extremity tumors may cause pain and limited motion, while intra-abdominal desmoids may lead to weight loss, cachexia, and malaise. Both sporadic and FAP-related desmoid tumors affect quality of life.

MRI is the preferred imaging method, showing moderate to strong gadolinium enhancement and a band sign. CT scans help diagnose abdominal wall and intra-abdominal tumors, while ultrasound is useful for extremity or abdominal wall tumors. PET-CT may differentiate recurrent cancer from desmoid tumors in FAP patients.

Histologically, desmoid tumors are firm, white or gray, resembling scar tissue. Biopsy analysis by an expert is needed to distinguish them from other neoplasms. Immunohistochemistry shows B-catenin positivity and other markers, with mutations in CTNNB1 or APC characteristic of desmoid tumors. CTNNB1 mutations exclude FAP, while APC mutations exclude sporadic desmoid tumors.

Desmoid Tumors Treatment

There is no standard approach for managing desmoid tumors, with most experts recommending observation for asymptomatic patients. For symptomatic patients, various treatments are considered. Over the past 20 years, medical management has advanced significantly. The goal of surgery is to preserve limb function and protect critical structures. While achieving an R0 resection is desirable, it is not essential. Positive margins (R1 or R2 resection) remain debated as a prognostic factor, and highly invasive surgery to achieve R0 is not warranted. Surgery is less commonly used due to high recurrence rates and morbidity.

With radiation and medical treatments, management has become more conservative. Radiation therapy is used as an adjuvant treatment when surgery leaves positive margins or is not possible. Doses exceeding 56 Gy are avoided due to complications. Nonrandomized studies suggest that adding radiation reduces local recurrence in patients with positive margins. Neoadjuvant radiation is not recommended. Systemic therapy is reserved for patients with rapid tumor growth or tumors threatening vital structures.

Desmoid tumors are generally resistant to chemotherapy, but certain regimens may offer benefits. Doxorubicin-based regimens have shown the best success rates. Doxorubicin combined with dacarbazine can induce partial responses, with slow tumor responses continuing months after treatment. In patients with FAP-associated desmoid tumors, adding nonsteroidal anti-inflammatory drugs like meloxicam can help. Liposomal doxorubicin has also been effective, inducing long-lasting responses. In pediatric patients, vinblastine combined with methotrexate has been beneficial, but it is toxic for adults, where vinblastine is replaced by vinorelbine. Imatinib (800 mg daily) has been effective for desmoid tumors, with positive responses even in patients without mutations in KIT, PDGRA, or PDGFRB. Sorafenib (400 mg daily) has extended progression-free survival in trials, with a 33% objective response rate compared to 20% in the placebo group. Common adverse events include rash, hypertension, diarrhea, and fatigue. Nirogacestat (150 mg twice daily) is approved by the US FDA for progressing desmoid tumors, showing significant benefits in progression-free survival, response rates, and symptom reduction. Common adverse events include diarrhea, nausea, fatigue, and rash. Hormonal therapy, often combined with COX-2 inhibitors, has become less favored and is no longer recommended.

Desmoid Tumors Epidemiology

The desmoid tumors epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the Incident cases of desmoid tumors, 10-year (mortality adjusted) prevalent cases of desmoid tumors, Mutation-specific cases of desmoid tumors, Gender-specific cases of desmoid tumors, and Tumor site-specific cases of desmoid tumors in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• Desmoid tumors account for <3% of soft tissue tumors. Their annual incidence is estimated to range between 1/250,000-1/500,000.
• In the US, an estimated 1,000-1,650 new patients are diagnosed with desmoid tumors yearly.
• In EU4 and the UK, the 10-year prevalent cases of desmoid tumors were ~4,500 cases in males and ~8,300 cases in females in 2023.
• Extra-abdominal cases were almost three time more than intra-abdominal cases for the year 2023, in Japan.
• More than 90% of cases of desmoid tumors are sporadic and associated with a mutation in the B-catenin gene (CTNNB1). A minority of desmoid tumors are diagnosed in patients with germline APC mutation, which manifests as FAP.
• Patients with desmoid tumors face many challenges, including delayed diagnosis or misdiagnosis due to lack of awareness by patients and providers; unpredictable disease course

Desmoid Tumors Drug Chapters

Marketed Drugs

OGSIVEO (nirogacestat): SpringWorks Therapeutics

OGSIVEO (nirogacestat) is an oral, selective, small molecule gamma secretase inhibitor developed by SpringWorks Therapeutics for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. SpringWorks is also evaluating nirogacestat as a potential treatment for patients with ovarian granulosa cell tumors and for patients with multiple myeloma as part of several B-cell maturation agent (BCMA) combination therapy regimens in collaboration with leaders in industry and academia.

OGSIVEO is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumor symptoms.

In November 2023, the US FDA approved OGSIVEO (nirogacestat), for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.

In February 2024, EMA validated the Marketing Authorization Application (MAA) for nirogacestat for the treatment of adults with desmoid tumors.

Emerging Drugs

AL102: Immunome/Ayala Pharmaceuticals

AL102 is an investigational small molecule gamma secretase inhibitor (GSI) designed to potently and selectively inhibit Notch 1, 2, 3, and 4. It is currently being evaluated in the Phase II/III RINGSIDE clinical studies in patients with progressing desmoid tumors. AL102 is designed to inhibit the expression of Notch gene targets by blocking the final cleavage step by the gamma-secretase required for Notch activation.

Ayala obtained an exclusive, worldwide license to develop and commercialize AL102 from Bristol-Myers Squibb Company in November 2017.

In November 2023, AL102 was granted Orphan Drug Designation (ODD) by the US FDA for the treatment of desmoid tumors. In September 2022, Ayala Pharmaceuticals reported that the US FDA has granted Fast Track designation (FTD) for AL102 for the treatment of progressing desmoid tumors

In March 2024, Immunome reported the successful completion of its purchase of AL102 and related drug candidate AL101 from Ayala Pharmaceuticals.

Tegavivint (BC2059): Iterion Therapeutics

Tegavivint is a potent and selective first-in-class small molecule inhibitor of Transducin Beta-like Protein One (TBL1), a novel downstream co-factor in the Wnt/beta-catenin signaling pathway. Increased expression of beta-catenin and TBL1 are associated with metastasis and poor prognosis in a broad range of tumor types. Tegavivint's targeting of TBL1 prevents TBL1/beta-catenin complex formation, specifically inhibiting beta-catenin's oncogenic transcriptional activity without disrupting key cell membrane functions that have been linked to toxicity common to other drugs in this pathway. The company completed Phase I clinical trial for Tegavivint in 2022.

In February 2021, Apollomics and Iterion Therapeutics announced an exclusive collaboration and license agreement to develop and commercialize Tegavivint in Greater China.

E7386: Eisai and PRISM BioLab

E7386 is a CBP/B-catenin inhibitor that inhibits protein-protein interactions between the transcription coactivator CREB-binding protein (CBP) and B-catenin, and regulates the Wnt signaling-dependent gene expression. Since E7386 acts on the CBP/B-catenin transcription complex located at the most downstream of the Wnt signaling, it is expected to inhibit not only ligand-dependent activation but also activation caused by gene mutations in Wnt signaling factors such as adenomatous polyposis coli (APC) and B-catenin.

February 2021, CBP/B-catenin inhibitor E7386, a medium-molecular weight compound created through collaboration research between Eisai and PRISM BioLab, achieved the clinical POC (Proof of Concept).

In April 2011, Eisai entered into a license and collaborative research and development agreement with PRISM BioLab concerning a CBP/B-catenin inhibitor and analogous compounds thereof

Drug Class Insight

Gamma Secretase Inhibitor (GSI)

Gamma secretase inhibitors (GSIs) are a class of compounds that target the gamma secretase complex, an essential protease involved in the cleavage of various transmembrane proteins, including the Notch receptors and amyloid precursor protein (APP). By inhibiting this complex, GSIs can modulate critical signaling pathways implicated in cancer and neurodegenerative diseases. AL102 interferes with the gamma secretase complex's ability to cleave Notch receptors. By blocking this pathway, AL102 can reduce tumor cell proliferation and promote apoptosis (programmed cell death).

TBL1 inhibitor

Transducin B-like protein 1 (TBL1) is a crucial component of the Wnt/B-catenin signaling pathway, which plays a significant role in various cellular processes, including gene transcription and cell proliferation. TBL1 interacts with B-catenin, facilitating its recruitment to Wnt target gene promoters, thereby promoting transcriptional activation. Tegavivint works by selectively disrupting the interaction between TBL1 and B-catenin. This disruption inhibits the nuclear translocation of B-catenin and promotes its degradation, thereby reducing B-catenin activity in cancer cells.

Desmoid Tumors Market Outlook

Desmoid tumor is a localized neoplasm with no metastatic potential but an unpredictable course. Asymptomatic patients can be monitored with a "watch and wait" approach, while symptomatic patients, especially those with Gardner's syndrome, may require systemic therapy. Surgery and hormonal therapy with or without NSAIDs are not first-line treatments. TKIs like sorafenib and GSIs like nirogacestat have shown significant benefits in Phase III trials, and new treatments targeting the Wnt pathway are in development. Cryoablation is promising for extra-abdominal desmoid tumors.

Surgery is recommended for active management, especially for abdominal wall desmoid tumors, with the goal of R0 resection. If R1 resection is unavoidable, it can be considered for functional or cosmetic reasons. For R1 resections, perioperative radiotherapy or reoperation is not recommended. If surgery isn't possible, moderate-dose radiotherapy can control the disease. Active surveillance with regular imaging is the standard approach unless progression or symptoms warrant treatment.

For desmoid tumors near critical structures, earlier treatment may be necessary. Treatment depends on the location: surgery is first for abdominal wall desmoid tumors, while systemic therapy is preferred for intra-abdominal desmoid tumors. Medical therapy is preferred for extremity and chest wall desmoid tumors, with surgery considered only for low-morbidity cases. Head & neck or intrathoracic desmoid tumors typically require medical therapy, with radiotherapy as an alternative in some cases.

FAP-associated desmoid tumors (Gardner syndrome) are more aggressive and require more intensive treatment. Biopsy should be used cautiously in patients with APC mutations. Treatment options include antihormonal therapies, NSAIDs, TKIs, and chemotherapy. TKIs like sorafenib and pazopanib are effective, with sorafenib showing the best results in trials. Chemotherapy options, including low-dose methotrexate or vinblastine, have response rates of 35-40%, with long-term disease control in many cases.

• Among the 7MM, the US accounted for the largest market size of desmoid tumor. i.e., USD ~60 million in 2023.
• Among EU4 and the UK, France accounted for the highest market size in 2023, while Spain occupied the lowest.
• The pipeline for desmoid tumors is limited and consist of therapies including Gamma Secretase Inhibitor (GSI), TBL1 inhibitor, CBP/B-catenin interaction inhibitor and others.

Key Updates

• In November 2024, SpringWorks updated about long-term efficacy and safety data from Phase III DeFi Trial, that longer-term treatment with nirogacestat was associated with further reductions in tumor size, increase in objective response rate (ORR) with additional partial responses (PRs) and complete responses (CRs), sustained improvements in desmoid tumor symptoms including pain, and a consistent safety profile compared to the April 2022 data cut utilized for the primary results of the trial.
• In November 2023, AL102 was granted Orphan Drug Designation (ODD) by the US FDA for the treatment of desmoid tumors.

Immunome expects to publish topline data for RINGSIDE Part B in the second half of 2025. In parallel, the company is also evaluating and performing the additional manufacturing and pharmacology work required to support a New Drug Application (NDA) submission.

Desmoid Tumors Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034. The landscape of desmoid tumors treatment has experienced an uptake of novel drugs. These innovative therapies are redefining standards of care. Furthermore, the increased uptake of these transformative drugs is a testament to the unwavering dedication of physicians, oncology professionals, and the entire healthcare community in their tireless pursuit of advancing treatment care. This momentous shift in treatment paradigms is a testament to the power of research, collaboration, and human resilience.

Desmoid Tumors Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I stage. 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 desmoid tumors 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. Some of the leaders like MD, Professor and Vice Chair Department of Critical Care Medicine and Director, PhD, and others. Their opinion helps to understand and validate current and emerging therapies and treatment patterns or desmoid tumors market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Delveinsight's analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the Mount Sinai Fuster Heart Hospital, Stanford Medicine, University School of Medicine in Atlanta, University of Florida, Sarcoma Medical Oncology, University of Texas, MD Anderson Cancer Center, University of California, University of Colorado Medicine in Aurora, University of Miami Health System in Florida, etc., were contacted. Their opinion helps understand and validate desmoid tumors epidemiology and market trends.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT and conjoint analysis. 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

OGSIVEO - Patient Support

SpringWorks CareConnections provides personalized support services and resources for patients taking OGSIVEO.

Coverage and Access Support

• Resources and assistance to support timely access to OGSIVEO
• Quick Start Program-SpringWorks CareConnections can help eligible commercially insured patients who are experiencing a qualified delay in their insurance coverage get started on OGSIVEO at no cost for a limited period of time.
• Bridge Program-Eligible commercially insured patients currently on OGSIVEO experiencing a qualified lapse in their insurance coverage may be able to receive OGSIVEO at no cost for a limited period of time.

Financial Assistance

• Connecting patients to financial assistance options that may be available.
• Commercial Copay Program-Eligible patients with commercial insurance may pay as little as a USD 0 copay for OGSIVEO.

Scope of the Report:

• The report covers a descriptive overview of desmoid tumor, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into desmoid tumors epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for desmoid tumors is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of the desmoid tumors 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 desmoid tumors market.

Desmoid Tumors Report Insights

• Patient Population
• Therapeutic Approaches
• Desmoid Tumors Pipeline Analysis
• Desmoid Tumors Market Size and Trends
• Market Opportunities
• Impact of Upcoming Therapies

Desmoid Tumors Report Key Strengths

• Eleven Years Forecast
• 7MM Coverage
• Desmoid Tumors Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

Desmoid Tumors Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs:

• What was the desmoid tumors market share (%) distribution in 2020 and what it would look like in 2034?
• What would be the desmoid tumors total market size as well as market size by therapies across the 7MM during the study period (2020-2034)?
• Which country will have the largest desmoid tumors market size during the study period (2020-2034)?
• What are the disease risks, burdens, and unmet needs of desmoid tumor?
• What is the historical desmoid tumors patient pool in the United States, EU4 (Germany, France, Italy, and Spain), and the UK, and Japan?
• What will be the growth opportunities across the 7MM concerning the patient population of desmoid tumor?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of desmoid tumor?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and licensing activities related to desmoid tumors therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• What are the clinical studies going on for desmoid tumors and their status?
• What are the key designations that have been granted for the emerging therapies for desmoid tumor?

Reasons to buy:

• The report will help in developing business strategies by understanding trends shaping and driving desmoid tumor.
• To understand the future market competition in the desmoid tumors market and Insightful review of the SWOT analysis of desmoid tumor.
• Organize sales and marketing efforts by identifying the best opportunities for desmoid tumors in the US, EU4 (Germany, France, Italy, and Spain), 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 desmoid tumors market.
• To understand the future market competition in the desmoid tumors market.