주요 7개국의 데스모이드 종양 시장 규모는 2023년 약 8,900만 달러에 달할 것으로 예상됩니다. 이 시장은 예측 기간 동안 상당한 CAGR로 증가할 것으로 예상됩니다. 주요 7개국 중 미국이 2023년 전체 시장의 약 68%를 차지하며 가장 큰 시장 규모를 차지했으며, 2023년 주요 7개국에서 발생한 데스모이드 종양은 총 3,400건이었으며, 그 중 미국이 가장 많은 발병 건수를 기록했습니다.
SpringWorks Therapeutics(OGSIVEO(nirogacestat)), Immunome/Ayala Pharmaceuticals(AL102), Iterion Therapeutics 및 Apollomics(Tegavivint)(BC2059)), Eisai 및 PRISM BioLab(E7386)과 같은 주요 기업들은 각각 다른 임상 개발 단계에서 주요 후보 물질을 평가하고 있으며, 예측 기간(2024-2034년) 동안 데스모이드 종양 시장에 큰 영향을 미칠 것으로 예상됩니다.
데스모이드 종양은 100만 명당 2-6명꼴로 발생하는 드문 간엽계 신생물로, 국소 침습성이 특징이지만 전이성이 없는 것이 특징입니다. 결합조직의 섬유아세포에서 발생하며, 상처 치유와 구조적 지지에 중요한 역할을 합니다. '침습성 섬유종증'이라고도 불리는 이 종양은 주로 젊은 층에서 발생하며, 특히 가족성 대장선종증(FAP) 환자에서 APC 유전자의 돌연변이와 관련이 있습니다. 전이성은 없지만, 데스모이드 종양은 국소 침습성, 통증 및 외과 적 제거의 어려움으로 인해 심각한 이환율을 유발할 수 있습니다.
데스모이드 종양은 FAP, 특히 APC 돌연변이로 인한 복부 사례에서 더 흔합니다. 데스모이드 종양은 종종 과거에 수술을 받은 부위에 발생하며, 수술 병력은 알려진 위험 요소입니다. 예방적 대장 절제술을 받은 환자에서 데스모이드 종양은 대장암보다 이환율과 사망률에 더 큰 영향을 미칩니다. 데스모이드 종양은 임신 중 또는 임신 후 여성에서 더 자주 발생하며, 복부 외상 및 에스트로겐 농도 증가가 원인인 것으로 추정됩니다. 임신과 관련된 데스모이드 종양은 일반적으로 예후가 좋습니다.
MRI는 선호되는 영상 진단 방법이며, 중등도에서 중등도의 가돌리늄 증강 및 띠 징후를 나타내며, CT 검사는 복벽 및 복강 내 종양의 진단에 유용하며, 초음파 검사는 사지 또는 복벽 종양에 유용하며, PET-CT는 FAP 환자의 재발 암과 데스 모이 드 종양을 감별 할 수 있습니다.
이 보고서는 주요 7개국 데스모이드 종양 시장을 조사하여 시장 개요와 함께 역학, 환자 동향, 새로운 치료법, 2034년까지의 시장 규모 예측, 의료 미충족 수요 등을 제공합니다.
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.
Study Period: 2020-2034
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.
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.
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 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.
Key Updates
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.
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
Financial Assistance