2023년 건활막거대세포종 시장 규모는 미국이 주요 7개국 중 가장 큰 시장으로 약 2억 4,000만 달러에 달하며, 2034년까지 더욱 확대될 것으로 예상됩니다. 다이이찌산쿄의 투랄리오는 FDA가 승인한 유일한 건초염 거대세포종 치료제입니다. 2020년 6월, 유럽에서 TURALIO의 승인은 생명을 위협할 수 있는 간독성 부작용으로 인해 기각되었고, 유럽에서는 건초염 거대세포종에 대한 비임상적 치료제로서의 승인은 취소되었습니다. 치명적인 건막염 거대세포종 치료에서 위험-편익 비율이 불리한 것으로 판단했습니다.
건활막거대세포종은 활막에서 유래한 조직의 비정상적인 증식으로 면역 세포, 특히 대식세포의 활성화를 유발하여 종괴 형성을 유발하는 질환입니다. 이러한 종양은 종종 성장 패턴(국소 또는 미만성) 및 부위(관절 내 또는 관절 외)에 따라 분류됩니다.
건활막거대세포종의 진단은 특징적인 증상의 확인, 자세한 병력, 철저한 임상 평가 및 다양한 전문 검사를 기반으로 이루어집니다. 이러한 종양의 초기 증상은 종종 모호하고 인식되지 않을 수 있습니다. TGCT의 진단을 확정하기 위해 엑스레이나 MRI와 같은 영상 진단이 자주 사용되며, 특정 경우에는 생검을 통해 진단을 내리기도 합니다.
실제 데이터 분석에 따르면, 활액막 거대세포종 치료는 수술이 가장 바람직한 치료법입니다. 수술은 건활막거대세포종 관리에 매우 효과적이지만 수술 후 재발이 상당히 많습니다. 수술은 다른 면역요법에 비해 상대적으로 저렴하지만, 매우 비싼 수술과 관련된 다른 비용이 상당히 많이 듭니다. 이 때문에 결국 수술의 총 비용은 상당히 비싸게 됩니다.
앞으로 몇 년 동안 미국 TGCT 시장은 크게 변화할 것으로 예상되며, 향후 출시될 제품인 빔셀티닙, 엠팍투주맙, 피미코티닙, AMB-05X가 주도하는 성장세를 보일 것으로 예상되며, TURALIO의 안전성 및 내약성에 대한 우려로 인해 많은 환자들이 이마티닙과 같은 비적응성 TKI와 인플릭시맙과 같은 mAbs를 선호하고 있습니다. Vimseltinib, emactuzumab, pimicotinib, AMB-05X의 안전성 프로파일과 개선된 효능으로 인해 치료 현장에서 쉽게 채택될 수 있을 것으로 예상됩니다. 치료 현장에서 쉽게 채택될 것으로 기대됩니다.
이 보고서는 주요 7개국의 건활막거대세포종(TSGCT) 시장을 조사하여 시장 개요와 함께 역학, 환자 동향, 새로운 치료법, 2034년까지의 시장 규모 예측, 의료 미충족 수요 등을 조사하여 전해드립니다.
DelveInsight's "Tenosynovial Giant Cell Tumor (TGCT) - Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of the Tenosynovial Giant Cell Tumor, historical and forecasted epidemiology as well as the Tenosynovial Giant Cell Tumor market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
Tenosynovial Giant Cell Tumor market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Tenosynovial Giant Cell Tumor market size from 2020 to 2034. The report also covers current Tenosynovial Giant Cell Tumor treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.
Study Period: 2020-2034
Tenosynovial Giant Cell Tumor Overview
Tenosynovial Giant Cell Tumor is an abnormal growth of tissue derived from the synovium that causes activation of immune cells, specifically macrophages, leading to mass formation. These tumors are often classified by their growth pattern (localized or diffused) and location (intra-or extra-articular).
Recurrences are quite common in Tenosynovial Giant Cell Tumors, a major concern for decisions regarding treatment choice.
Tenosynovial Giant Cell Tumor Diagnosis
The diagnosis of tenosynovial giant cell tumor is based on identifying characteristic symptoms, a detailed patient history, a thorough clinical evaluation, and a variety of specialized tests. The initial symptoms of these tumors are often vague and may go unrecognized. Consequently, there is usually a significant delay from the onset of symptoms until a diagnosis is made. Imaging techniques like X-ray and MRI are frequently used to confirm the diagnosis of TGCT, but biopsy is also used in certain cases.
The current approach to diagnosing these patients is quite similar across the 7MM, where the journey begins with a referral to an ortho-oncologist by a primary care provider upon its diagnosis. Upon diagnosis, the patients are evaluated based on their tumor-growth pattern, and assessments are done to know if they are amenable to surgery.
Recurrences of localized tenosynovial giant cell tumors are observed more commonly in the EU4 countries and the UK than in countries of the 7MM, like the US and Japan.
Tenosynovial Giant Cell Tumor Treatment
Based on real-world data analysis, surgery is the most preferred treatment for tenosynovial giant cell tumors. Although surgery is quite effective for the management of tenosynovial giant cell tumors, recurrences after surgery are quite common. Despite surgeries being comparatively cheap compared to other immunotherapeutic options, there are significant other costs associated with very high surgeries. This ends up making the total cost of surgery quite expensive.
TURALIO is approved only in the US, while its approval in the EU got rejected. Daiichi Sankyo is also conducting trials to obtain its approval in Japan. TURALIO's Marketing Authorization Application (MAA) was rejected by the European Commission (EC) because of its unfavorable risk-benefit ratio. TURALIO had potentially fatal hepatotoxic effects, although used to treat a non-fatal condition. Even in the US, its use is heavily monitored through the Risk Evaluation and Mitigation Strategy (REMS) Program, and its availability is restricted to certain specific centers.
Over the next few years, the US TGCT Market is expected to change substantially and experience growth, as it will be dominated by upcoming products, vimseltinib, emactuzumab, pimicotinib, and AMB-05X. The safety and tolerability concerns of TURALIO are hindering its adoption in the treatment setting, with many patients preferring the usage of off-label TKIs like imatinib and mAbs like infliximab. Vimseltinib, emactuzumab, pimicotinib, and AMB-05X, with their significantly better safety profiles and improved efficacy, are expected to be readily adopted in the treatment setting.
As the market is derived using a patient-based model, the Tenosynovial Giant Cell Tumor epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by incident cases of Tenosynovial Giant Cell Tumor, growth-pattern specific incident cases of Tenosynovial Giant Cell Tumor, gender-specific incident cases of localized Tenosynovial Giant Cell Tumor, gender-specific incident cases of diffuse Tenosynovial Giant Cell Tumor, tumor localization of localized Tenosynovial Giant Cell Tumor, tumor localization of diffuse Tenosynovial Giant Cell Tumor, and the total treated cases of Tenosynovial Giant Cell Tumor in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), United Kingdom, and Japan from 2020 to 2034. The total prevalent cases of Tenosynovial Giant Cell Tumor in the 7MM are projected to increase during the forecasted period.
The drug chapter segment of the Tenosynovial Giant Cell Tumor report encloses a detailed analysis of Tenosynovial Giant Cell Tumor marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the Tenosynovial Giant Cell Tumor clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, each drug's advantages and disadvantages, and the latest news and press releases.
Marketed Drugs
TURALIO (pexidartinib): Daiichi Sankyo
TURALIO (pexidartinib) is an orally administered small molecule tyrosine kinase inhibitor that targets colony-stimulating factor 1 receptor (CSF1R), KIT proto-oncogene receptor tyrosine kinase (KIT), and FMS-like tyrosine kinase 3 (FLT3) harboring an internal tandem duplication (ITD) mutation. The US FDA approves it for treating adult patients with symptomatic tenosynovial giant cell tumors associated with severe morbidity or functional limitations which are not amenable to improvement with surgery.
TURALIO received approval for the treatment of tenosynovial giant cell tumor patients in August 2019 in the United States. A new dosing regimen of TURALIO was introduced in February 2023, where the 200 mg capsule was discontinued and replaced by a 125 mg capsule. The new recommended dose, which the FDA approved in October 2022, is 125 mg capsules taken twice daily with a low-fat meal, instead of the former 200 mg capsules taken twice daily on an empty stomach. This was done to reduce the toxic effects of the drug.
TURALIO is expected to face heavy competition from therapies such as Ono Pharmaceuticals' vimseltinib, SynOx Therapeutics' emactuzumab, Abbisko Therapeutics' pimicotinib, and AmMax Bio's AMB-05X, which have demonstrated significantly better safety and efficacy profiles than TURALIO in clinical trials.
Emerging Drugs
Vimseltinib (DCC-3014): Ono Pharmaceuticals
Vimseltinib (DCC-3014), which is being developed by Ono Pharmaceuticals, is an orally administered, potent, and highly-selective switch-control kinase inhibitor of the colony-stimulating factor 1 receptor (CSF1R) with potential antineoplastic, macrophage checkpoint-inhibitory and immunomodulating activities. The drug could be the best-in-class treatment for tenosynovial giant cell tumor patients. The Phase I/II study demonstrated promising preliminary results, encouraging antitumor activity with clinical benefits and manageable adverse events. These results supported its continued evaluation in a Phase III MOTION study, which, if successful, will lead to the FDA approval of the drug before 2026. Key opinion Leaders (KOLs) are convinced with the tolerability and antitumor activity of vimseltinib in clinical trials and have shown a preference toward vimseltinib over imatinib and pexidartinib, for the treatment of tenosynovial giant cell tumors.
Drug Class Insights
The existing Tenosynovial Giant Cell Tumor treatment is mainly dominated by classes such as CSF1R inhibitors and TNF alpha inhibitors.
The approval of TURALIO for treating tenosynovial giant cell tumors established the efficacy of colony-stimulating factor 1 receptor (CSF1R) targeting the treatment of TGCT. Although TURALIO established the efficacy of CSF1R inhibition, it also had concerns regarding its potentially fatal hepatotoxic effects. Hence emerging CSF1R inhibitors are currently focusing more on improving their safety profiles.
Although large-scale clinical trials have not established the effectiveness of TNF alpha inhibition, they are still used as off-label in certain cases to manage tenosynovial giant cell tumors.
CSF1R inhibitors dominate the upcoming treatment landscape.
Tenosynovial Giant Cell Tumor treatment in the US has entered a new era, with the dynamics expected to change. Surgery is the primary therapeutic option for Tenosynovial Giant Cell Tumors, but it is frequently accompanied by recurrences and hampering the patient's functional status. When the tumor is removed as a whole, the likelihood of the recurrence of the tumor is reduced; however, complete removal of the tumor sometimes requires the mandatory undesirable removal of the functional structures of the patient. But if the tumor is removed partially to maintain a patient's functional status, recurrences are much more likely to appear.
TURALIO provided a new treatment option for patients with a significant need for systemic treatment. Its approval was set to solve a huge unmet need for treatment in patients with severe morbidity who were not amenable to surgery. Although it faced significant challenges regarding its adoption in the market due to its concerning safety and tolerability profile, causing fatal hepatotoxic effects in a non-fatal conditions. Due to the safety concerns of turalio, off-label treatments like imatinib are used frequently to treat tenosynovial giant cell tumors, although their efficacy is very limiting.
Currently, TURALIO is also being explored in the perioperative setting, where it is used in the neoadjuvant setting for tumor shrinkage, to help in appropriate resection, and in the adjuvant setting to prevent recurrences post-resection.
Patients and physicians have long cited a desire for effective therapy for tenosynovial giant cell tumors without sacrificing its safety and tolerability. The expected launch of upcoming therapies and a high unmet need for a therapy with a better safety profile than TURALIO will eventually facilitate the development of effective treatment options. However, the diagnosis of tenosynovial giant cell tumor is often associated with diagnostic delays and misdiagnosis due to its slow-growing nature, not well-differentiated symptoms, and lack of awareness of the disease among patients and providers. These factors often become a hindrance for both physicians and patients when adopting newer therapies.
Key players such as Ono Pharmaceuticals (vimseltinib), SynOx Therapeutics (emactuzumab), and others are evaluating their lead candidates in different stages of clinical development respectively. They aim to investigate their products for the treatment of Tenosynovial Giant Cell Tumor.
This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034. For example, for vimseltinib, we expect the drug uptake to be medium with a probability-adjusted peak share of 20%; years to the peak is expected to be 8 years from the year of launch.
Tenosynovial Giant Cell Tumor 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 information on collaborations, acquisitions and mergers, licensing, and patent details for Tenosynovial Giant Cell Tumor emerging therapies.
KOL Views
To keep up with current market trends, we take KOLs and SME's opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on Tenosynovial Giant Cell Tumor evolving treatment landscape, patient reliance on conventional therapies, patient's therapy switching acceptability, and drug uptake along with challenges related to accessibility, include Medical/scientific writers, Medical Oncologists, Professors, Medical Oncologists of the MD Anderson Cancer Center, Orthopaedic Oncologists of the Uniklinik Essen, and Others.
Delveinsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers such as MD Anderson Cancer Center, Nuffield Orthopaedic Centre, Fondazione IRCCS Istituto Nazionale Tumori, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or Tenosynovial Giant Cell Tumor 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 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.
Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, administration frequency, administration route, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.
In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in Tenosynovial Giant Cell Tumor trials, one of the most important primary outcome measures is the objective response rate of the therapy on TGCT patients.
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. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Market Access and Reimbursement
Reimbursement of rare disease therapies can be limited due to lack of supporting policies and funding, challenges of high prices, lack of specific approaches to evaluating rare disease drugs given limited evidence, and payers' concerns about budget impact. The high cost of rare disease drugs usually has a limited impact on the budget due to the small number of eligible patients being prescribed the drug. The US FDA has approved several rare disease therapies in recent years. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products independently.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs etc.
Market Insights
Epidemiology Insights
Current Treatment Scenario, Marketed Drugs, and Emerging Therapies