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혈관 주위 상피 세포종(PEComa) : 시장 인사이트, 역학 및 예측(2036년)

PEComa - Market Insights, Epidemiology, and Market Forecast - 2036

발행일: | 리서치사: 구분자 DelveInsight | 페이지 정보: 영문 117 Pages | 배송안내 : 2-10일 (영업일 기준)

    
    
    




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혈관 주위 상피 세포종(PEComa)에 대한 인사이트 및 동향

  • DelveInsight의 분석에 따르면, 2025년 주요 시장(미국, EU4(독일, 프랑스, 이탈리아, 스페인), 영국 및 일본)에서 혈관 주위 상피 세포종(PEComa) 시장 규모는 약 4,300만 달러인 것으로 나타났습니다.
  • PEComa는 소아부터 노인에 이르기까지 모든 연령층에서 발병이 확인되고 있습니다. 결절성 경화증(TSC)을 앓고 있는 소아에게서 흔히 나타나지만, 알려진 위험 요인이 없는 사람들에게서도 산발적으로 발생합니다. 악성 PEComa의 진단율은 인구 100만 명당 0.12-0.24명으로 추정됩니다.
  • PEComa의 치료 관리는 주로 종양의 절제 가능성과 분자적 특성에 기반한 맞춤형 접근법을 통해, 지속적인 질병 통제와 진행 예방에 중점을 두고 있습니다. 현재의 치료 전략에는 국소 병변에 대한 외과적 절제술 외에도, 시롤리무스나 에베로리무스 등의 mTOR 억제제, 특정 증례에 대한 세포독성 화학요법, 그리고 진행성 또는 난치성 질환에 대한 KEYTRUDA 등의 면역관문억제제를 포함한 새로운 치료법이 포함됩니다.
  • 현재 PEComa 치료제로 승인된 약물은 단 하나뿐입니다. 미국 식품의약국(FDA)은 2021년 11월에 FYARRO(시롤리무스-단백질 결합형)를 승인했습니다. 시롤리무스, 에베롤리무스, 템시롤리무스는 현재 미국 종합암네트워크(NCCN) 지침에서 PEComa에 대한 유효성이 인정된 전신 치료제로 기재되어 있습니다.
  • KEYTRUDA는 PEComa에 대한 면역관문억제제로서, 특히 진행성 또는 난치성 사례에 대한 연구가 진행되고 있으며, 초기 보고에 따르면 PD-L1을 발현하는 종양이나 돌연변이 부하가 높은 종양에서 항종양 활성이 시사되고 있으나, 그 사용은 여전히 임상시험 단계에 있으며 적응증 외 사용에 해당합니다. 치료 관리에는 국소 병변에 대한 외과적 절제, mTOR 억제제(시롤리무스, 에베로리무스) 및 특정 사례에서의 세포독성 화학요법도 포함되며, 이는 개별화된 분자 기반 치료 접근법을 반영하고 있습니다.

주요 7개국 혈관 주위 상피 세포종(PEComa) 시장 규모 및 전망

  • 혈관 주위 상피 세포종(PEComa) 시장 규모(2025년) : 약 4,500만 달러
  • 혈관 주위 상피 세포종(PEComa) 시장 규모 전망 : 1억 3,500만 달러
  • 혈관 주위 상피 세포종(PEComa) 시장 성장률(2026-2036년) : 연평균 성장률(CAGR) 8.1%

본 '혈관 주위 상피 세포종(PEComa)' 시장 보고서는 표준 치료, 임상 실무 및 진화하는 치료 알고리즘을 포함하여 현재 치료 시장 상황에 대한 종합적인 분석을 제공합니다. 본 보고서에서는 혈관 주위 상피 세포종(PEComa)의 환자 부담 동향, 수익 및 시장 점유율 동향, 정점 시 환자 점유율 및 치료법 보급 현황에 대한 분석을 평가함과 동시에, 전 세계 각 지역 시장 규모에 대한 상세한 평가 및 성장률 예측(과거 데이터 및 2022-2036년 예측)을 제공합니다. 본 보고서는 혈관 주위 상피유사 세포종(PEComa) 분야에서 주요 미충족 의료 수요를 부각시키고, 경쟁 구도 및 임상 현황을 분석하여 고부가가치 성장 기회를 도출함으로써, 향후 시장 성장 가능성에 대한 명확한 전망을 제시하고 있습니다.

혈관 주위 상피 세포종(PEComa) 시장을 주도하는 주요 요인

PEComa 발병률 증가

PEComa의 유병률 증가는 시장 확대의 주요 촉진요인이며, 환자 수는 2036년까지 상당한 연평균 성장률(CAGR)로 증가할 것으로 예측됩니다.

PEComa에서 표적 치료 및 병용 요법의 기회 확대

FYARRO(나브-시롤리무스)가 FDA 승인을 받은 유일한 전신 요법인 가운데, mTOR 억제제, VEGFR TKI, PD-1 억제제 등 새로운 치료 옵션의 등장으로 진행성 PEComa에 대한 표적 치료 및 병용 요법의 기회가 확대되고 있습니다.

혈관 주위 상피 세포종(PEComa)의 이해와 치료 알고리즘

혈관 주위 상피 세포종(PEComa)의 개요 및 진단

혈관 주위 상피양 육종은 멜라노사이트양 및 평활근양 특성을 모두 지닌 혈관 주위 상피양 세포를 특징으로 하는 희귀한 간엽성 종양입니다. 여러 장기(특히 자궁, 신장, 간, 후복막)에 발생할 수 있으며, 결절성 경화증과 관련이 있는 경우도 있습니다. 환자는 무증상인 경우가 많지만, 증상은 종양의 위치나 크기에 따라 달라집니다.

혈관 주위 상피 세포종(PEComa)의 진단

혈관 주위 상피양 세포 신생물의 진단은 주로 조직병리학적 검사 및 면역조직화학 검사에 근거합니다. 종양 세포는 특징적인 혈관 주위 상피양 형태를 보이며, 멜라노사이트 마커(HMB-45, Melan-A)와 평활근 마커(평활근 액틴)를 동시에 발현합니다. 영상 진단(CT 또는 MRI)은 종양의 위치와 확산 범위를 파악하는 데 도움이 되지만, 확정 진단을 위해서는 생검이 필요합니다. 악성 가능성을 판단하기 위해서는 종양의 크기, 괴사, 유사분열 활동, 침윤 등의 특징을 평가하는 것이 중요합니다.

혈관 주위 상피 세포종(PEComa)의 치료

혈관 주위 상피양 세포 신생물의 치료는 국소성 병변에 대해 절제면 음성 상태를 확보한 외과적 절제를 중심으로 이루어지며, 이는 여전히 주요한 근치적 치료법이지만 재발할 가능성이 있습니다. 진행성, 절제 불가능한 사례 또는 전이성 사례에 대해서는 FYARRO가 FDA 승인을 받은 유일한 전신 요법이며, 나노입자 제제를 통해 시롤리무스를 전달함으로써 종양에 대한 침투성이 향상되어 지속적인 임상적 효능을 보이고 있습니다. 그 밖의 mTOR 억제제(시롤리무스, 에베로리무스, 템시롤리무스)는 특히 FYARRO를 구할 수 없는 경우 등의 대체 요법으로 사용됩니다. 화학요법과 방사선 요법은 주로 완화적인 역할을 하지만, 그 효과는 제한적입니다. 한편, 표적 치료(VEGF/VEGFR 억제제)는 특정 증례에서 고려될 수 있습니다. 전반적으로 종양의 경과에 편차가 있기 때문에 치료에는 전문적인 관리와 치밀한 경과 관찰이 필요한 경우가 많습니다.

혈관 주위 상피 세포종(PEComa)의 역학

혈관 주위 상피 세포종(PEComa)의 역학 분석 및 예후에 관한 주요 조사 결과

  • DelveInsight사의 추산에 따르면, 2025년 주요 7개국에서 PEComa의 총 유병 건수는 약 408건이었습니다.
  • EU4 및 영국 중에서 PEComa 발생률이 가장 높은 국가는 독일이며, 그 다음으로 프랑스, 영국 순입니다.
  • 2025년 미국의 혈관 주위 상피유사 세포종(PEComa) 병기별 분포를 보면, 양성 종양이 약 67%를 차지하여 가장 많았고, 그 다음으로 국소 침습성(악성 PEComa)이 약 33%를 차지했습니다.
  • PEComa의 경우 여성에서 발병 비율이 높고 발병 연령도 낮은 경향이 확인되고 있는 만큼, 특히 감수성 패턴을 파악하고 장기적인 합병증을 예방하기 위해 발병 초기부터 질환에 대처해야 한다는 관점에서 성별에 따른 선별 검사 및 조기 개입 전략의 중요성이 부각되고 있습니다.

혈관 주위 상피 세포종(PEComa) 시장 전망

혈관 주위 상피 세포종(PEComa) 시장은 수술이나 제한적인 전신 요법에 의존하던 방식에서 벗어나, 보다 표적화된 작용기전에 기반한 치료 방식으로 전환되고 있습니다. FYARRO의 승인은 중요한 이정표이며, mTOR 경로 억제의 유효성이 입증됨에 따라 진행성 또는 전이성 질환의 치료 성과가 향상되었습니다. mTOR 억제제, 면역관문억제제 및 항혈관신생 요법에 대한 연구가 진행됨에 따라, 치료 전망은 다른 희귀암과 마찬가지로 진화하고 있으며, 조기 표적 치료제의 승인이 추가적인 혁신과 파이프라인 확장을 주도하고 있습니다. 이러한 진전에 따라 치료 선택지가 확대되고, 정밀 의학에 기반한 치료 전략이 촉진되고 있습니다.

미국은 여전히 주요 시장이며, 그 배경에는 해당 질환에 대한 높은 인지도, 뛰어난 진단 능력, 전문 육종 센터에 대한 접근성, 그리고 새로운 치료법의 신속한 도입이 있습니다. 전반적으로, 분자진단, 표적 치료 및 희귀암 연구의 발전이 꾸준한 시장 성장과 환자 예후 개선을 뒷받침할 것으로 예측됩니다.

  • 추산에 따르면, 혈관 주위 상피 세포종(PEComa) 시장 규모가 가장 큰 곳은 미국이며, 2036년까지 약 1억 1,400만 달러에 달할 것으로 전망됩니다.
  • PEComa의 치료 양상은 기존의 치료법에서 분자 표적 치료를 중심으로 한 전략으로 전환됨에 따라 변화하고 있습니다. TSC1/TSC2 변이를 가진 종양에서 효능이 입증된 시롤리무스나 에베롤리무스 등의 mTOR 억제제 사용이 확대되는 한편, 진행성 또는 난치성 사례에서는 키트루다 등의 면역요법이나 그 밖의 정밀의료 접근법의 새로운 활용이 주목받고 있습니다.
  • PEComa의 지역별 시장 동향을 살펴보면, 매출액 기준으로는 미국이 1위를 차지하고 있는 반면, 일본 및 유럽연합(EU) 시장은 꾸준한 성장세를 보이고 있습니다. 이는 진단에 대한 인식 제고, 표적 치료에 대한 접근성 확대, 그리고 희귀암 치료 분야에서 새로운 치료 접근법의 도입 확대를 반영한 것입니다.
  • 2036년에는 PEComa에 대한 모든 치료법 중에서 미국에서 FYARRO가 가장 높은 매출을 올릴 것으로 추정됩니다.
  • mTOR 억제제 : 시롤리무스-단백질 결합형(ABI-009, FYARRO)은 시롤리무스의 알부민 결합 제제로, 세포의 성장, 증식 및 혈관 신생을 조절하는 핵심 인자인 mTOR 경로를 선택적으로 억제합니다. 종양으로의 약물 전달 및 생체 이용률을 높임으로써, mTOR이 관여하는 질환에서 표적화된 항종양 활성을 나타내고 있으며, 이는 나노입자를 이용한 경로 특이적 치료법의 가능성을 뒷받침하고 있습니다.
  • 면역관문억제제 : 펨브롤리주맙(KEYTRUDA)은 PD-1 수용체를 표적으로 하는 인간화 단일클론 항체로, PD-1과 PD-L1/PD-L2 간의 상호작용을 억제함으로써 T세포를 매개로 한 항종양 면역 반응을 회복시킵니다. 이 약제는 여러 악성 종양에서 폭넓은 효능을 보여주고 있으며, 이는 면역 체크포인트 억제가 현대 암 면역요법의 기반으로서 역할을 하고 있음을 입증하고 있습니다.

자주 묻는 질문

  • 2025년 혈관 주위 상피 세포종(PEComa) 시장 규모는 어떻게 되나요?
  • 혈관 주위 상피 세포종(PEComa)의 연평균 성장률(CAGR)은 어떻게 되나요?
  • 혈관 주위 상피 세포종(PEComa) 치료에 사용되는 주요 약물은 무엇인가요?
  • PEComa의 발병률은 어떤 경향을 보이고 있나요?
  • PEComa의 진단 방법은 무엇인가요?
  • PEComa의 치료 관리에는 어떤 방법이 포함되나요?
  • PEComa의 주요 발생 국가는 어디인가요?

목차

제1장 주요 인사이트

제2장 서론

제3장 주요 요약

제4장 주요 이벤트

제5장 역학 및 예측 조사 방법

제6장 PEComa : 시장 개요

제7장 혈관 주위 상피 세포종(PEComa) 배경 및 개요

제8장 혈관 주위 상피 세포종(PEComa) 치료 가이드라인

제9장 혈관 주위 상피 세포종(PEComa) 역학 및 환자 인구

제10장 혈관 주위 상피 세포종(PEComa) 환자 경과

제11장 시판 치료제

제12장 신흥 치료제

제13장 혈관 주위 상피 세포종(PEComa) : 주요 7개국에 의한 해석

제14장 혈관 주위 상피 세포종(PEComa) 미충족 수요

제15장 혈관 주위 상피 세포종(PEComa) SWOT 분석

제16장 혈관 주위 상피 세포종(PEComa)에 관한 KOL(Key Opinion Leader)의 견해

제17장 혈관 주위 상피 세포종(PEComa) 시장 참여와 적용

제18장 부록

제19장 DelveInsight의 서비스 내용

제20장 면책사항

제21장 DelveInsight에 대해

KTH

Perivascular Epithelioid Cell Neoplasm (PEComa) Insights and Trends

  • According to DelveInsight's analysis, PEComa market size was found to be ~USD 43 million in the leading markets (the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan) in 2025.
  • A concentration of PEComas have been found in people of all ages, from children to older adults. Although they often occur in children with Tuberous Sclerosis Complex (TSC), they also occur sporadically in people with no known risk factors. Malignant PEComas are estimated to be diagnosed in 0.12-0.24 per 1 million people.
  • Therapeutic management of PEComa is focused on achieving durable disease control and preventing progression, primarily through a personalized approach guided by tumor resectability and molecular characteristics. Current treatment strategies include surgical resection for localized disease, along with systemic options such as mTOR inhibitors like Sirolimus and Everolimus, cytotoxic chemotherapy in select cases, and emerging therapies including immune checkpoint inhibitors such as KEYTRUDA for advanced or refractory disease.
  • Currently, there is only one approved therapy for the treatment of PEComa. The US FDA approved FYARRO (sirolimus protein-bound) in November 2021. Sirolimus, everolimus, and temsirolimus are currently listed in the National Comprehensive Cancer Network (NCCN) guidelines as systemic therapy agents with activity in PEComa.
  • KEYTRUDA is being explored as an immune checkpoint inhibitor for PEComa, particularly in advanced or refractory cases, with early reports suggesting potential antitumor activity in tumors expressing PD-L1 or harboring high mutational burden, though its use remains investigational and off-label; therapeutic management also includes surgical resection for localized disease, mTOR inhibitors (Sirolimus, Everolimus), and cytotoxic chemotherapy in select cases, reflecting a personalized, molecularly guided treatment approach.

Perivascular Epithelioid Cell Neoplasm (PEComa) Market Size and Forecast in the 7MM

  • 2025 PEComa Market Size: ~USD 45 million
  • 2036 Projected PEComa Market Size: USD 135 million
  • PEComa Growth Rate (2026-2036): 8.1% CAGR

DelveInsight's 'Perivascular Epithelioid Cell Neoplasm (PEComa) - Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of the Perivascular Epithelioid Cell Neoplasm (PEComa), historical and forecasted epidemiology, as well as the Perivascular Epithelioid Cell Neoplasm (PEComa) market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

The Perivascular Epithelioid Cell Neoplasm (PEComa) market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates, Perivascular Epithelioid Cell Neoplasm (PEComa) patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment, and growth rate projections (Historical & Forecast 2022-2036) across global regions. The report highlights key unmet medical needs in Perivascular Epithelioid Cell Neoplasm (PEComa) and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.

Key Factors Driving the Perivascular Epithelioid Cell Neoplasm (PEComa) Market

Rising PEComa Incidence

The increasing incidence of PEComa is a key driver of market expansion, with cases expected to grow at a significant CAGR through 2036.

Expanding Targeted and Combination Therapy Opportunities in PEComa

With FYARRO (nab sirolimus) as the only FDA approved systemic therapy, emerging options including mTOR inhibitors, VEGFR TKIs, and PD 1 inhibitors highlight growing opportunities for targeted and combination approaches in advanced PEComa.

Perivascular Epithelioid Cell Neoplasm (PEComa) Understanding and Treatment Algorithm

Perivascular Epithelioid Cell Neoplasm (PEComa) Overview and Diagnosis

Perivascular Epithelioid Cell Neoplasm is a rare mesenchymal tumor characterized by perivascular epithelioid cells with both melanocytic and smooth muscle features. It can occur in multiple organs (especially uterus, kidney, liver, retroperitoneum) and is sometimes linked to Tuberous Sclerosis Complex. Patients are often asymptomatic, but symptoms depend on tumor location and size.

Perivascular Epithelioid Cell Neoplasm (PEComa) Diagnosis

Diagnosis of Perivascular Epithelioid Cell Neoplasm is primarily based on histopathological examination and immunohistochemistry. Tumor cells show characteristic perivascular epithelioid morphology and co-express melanocytic markers (HMB-45, Melan-A) and smooth muscle markers (smooth muscle actin). Imaging techniques (CT or MRI) help in identifying tumor location and extent, but definitive diagnosis requires biopsy. Assessment of features such as tumor size, necrosis, mitotic activity, and invasion is important to determine malignant potential.

Perivascular Epithelioid Cell Neoplasm (PEComa) Treatment

Treatment of Perivascular Epithelioid Cell Neoplasm centers on surgical resection with negative margins for localized disease, which remains the primary curative approach, though recurrence can occur. For advanced, unresectable, or metastatic cases, FYARRO is the only FDA-approved systemic therapy, delivering sirolimus via a nanoparticle formulation with improved tumor penetration and showing durable clinical responses. Other mTOR inhibitors (sirolimus, everolimus, temsirolimus) are used as alternatives, particularly when FYARRO is not accessible. Chemotherapy and radiotherapy play mainly palliative roles with limited effectiveness, while targeted therapies (VEGF/VEGFR inhibitors) may be considered in select cases. Overall, management often requires specialized care and close follow-up due to variable tumor behavior.

Perivascular Epithelioid Cell Neoplasm (PEComa) Unmet Needs

The section "unmet needs of Perivascular Epithelioid Cell Neoplasm (PEComa)" outlines the critical gaps between the current state of patient care, diagnosis, and the ideal & effective management of the disease. It highlights the obstacles experienced by patients, clinicians, and researchers and identifies potential solutions for future progress.

1. Lack of effective and targeted treatment options

2. No standard treatment protocol

3. Need for accurate diagnostic techniques at an earlier stage of the disease

4. Unclear selection of combination strategies, and others.....

Comprehensive unmet needs insights in Perivascular Epithelioid Cell Neoplasm (PEComa) and their strategic implications are provided in the full report....

Perivascular Epithelioid Cell Neoplasm (PEComa) Epidemiology

Key Findings from Perivascular Epithelioid Cell Neoplasm (PEComa) Epidemiological Analysis and Forecast

  • According to DelveInsight's estimates, the total prevalent cases of PEComa in the 7MM were approximately 408 in 2025.
  • Among EU4 and the UK, Germany accounts for the highest incidence of PEComas, followed by France and the UK.
  • In 2025, Perivascular Epithelioid Cell Neoplasm (PEComa) by stage in the US, benign tumor is the most prevalent subtype with ~67% followed by local aggressive behavior (malignant PEComa) accounting for ~33%.
  • The observed female predominance and earlier onset of PEComa highlight the importance of sex-specific screening and early intervention strategies, particularly in recognizing susceptibility patterns and addressing disease at onset to prevent long-term complications.

Perivascular Epithelioid Cell Neoplasm (PEComa) Drug Chapters & Competitive Analysis

The PEComa drug chapter provides a detailed, market-focused review of approved therapies and the emerging pipeline across Phase I-III clinical trials. It covers the mechanism of action, clinical trial data, regulatory approvals, patents, collaborations, and strategic partnerships for each therapy, along with their advantages, limitations, and recent developments. This section offers critical insights into the PEComa treatment landscape, supporting market assessment, competitive analysis, and growth forecasting for the PEComa therapeutics market.

Approved Therapies for Perivascular Epithelioid Cell Neoplasm (PEComa)

Sirolimus protein-bound/ABI-009 (FYARRO): Kaken Pharmaceuticals

FYARRO developed by Kaken Pharmaceutical, is an intravenous nanoparticle albumin-bound mTOR inhibitor approved in 2021 for the treatment of adults with locally advanced, unresectable, or metastatic PEComa. By targeting the mTOR signaling pathway, often dysregulated in PEComa (commonly associated with TSC1/2 alterations), it helps inhibit tumor growth and proliferation. Administered on a 21-day cycle, FYARRO has shown durable clinical responses, addressing a critical unmet need and representing a significant advancement in targeted therapy for this rare tumor.

In May 2022, Aadi Bioscience reported it received notification of a product-specific, permanent J-code for FYARROTM (sirolimus protein-bound particles for injectable suspension) (albumin-bound) for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic malignant PEComa. Under the Healthcare Common Procedure Coding System (HCPCS), the J-code (J9331) became effective on July 1, 2022.

Perivascular Epithelioid Cell Neoplasm (PEComa) Pipeline Analysis

Pembrolizumab (KEYTRUDA): Merck Sharp & Dohme

KEYTRUDA developed by Merck Sharp & Dohme, is a humanized monoclonal IgG4 antibody that targets the programmed death-1 (PD-1) receptor, a key immune checkpoint involved in tumor immune evasion. By blocking PD-1 interaction with its ligands (PD-L1 and PD-L2), KEYTRUDA enhances T-cell-mediated immune responses against tumor cells. Although not specifically approved for Perivascular Epithelioid Cell Neoplasm, it has been explored in advanced or refractory cases, where it may help promote anti-tumor activity in select patients, particularly those with immunogenic tumor profiles.

Perivascular Epithelioid Cell Neoplasm (PEComa) Key Players, Market Leaders and Emerging Companies

  • Aadi Bioscience
  • Merck Sharp & Dohme, and others

Perivascular Epithelioid Cell Neoplasm (PEComa) Drug Updates

  • In March 2025, Aadi Bioscience has been reorganized into two entities. Following the divestiture of Aadi Sub to KAKEN, KAKEN assumes ownership of Aadi Sub, including the Aadi name, trademark, and the FYARRO(R) business. The remaining parent company has been renamed Whitehawk Therapeutics, focusing on its portfolio of ADC assets.
  • In December 2024, Aadi Bioscience entered an agreement to sell its commercial drug FYARRO to KAKEN Pharmaceutical for USD 100 million, alongside a USD 100 million PIPE equity financing and an exclusive license for a three-asset preclinical ADC portfolio in collaboration with WuXi Biologics and Hangzhou DAC. In March 2025, the company's stockholders approved the sale of FYARRO to KAKEN.

Drug Class Insights

Perivascular Epithelioid Cell Neoplasm (PEComa) Market Outlook

The Perivascular Epithelioid Cell Neoplasm market is undergoing a shift from reliance on surgery and limited systemic therapies toward a more targeted, mechanism-driven treatment approach. The approval of FYARRO represents a major milestone, validating mTOR pathway inhibition and improving outcomes in advanced or metastatic disease. With growing research into mTOR inhibitors, immune checkpoint inhibitors, and anti-angiogenic therapies, the treatment landscape is evolving similarly to other rare cancers, where early targeted approvals drive further innovation and pipeline expansion. These developments are expanding therapeutic options and encouraging precision-based treatment strategies.

The United States remains the dominant market, driven by higher disease awareness, better diagnostic capabilities, access to specialized sarcoma centers, and faster adoption of novel therapies. Overall, advancements in molecular diagnostics, targeted therapies, and rare cancer research are expected to support steady market growth and improved patient outcomes.

  • According to the estimates, the largest market size of PEComa was captured by the United States, i.e., ~USD 114 million by 2036.
  • The PEComa treatment landscape is evolving as management shifts from conventional approaches toward more molecularly targeted strategies, with mTOR inhibitors such as Sirolimus and Everolimus driving adoption due to their demonstrated efficacy in TSC1/TSC2-altered tumors, while emerging use of immunotherapies like Keytruda and other precision approaches is gaining interest in advanced or refractory cases.
  • Regional market dynamics for PEComa show the United States leading in revenue, while markets in Japan and European Union demonstrate steady growth, reflecting improved diagnostic awareness, greater access to targeted therapies and increasing adoption of emerging treatment approaches in rare tumor management.
  • In 2036, among all the therapies for PEComa, the highest revenue is estimated to be generated by FYARRO, in the United States.

Drug Class/Insights into Leading Emerging and Marketed Therapies in Perivascular Epithelioid Cell Neoplasm (PEComa) (2022-2036 Forecast)

The PEComa market comprises targeted mTOR inhibitors, anti-angiogenic therapies, and emerging immunotherapies, alongside surgical and supportive treatments, each addressing key molecular drivers such as mTOR pathway dysregulation and tumor angiogenesis underlying PEComa progression.

  • mTOR inhibitors: Sirolimus protein-bound (ABI-009, FYARRO) is an albumin-bound formulation of sirolimus that selectively inhibits the mTOR pathway, a central regulator of cell growth, proliferation, and angiogenesis. By enhancing tumor drug delivery and bioavailability, it demonstrates targeted antitumor activity in mTOR-driven diseases, supporting the potential of nanoparticle-based, pathway-specific therapies.
  • Immune checkpoint inhibitors: Pembrolizumab (KEYTRUDA), is a humanized monoclonal antibody that targets the PD-1 receptor, blocking its interaction with PD-L1/PD-L2 to restore T-cell-mediated antitumor immune responses. It has demonstrated broad efficacy across multiple malignancies, validating the role of immune checkpoint blockade as a cornerstone of modern cancer immunotherapy.

Perivascular Epithelioid Cell Neoplasm (PEComa) Drug Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during the forecast period (2026-2036). The analysis covers the PEComa market's uptake by drugs, patient uptake by therapy, and sales of each drug.

The uptake of FYARRO in advanced PEComa is expected to be gradual but meaningful, given its targeted mechanism as an mTOR inhibitor and its role in addressing a high unmet need in this rare tumor population. Adoption is likely to be supported by its demonstrated clinical benefit, increasing physician awareness, and the lack of effective alternative therapies for malignant PEComa. However, uptake may be moderated by its high annual treatment cost and the need for consistent reimbursement support, although favorable payer coverage and streamlined access pathways are expected to facilitate broader patient access over time.

Market Access and Reimbursement of PEComa

  • The US

By March 2022, most US payers covering the largest patient populations had reviewed and implemented formal FYARRO coverage policies, with the majority aligning prior authorization requirements with the prescribing information and imposing minimal restrictions. In addition, CMS approved a permanent J-code for FYARRO, effective July 1, 2022, streamlining reimbursement across all care settings. These developments, together with comprehensive patient support programs under Aadi Assist, have enhanced patient access since FDA approval. Support programs include a Temporary Supply Program providing up to 60 days of FYARRO for patients experiencing insurance delays, a Co-pay Support Program offering up to USD 25,000 annually for commercially insured patients, and a Patient Assistance Program (PAP) enabling uninsured or underinsured patients to access the drug at no cost, subject to eligibility verification by a case manager.

Reimbursement is a crucial factor that affects the drug's access to the market. 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, many payment models are being considered by payers and other industry insiders.

Further details are provided in the final report....

Perivascular Epithelioid Cell Neoplasm (PEComa) Therapies Price Scenario & Trends

Pricing and analogue assessment of PEComa therapies highlights evolving price dynamics structures. This section summarizes the cost of approved treatments, closest and most appropriate analogue selection for emerging therapies, and understanding of how pricing influences market access, adherence, and long-term uptake.

  • Pricing of Perivascular Epithelioid Cell Neoplasm (PEComa) Approved Drugs

FYARRO is administered in 21-day treatment cycles, with an approximate cost of USD 39,000 per cycle. Assuming a total of 17 cycles over a 12-month period, the estimated annual treatment cost reaches about USD 501,024. This high annual cost highlights the significant financial burden associated with therapy and underscores the importance of robust reimbursement mechanisms to ensure patient access.

Further details are provided in the final report....

Industry Experts and Physician Views for Perivascular Epithelioid Cell Neoplasm (PEComa)

To keep up with PEComa market trends, we take Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on the PEComa emerging therapies, evolving treatment landscape, patient adherence to conventional therapies, therapy switching trends, drug adoption and uptake, accessibility challenges, and epidemiology and real-world prescription patterns in PEComa, including MD, PhD, Instructor, Postdoctoral Researcher, Professor, Researcher, and others.

DelveInsight's analysts connected with 8+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as the Memorial Sloan Kettering Cancer Center, Strasbourg-Europe Cancer Institute (ICANS), and the University of Nottingham, etc. were contacted. Their opinion helps understand and validate current and emerging PEComa therapies, highlight unmet medical needs, provide epidemiological context, and support strategic decisions for market access, therapy adoption, and pipeline prioritization in PEComa.

Qualitative Analysis: SWOT and Conjoint Analysis

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

In the SWOT analysis of Perivascular Epithelioid Cell Neoplasm (PEComa), 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.

Conjoint analysis analyzes emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

The team of analysts analyzes promising 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, whereas the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are majorly observed. In addition, the scoring is also based on the route of administration, order of entry, 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.

Scope of the Report:

  • The report covers a segment of key events, an executive summary, a descriptive overview of PEComa, explaining its causes, signs and symptoms, pathogenesis, and currently available treatments.
  • Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of the diagnosis rate, and disease progression along treatment guidelines.
  • Additionally, an all-inclusive account of both the current and emerging treatments, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
  • A detailed review of the PEComa market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
  • The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM PEComa market.

Report Insights

  • Perivascular Epithelioid Cell Neoplasm (PEComa) Patient Population Forecast
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Therapeutics Market Size
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Pipeline Analysis
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Market Size and Trends
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Market Opportunity (Current and forecasted)

Report Key Strengths

  • Epidemiology-based (Epi-based) Bottom-up Forecasting
  • Artificial Intelligence (AI)-enabled Market Research Report
  • 11-year forecast
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Market Outlook (North America, Europe, Asia-Pacific)
  • Patient Burden Trends (by geography)
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Treatment Addressable Market (TAM)
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Competitive Landscape
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Major Companies Insights
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Price Trends and Analogue Assessment
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Therapies Drug Adoption/Uptake
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Therapies Peak Patient Share analysis

Report Assessment

  • Perivascular Epithelioid Cell Neoplasm (PEComa) Current Treatment Practices
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Unmet Needs
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Clinical Development Analysis
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Emerging Drugs Product Profiles
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Market Attractiveness
  • Perivascular Epithelioid Cell Neoplasm (PEComa) Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs:

Market Insights

  • What was the Perivascular Epithelioid Cell Neoplasm (PEComa) market size, the market size by therapies, market share (%) distribution in 2025, and what would it look like by 2036? What are the contributing factors for this growth?
  • What are the anticipated pricing variations among different geographies for the emerging therapies in the future?
  • What can be the future treatment paradigm of Perivascular Epithelioid Cell Neoplasm (PEComa)?
  • What are the disease risks, burdens, and unmet needs of Perivascular Epithelioid Cell Neoplasm (PEComa)? What will be the growth opportunities across the 7MM concerning the patient population with Perivascular Epithelioid Cell Neoplasm (PEComa)?
  • Who is the major future competitor in the market, and how will the competitors affect their market share?
  • What are the current options for the treatment of Perivascular Epithelioid Cell Neoplasm (PEComa)? What are the current guidelines for treating Perivascular Epithelioid Cell Neoplasm (PEComa) in the US, Europe, and Japan?

Reasons to Buy:

  • The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Perivascular Epithelioid Cell Neoplasm (PEComa) market.
  • Bottom up forecasting builds from the affected population to product forecasts, delivering a robust, data driven approach ideal for new therapies and novel classes.
  • Insights on patient burden/disease incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
  • Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
  • Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
  • Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
  • To understand KOLs' perspectives on the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
  • Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
  • This Artificial Intelligence (AI) enabled report summarize and simplify complex datasets within the report into clear, actionable insights for stakeholders, investors, and healthcare providers, enabling faster, data driven decisions.

Table of Contents

1. Key Insights

2. Report Introduction

3. Executive Summary

4. Key Events

  • 4.1. Upcoming Key Catalysts
  • 4.2. Key Transactions And Collaborations
  • 4.3. News Flow

5. Epidemiology and Market Forecast Methodology

6. PEComa Market Overview at a Glance

  • 6.1. Emerging Landscape Analysis (By Phase, Molecule Type, and RoA)
  • 6.2. Market Share (%) Distribution of PEComa By Therapies in the 7MM, in 2025
  • 6.3. Market Share (%) Distribution of PEComa By Therapies in the 7MM, in 2036

7. Disease Background and Overview of Perivascular Epithelioid Cell Neoplasm (PEComa)

  • 7.1. Introduction
  • 7.2. Types
  • 7.3. Symptoms
  • 7.4. Causes
  • 7.5. Pathophysiology
  • 7.6. Diagnosis
  • 7.7. Treatment

8. Treatment Guidelines of Perivascular Epithelioid Cell Neoplasm (PEComa)

  • 8.1. American College of Rheumatology (2021)
  • 8.2. EULAR/Pres Recommendations for the Diagnosis and Management of Still's Disease
  • 8.3. Japan College of Rheumatology Clinical Practice Guidelines (2024)

9. Epidemiology and Patient Population of Perivascular Epithelioid Cell Neoplasm (PEComa)

  • 9.1. Key Findings
  • 9.2. Assumptions and Rationale
  • 9.3. Diagnosed Incident Cases of PEComa in the 7MM
  • 9.4. The United States
    • 9.4.1. Total Incident Cases of PEComa in the United States
    • 9.4.2. Stage-specific Incident Cases of PEComa in the United States
    • 9.4.3. Gender-specific Incident Cases of PEComa in the United States
    • 9.4.4. PEComa Cases by Clinical Ptresentation in the United States
    • 9.4.5. Age-specific Incident Cases of PEComa in United States
  • 9.5. EU4 and the UK
    • 9.5.1. Total Incident Cases of PEComa in the EU4 and the UK
    • 9.5.2. Stage-specific Incident Cases of PEComa in the EU4 and the UK
    • 9.5.3. Gender-specific Incident Cases of PEComa in the EU4 and the UK
    • 9.5.4. PEComa Cases by Clinical Ptresentation in the EU4 and the UK
    • 9.5.5. Age-specific Incident Cases of PEComa in EU4 and the UK
  • 9.6. Japan
    • 9.6.1. Total Incident Cases of PEComa in the Japan
    • 9.6.2. Stage-specific Incident Cases of PEComa in the Japan
    • 9.6.3. Gender-specific Incident Cases of PEComa in the Japan
    • 9.6.4. PEComa Cases by Clinical Ptresentation in the Japan
    • 9.6.5. Age-specific Incident Cases of PEComa in Japan

10. Patient Journey of Perivascular Epithelioid Cell Neoplasm (PEComa)

11. Marketed Therapies

  • 11.1. Marketed Competitive Landscape of Perivascular Epithelioid Cell Neoplasm (PEComa)
  • 11.2. Sirolimus protein-bound/ABI-009 (FYARRO): Kaken
    • 11.2.1. Drug Description
    • 11.2.2. Regulatory Milestones
    • 11.2.3. Other Developmental Activities
    • 11.2.4. Summary of Pivotal Trials
    • 11.2.5. Clinical Development
      • 11.2.5.1. Clinical Trial Information
    • 11.2.6. Analyst Views

12. Emerging Drugs

  • 12.1. Emerging Competitive Landscape of Perivascular Epithelioid Cell Neoplasm (PEComa)
  • 12.2. Pembrolizumab (KEYTRUDA): Merck Sharp & Dohme
    • 12.2.1. Drug Description
    • 12.2.2. Other Developmental Activities
    • 12.2.3. Clinical Development
      • 12.2.3.1. Clinical Trials Information
    • 12.2.4. Analyst Views

13. Perivascular Epithelioid Cell Neoplasm (PEComa): 7MM Analysis

  • 13.1. Key Findings
  • 13.2. Market Outlook of Perivascular Epithelioid Cell Neoplasm (PEComa)
  • 13.3. Key Market Forecast Assumptions
    • 13.3.1. Cost Assumptions
  • 13.4. Conjoint Analysis of Perivascular Epithelioid Cell Neoplasm (PEComa)
  • 13.5. Total Market Size of PEComa in the 7MM
  • 13.6. Total Market Size of PEComa by Therapies in the 7MM
  • 13.7. The United States
    • 13.7.1. Total Market Size of PEComa in the United States
    • 13.7.2. Total Market Size of PEComa by Therapies in the United States
  • 13.8. EU4 and the UK
    • 13.8.1. Total Market Size of PEComa in EU4 and the UK
    • 13.8.2. Total Market Size of PEComa by Therapies in EU4 and the UK
  • 13.9. Japan
    • 13.9.1. Total Market Size of PEComa in Japan
    • 13.9.2. Total Market Size of PEComa by Therapies in Japan

14. Unmet Needs of Perivascular Epithelioid Cell Neoplasm (PEComa)

15. SWOT Analysis of Perivascular Epithelioid Cell Neoplasm (PEComa)

16. KOL Views of Perivascular Epithelioid Cell Neoplasm (PEComa)

17. Market Access and Reimbursement of Perivascular Epithelioid Cell Neoplasm (PEComa)

  • 17.1. The United States
  • 17.2. EU4 and the UK
    • 17.2.1. Germany
    • 17.2.2. France
    • 17.2.3. Italy
    • 17.2.4. Spain
    • 17.2.5. United Kingdom
  • 17.3. Japan
  • 17.4. Summary and comparison of Market Access and Pricing Policy Developments in 2025
  • 17.5. Market Access and Reimbursement of PEComa Therapies

18. Appendix

  • 18.1. Bibliography
  • 18.2. Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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