세계의 폼페병 치료제 시장 : 산업 규모, 점유율, 동향, 기회, 예측, 치료 유형별, 투여 경로별, 최종사용자별, 지역별, 경쟁별(2020-2030년)
Pompe Disease Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, & Forecast, Segmented By Treatment Type, By Route of Administration, By End User, By Region, & Competition, 2020-2030F
상품코드:1698062
리서치사:TechSci Research
발행일:2025년 03월
페이지 정보:영문 180 Pages
라이선스 & 가격 (부가세 별도)
ㅁ Add-on 가능: 고객의 요청에 따라 일정한 범위 내에서 Customization이 가능합니다. 자세한 사항은 문의해 주시기 바랍니다.
한글목차
폼페병 치료제 세계 시장 규모는 2024년 15억 달러에 달했고, 2030년까지 예측 기간 동안 연평균 4.40%의 꾸준한 성장이 예상됩니다.
세계 폼페병 치료제 시장은 희귀질환 및 희귀질환 치료제 산업에서 빠르게 발전하고 있는 분야로, 과학의 발전과 혁신적인 치료법에 대한 투자 증가에 힘입어 빠르게 성장하고 있습니다. 폼페병(글리코겐 저장병 II형)은 산성 알파-글루코시다아제(GAA)의 결핍으로 인해 근육 조직에 글리코겐이 축적되는 희귀한 라이소좀 질환입니다. 그 결과, 진행성 근력 저하와 심각한 호흡기 합병증을 유발하여 표적화된 치료 개입이 필요합니다.
시장 개요
예측 기간
2026-2030년
시장 규모 : 2024년
15억 달러
시장 규모 : 2030년
19억 5,000만 달러
CAGR : 2025-2030년
4.40%
급성장 부문
효소대체요법(ERT)
최대 시장
북미
시장은 주로 글리코겐 청소율과 임상 결과를 개선하는 Myozyme(alglucosidase alfa), Lumizyme, 최근 Nexviazyme(avalglucosidase alfa)과 같은 효소대체요법(ERT)에 의해 형성되고 있습니다. 또한, 2023년 폼비리치(시파글루코시다아제 알파)와 옵디보(미글루스타트)가 승인되면서 특히 기존 ERT에 반응이 불충분한 야간발작형 폼페병(LOPD) 환자들의 치료 옵션이 넓어졌습니다.
이러한 발전에도 불구하고, 높은 치료 비용과 제한된 신생아 선별검사 프로그램으로 인해 진단 지연과 질병 진행이 시장 침투의 제약요인으로 작용하고 있습니다. 그러나 유전자 치료와 차세대 치료제가 획기적인 치료법으로 부상하고 있으며, 현재 진행 중인 임상시험은 보다 내구성 있고 비용 효율적인 치료법을 제공하는 것을 목표로 하고 있습니다.
시장 성장 촉진요인
질병에 대한 인식 및 진단 향상
주요 시장 이슈
제한된 질병 인식 및 진단 지연
주요 시장 동향
개인맞춤의료와 정밀치료
목차
제1장 개요
제2장 조사 방법
제3장 주요 요약
제4장 고객의 소리
제5장 폼페병 치료제 시장 전망
시장 규모와 예측
금액별
시장 점유율과 예측
치료 유형별(약물 요법, 효소대체요법(ERT), 물리치료, 샤프롱 첨단 대체 요법)
투여 경로(경구, 비경구)
최종사용자별(병원, 전문 클리닉, 기타)
지역별
기업별(2024년)
시장 맵
제6장 북미의 폼페병 치료제 시장 전망
시장 규모와 예측
시장 점유율과 예측
북미 : 국가별 분석
미국
캐나다
멕시코
제7장 유럽의 폼페병 치료제 시장 전망
시장 규모와 예측
시장 점유율과 예측
유럽 : 국가별 분석
독일
영국
이탈리아
프랑스
스페인
제8장 아시아태평양의 폼페병 치료제 시장 전망
시장 규모와 예측
시장 점유율과 예측
아시아태평양 : 국가별 분석
중국
인도
일본
한국
호주
제9장 남미의 폼페병 치료제 시장 전망
시장 규모와 예측
시장 점유율과 예측
남미 : 국가별 분석
브라질
아르헨티나
콜롬비아
제10장 중동 및 아프리카의 폼페병 치료제 시장 전망
시장 규모와 예측
시장 점유율과 예측
중동 및 아프리카 : 국가별 분석
남아프리카공화국
사우디아라비아
아랍에미리트
제11장 시장 역학
성장 촉진요인
과제
제12장 시장 동향과 발전
최근 동향
제품 출시
인수합병(M&A)
제13장 세계의 폼페병 치료제 시장 : SWOT 분석
제14장 경쟁 구도
Genzyme Corp
Amicus Therapeutics Inc
Valerion Therapeutics LLC
Audentes Therapeutics Inc
Actus Therapeutics Inc
BioMarin Pharmaceutical Inc
EpiVax inc
Oxyrane Co Ltd.
Sangamo BioSciences Inc
Avrobio Inc
Spark Therapeutics Inc.
제15장 전략적 제안
제16장 리서치사에 대해 & 면책사항
LSH
영문 목차
영문목차
Global Pompe Disease Therapeutics Market was valued at USD 1.50 Billion in 2024 and is anticipated to project steady growth in the forecast period with a CAGR of 4.40% through 2030. The Global Pompe Disease Therapeutics Market is a rapidly evolving segment within the rare disease and orphan drug industry, driven by scientific advancements and increasing investment in innovative therapies. Pompe disease, or glycogen storage disease type II, is a rare lysosomal disorder caused by the deficiency of acid alpha-glucosidase (GAA), leading to glycogen accumulation in muscle tissues. This results in progressive muscle weakness and severe respiratory complications, necessitating targeted therapeutic interventions.
Market Overview
Forecast Period
2026-2030
Market Size 2024
USD 1.50 Billion
Market Size 2030
USD 1.95 Billion
CAGR 2025-2030
4.40%
Fastest Growing Segment
Enzyme Replacement Therapy (ERT)
Largest Market
North America
The market is primarily shaped by enzyme replacement therapies (ERTs) such as Myozyme (alglucosidase alfa), Lumizyme, and the more recent Nexviazyme (avalglucosidase alfa), which offer improved glycogen clearance and clinical outcomes. Additionally, the approval of Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat) in 2023 has expanded treatment options for patients, particularly those with late-onset Pompe disease (LOPD) who exhibit suboptimal responses to conventional ERTs.
Despite these advancements, market penetration is constrained by high treatment costs and limited newborn screening programs, leading to delayed diagnoses and disease progression. However, gene therapy and next-generation therapeutics are emerging as potential breakthroughs, with ongoing clinical trials aiming to provide more durable and cost-effective treatment alternatives.
Key Market Drivers
Increased Disease Awareness and Diagnosis
Pompe disease, often considered a rare or orphan disease, historically suffered from a lack of recognition and understanding. Pompe disease is a complex, multisystem genetic disorder with a prevalence of approximately 1 in 40,000 individuals in the United States. Increasing awareness of this rare condition is crucial, as early diagnosis and timely intervention can significantly improve patient outcomes and quality of life. Efforts to enhance disease recognition are being driven by medical institutions, patient advocacy groups, and biotech companies focused on expanding access to diagnostics and treatment. Newborn screening programs, physician education initiatives, and targeted awareness campaigns are playing a pivotal role in identifying patients earlier and facilitating access to advanced therapies. Patients faced delayed or misdiagnoses, leading to prolonged suffering and a dearth of effective treatments. Advocacy groups dedicated to Pompe disease have played a pivotal role in driving disease awareness. These organizations tirelessly work to raise public awareness, educate healthcare professionals, and provide support to affected individuals and their families. By leveraging social media, organizing awareness campaigns, and collaborating with healthcare institutions, these groups have succeeded in putting Pompe disease on the radar. The evolution of diagnostic technologies is another catalyst for increased disease awareness. Cutting-edge tools such as genetic testing and biomarker identification have revolutionized the diagnostic process. Physicians can now identify Pompe disease more accurately and swiftly, enabling timely intervention and treatment initiation. This, in turn, has elevated the importance of early diagnosis in improving patient outcomes.
Key Market Challenges
Limited Disease Awareness and Late Diagnosis
One of the foremost challenges in the Pompe Disease Therapeutics Market is the limited awareness of the disease itself, leading to delayed or missed diagnoses. Pompe disease is considered a rare condition, making it less well-known among healthcare professionals and the general public. This lack of awareness often results in late-stage diagnoses, which can significantly impact treatment outcomes.
Pompe disease's rarity, with an estimated incidence of 1 in 40,000 to 1 in 300,000 births, contributes to its obscurity. As healthcare professionals encounter rare diseases less frequently, the likelihood of prompt recognition and diagnosis diminishes. Pompe disease presents with symptoms that can overlap with those of other more common conditions, such as muscle weakness and respiratory problems. This lack of distinctiveness can lead to misdiagnoses or a failure to consider Pompe disease as a potential cause.
Key Market Trends
Personalized Medicine and Precision Therapies
One of the most significant trends in the Pompe Disease Therapeutics Market is the shift towards personalized medicine and precision therapies. Traditional treatment approaches often took a one-size-fits-all approach, but advancements in genetics and diagnostics have paved the way for tailored treatments that address the specific genetic mutations of individual patients. Advances in genetic research have provided insights into the precise genetic mutations that cause Pompe disease. This knowledge allows for the development of therapies that target the specific genetic defects in individual patients, maximizing treatment efficacy. Pharmaceutical companies are investing in innovative approaches, such as gene therapy and RNA-based therapies, that can be customized to target specific genetic mutations. These therapies hold the potential to correct the underlying genetic causes of Pompe disease. Advanced diagnostic tools, including genetic testing and biomarker identification, enable healthcare providers to identify the specific genetic mutations in Pompe disease patients. This information guides treatment decisions, ensuring that therapies are tailored to each patient's unique genetic profile.
Key Market Players
Genzyme Corp
Amicus Therapeutics Inc
Valerion Therapeutics LLC
Audentes Therapeutics Inc
Actus Therapeutics Inc
BioMarin Pharmaceutical Inc
EpiVax inc
Oxyrane Co Ltd.
Sangamo BioSciences Inc
Avrobio Inc
Spark Therapeutics Inc
Report Scope:
In this report, the Global Pompe Disease Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:
Pompe Disease Therapeutics Market, By Treatment Type:
Drugs
Enzyme Replacement Therapy
Physical Therapy
Chaperone-Advanced Replacement Therapy
Pompe Disease Therapeutics Market, By Route of Administration:
Oral
Parenteral
Pompe Disease Therapeutics Market, By End User:
Hospitals & Speciality Clinics
Other
Pompe Disease Therapeutics Market, By Region:
North America
United States
Canada
Mexico
Europe
France
United Kingdom
Italy
Germany
Spain
Asia-Pacific
China
India
Japan
Australia
South Korea
South America
Brazil
Argentina
Colombia
Middle East & Africa
South Africa
Saudi Arabia
UAE
Competitive Landscape
Company Profiles: Detailed analysis of the major companies present in the Global Pompe Disease Therapeutics Market.
Available Customizations:
Global Pompe Disease Therapeutics market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:
Company Information
Detailed analysis and profiling of additional market players (up to five).
Table of Contents
1. Product Overview
1.1. Market Definition
1.2. Scope of the Market
1.2.1. Markets Covered
1.2.2. Years Considered for Study
1.2.3. Key Market Segmentations
2. Research Methodology
2.1. Objective of the Study
2.2. Baseline Methodology
2.3. Key Industry Partners
2.4. Major Association and Secondary Sources
2.5. Forecasting Methodology
2.6. Data Triangulation & Validation
2.7. Assumptions and Limitations
3. Executive Summary
3.1. Overview of the Market
3.2. Overview of Key Market Segmentations
3.3. Overview of Key Market Players
3.4. Overview of Key Regions/Countries
3.5. Overview of Market Drivers, Challenges, Trends
4. Voice of Customer
5. Pompe Disease Therapeutics Market Outlook
5.1. Market Size & Forecast
5.1.1. By Value
5.2. Market Share & Forecast
5.2.1. By Treatment Type (Drugs, Enzyme Replacement Therapy, Physical Therapy, Chaperone-Advanced Replacement Therapy)
5.2.2. By Route of Administration (Oral, Parenteral)
5.2.3. By End User (Hospitals & Speciality Clinics, Other)
5.2.4. By Region
5.2.5. By Company (2024)
5.3. Market Map
6. North America Pompe Disease Therapeutics Market Outlook
6.1. Market Size & Forecast
6.1.1. By Value
6.2. Market Share & Forecast
6.2.1. By Treatment Type
6.2.2. By Route of Administration
6.2.3. By End User
6.2.4. By Country
6.3. North America: Country Analysis
6.3.1. United States Pompe Disease Therapeutics Market Outlook
6.3.1.1. Market Size & Forecast
6.3.1.1.1. By Value
6.3.1.2. Market Share & Forecast
6.3.1.2.1. By Treatment Type
6.3.1.2.2. By Route of Administration
6.3.1.2.3. By End User
6.3.2. Canada Pompe Disease Therapeutics Market Outlook
6.3.2.1. Market Size & Forecast
6.3.2.1.1. By Value
6.3.2.2. Market Share & Forecast
6.3.2.2.1. By Treatment Type
6.3.2.2.2. By Route of Administration
6.3.2.2.3. By End User
6.3.3. Mexico Pompe Disease Therapeutics Market Outlook
6.3.3.1. Market Size & Forecast
6.3.3.1.1. By Value
6.3.3.2. Market Share & Forecast
6.3.3.2.1. By Treatment Type
6.3.3.2.2. By Route of Administration
6.3.3.2.3. By End User
7. Europe Pompe Disease Therapeutics Market Outlook
7.1. Market Size & Forecast
7.1.1. By Value
7.2. Market Share & Forecast
7.2.1. By Treatment Type
7.2.2. By Route of Administration
7.2.3. By End User
7.2.4. By Country
7.3. Europe: Country Analysis
7.3.1. Germany Pompe Disease Therapeutics Market Outlook
7.3.1.1. Market Size & Forecast
7.3.1.1.1. By Value
7.3.1.2. Market Share & Forecast
7.3.1.2.1. By Treatment Type
7.3.1.2.2. By Route of Administration
7.3.1.2.3. By End User
7.3.2. United Kingdom Pompe Disease Therapeutics Market Outlook
7.3.2.1. Market Size & Forecast
7.3.2.1.1. By Value
7.3.2.2. Market Share & Forecast
7.3.2.2.1. By Treatment Type
7.3.2.2.2. By Route of Administration
7.3.2.2.3. By End User
7.3.3. Italy Pompe Disease Therapeutics Market Outlook
7.3.3.1. Market Size & Forecast
7.3.3.1.1. By Value
7.3.3.2. Market Share & Forecast
7.3.3.2.1. By Treatment Type
7.3.3.2.2. By Route of Administration
7.3.3.2.3. By End User
7.3.4. France Pompe Disease Therapeutics Market Outlook
7.3.4.1. Market Size & Forecast
7.3.4.1.1. By Value
7.3.4.2. Market Share & Forecast
7.3.4.2.1. By Treatment Type
7.3.4.2.2. By Route of Administration
7.3.4.2.3. By End User
7.3.5. Spain Pompe Disease Therapeutics Market Outlook
7.3.5.1. Market Size & Forecast
7.3.5.1.1. By Value
7.3.5.2. Market Share & Forecast
7.3.5.2.1. By Treatment Type
7.3.5.2.2. By Route of Administration
7.3.5.2.3. By End User
8. Asia-Pacific Pompe Disease Therapeutics Market Outlook
8.1. Market Size & Forecast
8.1.1. By Value
8.2. Market Share & Forecast
8.2.1. By Treatment Type
8.2.2. By Route of Administration
8.2.3. By End User
8.2.4. By Country
8.3. Asia-Pacific: Country Analysis
8.3.1. China Pompe Disease Therapeutics Market Outlook
8.3.1.1. Market Size & Forecast
8.3.1.1.1. By Value
8.3.1.2. Market Share & Forecast
8.3.1.2.1. By Treatment Type
8.3.1.2.2. By Route of Administration
8.3.1.2.3. By End User
8.3.2. India Pompe Disease Therapeutics Market Outlook
8.3.2.1. Market Size & Forecast
8.3.2.1.1. By Value
8.3.2.2. Market Share & Forecast
8.3.2.2.1. By Treatment Type
8.3.2.2.2. By Route of Administration
8.3.2.2.3. By End User
8.3.3. Japan Pompe Disease Therapeutics Market Outlook
8.3.3.1. Market Size & Forecast
8.3.3.1.1. By Value
8.3.3.2. Market Share & Forecast
8.3.3.2.1. By Treatment Type
8.3.3.2.2. By Route of Administration
8.3.3.2.3. By End User
8.3.4. South Korea Pompe Disease Therapeutics Market Outlook
8.3.4.1. Market Size & Forecast
8.3.4.1.1. By Value
8.3.4.2. Market Share & Forecast
8.3.4.2.1. By Treatment Type
8.3.4.2.2. By Route of Administration
8.3.4.2.3. By End User
8.3.5. Australia Pompe Disease Therapeutics Market Outlook
8.3.5.1. Market Size & Forecast
8.3.5.1.1. By Value
8.3.5.2. Market Share & Forecast
8.3.5.2.1. By Treatment Type
8.3.5.2.2. By Route of Administration
8.3.5.2.3. By End User
9. South America Pompe Disease Therapeutics Market Outlook
9.1. Market Size & Forecast
9.1.1. By Value
9.2. Market Share & Forecast
9.2.1. By Treatment Type
9.2.2. By Route of Administration
9.2.3. By End User
9.2.4. By Country
9.3. South America: Country Analysis
9.3.1. Brazil Pompe Disease Therapeutics Market Outlook
9.3.1.1. Market Size & Forecast
9.3.1.1.1. By Value
9.3.1.2. Market Share & Forecast
9.3.1.2.1. By Treatment Type
9.3.1.2.2. By Route of Administration
9.3.1.2.3. By End User
9.3.2. Argentina Pompe Disease Therapeutics Market Outlook
9.3.2.1. Market Size & Forecast
9.3.2.1.1. By Value
9.3.2.2. Market Share & Forecast
9.3.2.2.1. By Treatment Type
9.3.2.2.2. By Route of Administration
9.3.2.2.3. By End User
9.3.3. Colombia Pompe Disease Therapeutics Market Outlook
9.3.3.1. Market Size & Forecast
9.3.3.1.1. By Value
9.3.3.2. Market Share & Forecast
9.3.3.2.1. By Treatment Type
9.3.3.2.2. By Route of Administration
9.3.3.2.3. By End User
10. Middle East and Africa Pompe Disease Therapeutics Market Outlook
10.1. Market Size & Forecast
10.1.1. By Value
10.2. Market Share & Forecast
10.2.1. By Treatment Type
10.2.2. By Route of Administration
10.2.3. By End User
10.2.4. By Country
10.3. MEA: Country Analysis
10.3.1. South Africa Pompe Disease Therapeutics Market Outlook
10.3.1.1. Market Size & Forecast
10.3.1.1.1. By Value
10.3.1.2. Market Share & Forecast
10.3.1.2.1. By Treatment Type
10.3.1.2.2. By Route of Administration
10.3.1.2.3. By End User
10.3.2. Saudi Arabia Pompe Disease Therapeutics Market Outlook
10.3.2.1. Market Size & Forecast
10.3.2.1.1. By Value
10.3.2.2. Market Share & Forecast
10.3.2.2.1. By Treatment Type
10.3.2.2.2. By Route of Administration
10.3.2.2.3. By End User
10.3.3. UAE Pompe Disease Therapeutics Market Outlook
10.3.3.1. Market Size & Forecast
10.3.3.1.1. By Value
10.3.3.2. Market Share & Forecast
10.3.3.2.1. By Treatment Type
10.3.3.2.2. By Route of Administration
10.3.3.2.3. By End User
11. Market Dynamics
11.1. Drivers
11.2. Challenges
12. Market Trends & Developments
12.1. Recent Developments
12.2. Product Launches
12.3. Mergers & Acquisitions
13. Global Pompe Disease Therapeutics Market: SWOT Analysis
