囊腫纖維化市場－全球及區域分析：按國家/地區分類－分析與預測（2025-2035）

Cystic Fibrosis Market - A Global and Regional Analysis: Focus on Country and Region - Analysis and Forecast, 2025-2035

出版日期: 2025年11月10日 | 出版商:

BIS Research | 英文 90 Pages | 商品交期: 1-5個工作天內

價格

簡介目錄圖表

全球囊腫纖維化市場分析與預測（2025-2035）

囊腫纖維化是一種遺傳性疾病，主要影響肺部和消化器官系統，其特徵是產生濃稠黏液。

這種黏液會阻塞呼吸道，導致呼吸道感染疾病、消化器官系統問題以及其他各種健康問題。它是兒童和青少年中最常見的危及生命的遺傳性疾病之一。囊腫纖維化是由CFTR基因（囊腫纖維化跨膜傳導調節因子）突變引起的，該基因調節細胞內外鹽和水的流動。

囊腫纖維化市場的發展主要受患者數量增加、標靶治療進步以及精準醫療日益普及的推動。囊腫纖維化是一種遺傳性疾病，在西方國家，尤其是美國和歐洲等國家，發生率較高。根據囊腫纖維化基金會統計，光在美國就有超過3萬名患者。確診患者數量的持續成長是推動囊腫纖維化藥物需求成長的主要因素，尤其是在人口老化和新治療方法不斷湧現的背景下。

此外，CFTR調變器療法的引入極大地改變了囊腫纖維化的治療模式。這些藥物透過針對囊腫纖維化的根本病因—CFTR基因，已被證實能夠顯著改善患者的生活品質和肺功能，從而延長平均壽命並減少住院次數，進一步推動了市場成長。

精準醫療在囊腫纖維化市場的重要性日益凸顯。基因檢測和個人化治療正變得越來越普及，尤其是在識別導致患者囊腫纖維化的基因突變方面。這一趨勢可能會推動針對特定突變的藥物（例如，針對不同基因型設計的CFTR調變器）的需求。這種關注點使得高度個人化的治療方案成為可能，從而有望提高治療效果。此外，醫護人員和患者對囊腫纖維化的認知不斷提高，以及新生兒篩檢計畫的進展，使得早期診斷和治療成為可能。早期治療可以延緩疾病進展、減輕症狀並改善患者預後。反過來，這又推動了患者群體的成長和對囊腫纖維化治療需求的增加。

雖然囊腫纖維化新藥的問世為患者帶來了希望，但其高昂的價格仍然令人擔憂。這些費用嚴重阻礙了患者獲得治療，尤其是在中低收入國家以及無保險或保險不足的人群中。囊腫纖維化藥物的高昂價格給患者和醫療保健系統都帶來了巨大的挑戰。

囊腫纖維化患者可能需要複雜的合併用藥聯合治療，包括肺功能治療、酵素替代療法和CFTR調變器治療。堅持這些治療方案可能充滿挑戰，導致患者不遵醫囑，最終治療失敗。維持規律的治療方案會帶給患者沉重的生理、心理和經濟負擔。雖然CFTR調變器已顯著改善了許多患者的病情，但並非對所有囊腫纖維化突變都有效。這意味著，對於某些患者群體，特別是那些攜帶罕見突變且對現有CFTR調變器無反應的患者而言，治療選擇仍然有限。針對這些罕見突變的藥物研發仍是一項挑戰。

基因療法具有直接糾正致病基因突變的潛力，已成為囊腫纖維化治療領域的突破性進展。基於CRISPR技術的治療方法和其他基因編輯技術正在研發中，旨在提供長期甚至永久性的解決方案。此外，囊腫纖維化孤兒藥市場也不斷擴大，FDA和EMA等監管機構正在加速核准並授予針對囊腫纖維化的藥物市場獨佔權。由於囊腫纖維化被歸類為罕見疾病，製藥公司可以享受延長專利保護期和稅額扣抵等經濟誘因。這鼓勵了更多公司投資於囊腫纖維化藥物的研發，從而催生出創新治療方法。

雖然囊腫纖維化在西方國家更為常見，但新興市場的全球認知度和診斷能力正在不斷提高。隨著亞太和拉丁美洲等地區醫療基礎設施的不斷完善，這些地區為囊腫纖維化藥物生產商帶來了巨大的成長機會。擴大基因檢測和CFTR調變器療法的普及範圍，有望釋放一個巨大的潛在市場。

此外，許多公司正在研發藥物，以應對囊腫纖維化治療領域尚未解決的難題，例如用於治療呼吸道感染疾病的新型抗生素和針對罕見囊腫纖維化突變的藥物。這些創新解決方案為從小型生物技術公司到大型製藥公司等各類企業提供了新的成長機會。對生物製藥和標靶治療的投資具有很高的獲利潛力。

囊腫纖維化市場的競爭格局是由少數幾家大型製藥和生物技術公司之間的激烈競爭所塑造的，這些公司有可能顛覆囊腫纖維化市場，尤其是在基因療法被確立為長期解決方案的情況下。

本報告對全球囊腫纖維化市場進行了分析，概述了市場狀況，並介紹了區域趨勢和參與該市場的公司的概況。

各公司的主要策略和發展
- 資金籌措活動
- 併購
- 監管部門核准
- 夥伴關係、合作與擴張
公司簡介
- Vertex Pharmaceuticals Incorporated
- Gilead Sciences, Inc
- Novartis AG
- Sumitomo Pharma (Spirovant Sciences, Inc.)
- Boehringer Ingelheim International GmbH
- Krystal Biotech, Inc.
- 其他

第4章調查方法

簡介目錄圖表

Product Code: BHL3381SA

Global Cystic Fibrosis Market, Analysis and Forecast: 2025-2035

Cystic fibrosis is a genetic disorder that primarily affects the lungs and digestive system, characterized by the production of thick, sticky mucus that can clog airways and lead to respiratory infections, digestive issues, and a range of other health problems. It is one of the most common life-limiting inherited diseases in children and young adults. Cystic fibrosis is caused by mutations in the CFTR gene (Cystic Fibrosis Transmembrane Conductance Regulator), which is responsible for the regulation of salt and water movement in and out of cells.

The cystic fibrosis market is driven by increasing prevalence of cystic fibrosis, advances in targeted therapies, and expanding focus on precision medicine. Cystic fibrosis is a genetic disease with significant prevalence in Western populations, especially in countries like the U.S. and Europe. According to the Cystic Fibrosis Foundation, there are over 30,000 people with cystic fibrosis in the U.S. alone. The steady increase in the number of diagnosed patients is a key driver for the growing demand for cystic fibrosis drugs, especially as the population ages and new treatments become available.

Moreover, the introduction of CFTR modulator therapies has dramatically transformed the treatment landscape for cystic fibrosis. Drugs have proven to significantly improve the quality of life and lung function for patients by targeting the underlying cause of cystic fibrosis mutations in the CFTR gene. These drugs have led to improved life expectancy and reduced hospital admissions, driving further market growth.

Precision medicine is becoming increasingly important in the cystic fibrosis market. Genetic testing and personalized therapies are on the rise, especially in understanding which mutations are causing cystic fibrosis in patients. This trend is likely to boost demand for drugs tailored to specific mutations (e.g., CFTR modulators designed for different genotypes). This focus allows for highly personalized treatment plans, increasing the effectiveness of therapy. Also, greater awareness among healthcare providers and patients about cystic fibrosis, coupled with advancements in newborn screening programs, has led to earlier diagnosis and treatment. Early treatment can delay disease progression, reduce the severity of symptoms, and improve patient outcomes. The result is an expanded patient pool and a growing demand for cystic fibrosis therapies.

The introduction of novel drugs for cystic fibrosis has brought hope for patients but also raised concerns due to their high costs. These costs pose a significant barrier to accessibility, particularly in low- and middle-income countries and among uninsured or underinsured populations. The high price tag of cystic fibrosis drugs is a major challenge for both patients and healthcare systems.

Also, patients with cystic fibrosis require complex, multi-drug regimens, which can involve pulmonary therapies, enzyme replacement, and CFTR modulator therapies. Adherence to these treatments can be difficult, leading to non-compliance and treatment failures. Maintaining a consistent therapy routine can be physically, emotionally, and financially exhausting for patients. While CFTR modulators have brought significant improvements for many cystic fibrosis patients, they are not effective for all cystic fibrosis mutations. This means that certain patient populations still have limited treatment options, particularly those with rarer mutations that do not respond to available CFTR modulators. Drug development for these rare mutations remains a challenge.

Gene therapy is emerging as a groundbreaking treatment approach for cystic fibrosis, offering the potential to directly correct the genetic mutations that cause the disease. The development of CRISPR-based therapies and other gene-editing technologies is being explored to provide long-term or permanent solutions. Also, the orphan drug market for cystic fibrosis is growing, with regulatory agencies like the FDA and EMA granting fast-track approvals and market exclusivity to drugs targeting cystic fibrosis. This is because cystic fibrosis is classified as a rare disease, allowing drug manufacturers to enjoy financial incentives, including extended patent protection and tax credits. This has encouraged more companies to invest in cystic fibrosis drug development, leading to innovative treatments.

While cystic fibrosis is more common in Western populations, global awareness and diagnostic capabilities are increasing in emerging markets. As healthcare infrastructure improves in regions like Asia-Pacific and Latin America, these regions represent significant growth opportunities for cystic fibrosis drug manufacturers. Increased access to genetic testing and CFTR modulator therapies could unlock a large, untapped market.

Moreover, numerous companies are developing drugs to address gaps in the cystic fibrosis treatment landscape, such as novel antibiotics for respiratory infections and drugs targeting rare cystic fibrosis mutations. These innovative solutions offer new growth prospects for both small biotech firms and large pharmaceutical companies. Investment in biologics and targeted therapies presents high revenue potential.

The competitive landscape of the cystic fibrosis market is shaped by strong competition from a few key pharmaceutical and biotechnology firms. These companies have the potential to disrupt the cystic fibrosis market, especially if gene therapies become a viable long-term solution.

Executive Summary

Scope and Definition

Market/Product Definition

Inclusion and Exclusion

Key Questions Answered

Analysis and Forecast Note

1. Global Cystic Fibrosis Market: Industry Outlook

1.1 Market Overview
1.2 Epidemiological Analysis
1.3 Regulatory Landscape
1.4 Key Trends
1.5 Clinical Trial Analysis
1.6 Market Dynamics
- 1.6.1 Impact Analysis
- 1.6.2 Market Drivers
- 1.6.3 Market Restraint
- 1.6.4 Market Opportunities

2. Global Cystic Fibrosis Market, by Region, $Million, 2023-2035

2.1 North America
- 2.1.1 Market Dynamics
- 2.1.2 Market Size and Forecast
  - 2.1.2.1 North America Cystic Fibrosis Market, by Country
    - 2.1.2.1.1 U.S.
2.2 Europe
- 2.2.1 Market Dynamics
- 2.2.2 Market Size and Forecast
  - 2.2.2.1 Europe Cystic Fibrosis Market, by Country
    - 2.2.2.1.1 Germany
    - 2.2.2.1.2 U.K.
    - 2.2.2.1.3 France
    - 2.2.2.1.4 Italy
    - 2.2.2.1.5 Spain
2.3 Asia-Pacific
- 2.3.1 Market Dynamics
- 2.3.2 Market Size and Forecast
  - 2.3.2.1 Asia-Pacific Cystic Fibrosis Market, by Country
    - 2.3.2.1.1 Japan

3. Global Cystic Fibrosis Market - Competitive Landscape and Company Profiles

3.1 Key Strategies and Developments by Company
- 3.1.1 Funding Activities
- 3.1.2 Mergers and Acquisitions
- 3.1.3 Regulatory Approvals
- 3.1.4 Partnerships, Collaborations, and Business Expansions
3.2 Company Profiles
- 3.2.1 Vertex Pharmaceuticals Incorporated
  - 3.2.1.1 Overview
  - 3.2.1.2 Product Portfolio
  - 3.2.1.3 Target Customers
  - 3.2.1.4 Key Personnel
  - 3.2.1.5 Analyst View
- 3.2.2 Gilead Sciences, Inc
  - 3.2.2.1 Overview
  - 3.2.2.2 Product Portfolio
  - 3.2.2.3 Target Customers
  - 3.2.2.4 Key Personnel
  - 3.2.2.5 Analyst View
- 3.2.3 Novartis AG
  - 3.2.3.1 Overview
  - 3.2.3.2 Product Portfolio
  - 3.2.3.3 Target Customers
  - 3.2.3.4 Key Personnel
  - 3.2.3.5 Analyst View
- 3.2.4 Sumitomo Pharma (Spirovant Sciences, Inc.)
  - 3.2.4.1 Overview
  - 3.2.4.2 Product Portfolio
  - 3.2.4.3 Target Customers
  - 3.2.4.4 Key Personnel
  - 3.2.4.5 Analyst View
- 3.2.5 Boehringer Ingelheim International GmbH
  - 3.2.5.1 Overview
  - 3.2.5.2 Product Portfolio
  - 3.2.5.3 Target Customers
  - 3.2.5.4 Key Personnel
  - 3.2.5.5 Analyst View
- 3.2.6 Krystal Biotech, Inc.
  - 3.2.6.1 Overview
  - 3.2.6.2 Product Portfolio
  - 3.2.6.3 Target Customers
  - 3.2.6.4 Key Personnel
  - 3.2.6.5 Analyst View
- 3.2.7 Others