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市场调查报告书
商品编码
1733396
2026 年至 2032 年 Alpha-1 抗胰蛋白酶缺乏症增强疗法市场(按产品类型、最终用户和地区)Alpha-1 Antitrypsin Deficiency Augmentation Therapy Market by Product Type (Glassia, Aralast NP, Prolastin C, Zemaira/Respreeza), End-User (Hospitals, Specialty Clinics), & Region for 2026-2032 |
α-1 抗胰蛋白酶缺乏症盛行率的不断上升以及人们对增强疗法在控制肺部疾病方面的益处的认识不断提高是推动 α-1 抗胰蛋白酶缺乏症增强疗法市场增长的主要力量。根据 Verified Market Research 分析师的预测,α-1 抗胰蛋白酶缺乏症增强疗法市场在 2024 年的价值约为 15.9 亿美元,并在预测期内达到 26.9 亿美元的估值。
为了满足 α-1 抗胰蛋白酶缺乏症患者未满足的医疗需求,对增强治疗方案的需求日益增长,持续的研究和开发工作正在推动 α-1 抗胰蛋白酶缺乏症增强治疗市场向前发展。这将有助于市场在 2024 年至 2032 年期间以 6.8% 的复合年增长率成长。
Alpha-1 抗胰蛋白酶缺乏症增强疗法市场定义/概述
Alpha-1 抗胰蛋白酶缺乏症增强疗法是治疗方法,即身体无法产生足够的 Alpha-1 抗胰蛋白酶 (AAT) 蛋白,从而增加肺部和肝损伤的风险。这种治疗方法包括定期静脉注射由人体血浆製成的纯 AAT 蛋白,以增加血液中的 AAT 水平。这种治疗方法试图保护肺部免受白血球产生的酵素造成的损害,特别是对于易患肺气肿或慢性阻塞性肺病(COPD)等肺部疾病的人。
此外,增强疗法已被证明可以减缓肺部疾病的进展,并最大限度地减少 α-1 抗胰蛋白酶缺乏症患者的病情恶化次数。研究还表明,这种缺陷可能对肝病有益。
Alpha-1 抗胰蛋白酶缺乏症 (AATD) 是一种导致严重肺部和肝臟疾病的遗传性疾病。医疗保健专业人员和公众对 AATD 的认识不断提高,从而可以更早、更频繁地进行诊断。再加上全球基因筛检方法的兴起,适合接受增强疗法的患者群体正在不断扩大,从而推动市场成长。
先进的生物技术方法和基因治疗的进步是 AATD 增强疗法市场的主要驱动力。这些发展不仅提高了现有治疗方法的有效性和安全性,而且促进了新治疗方法的发现。例如,重组 DNA 技术的创新有可能提供更稳定、更有效的 α-1 抗胰蛋白酶,使患者能够更好地控制 AATD 症状并改善他们的整体健康。
此外,政府试图改善医疗保健基础设施并鼓励孤儿药的研究和註册也推动了 AATD 增强疗法市场的发展。在许多地区,AATD 治疗都受到优惠的报销规定和罕见疾病研究激励措施的支持,使患者更能负担得起。这些措施有可能大幅减轻患者的经济负担,同时提高增强疗法的接受度。
α1-抗胰蛋白酶缺乏症 (AATD) 的增强疗法通常非常昂贵,限制了其普及,特别是在医疗体係不发达的地区和保险覆盖率较差的国家。成本高昂是由于生产 α-1 抗胰蛋白酶蛋白的难度,以及该药物被归类为孤儿药物。这些经济障碍阻碍了患者获得所需的治疗方法,从而限制了市场的成长潜力。
此外,目前 AATD 增强治疗选择有限,其效用和成本效益仍有争议。有些患者对常规药物治疗没有反应,而透过增强疗法来阻止肺部疾病的进展,其成功率也各不相同。此外,终生治疗的需要对患者的依从性和满意度提出了挑战。治疗选择有限,加上药物疗效参差不齐,对市场扩张和病患治疗效果构成了挑战。
The growing prevalence of Alpha-1 Antitrypsin deficiency and growing awareness of the benefits of augmentation therapy in controlling lung disorders are key forces fueling the growth of the Alpha-1 Antitrypsin deficiency augmentation therapy market. According to the analyst from Verified Market Research, the Alpha-1 Antitrypsin deficiency augmentation therapy market is estimated to reach a valuation of USD 2.69 Billion over the forecast subjugating around USD 1.59 Billion valued in 2024.
The growing need for enhanced therapy options to address the unmet medical needs of Alpha-1 Antitrypsin deficiency patients, together with continuing research & development initiatives, is propelling the Alpha-1 Antitrypsin deficiency augmentation therapy market forward. It enables the market to grow at a CAGR of 6.8% from 2024 to 2032.
Alpha-1 Antitrypsin Deficiency Augmentation Therapy Market: Definition/Overview
Alpha-1 Antitrypsin Deficiency Augmentation Therapy is a medical treatment that addresses a hereditary problem in which the body does not create enough alpha-1 antitrypsin (AAT) protein, which increases the risk of lung and liver disorders. The therapy entails injecting pure AAT protein generated from human plasma intravenously regularly to boost AAT levels in the blood. The therapy seeks to protect the lungs against damage caused by enzymes generated by white blood cells, especially in people who are prone to lung disorders such as emphysema and chronic obstructive pulmonary disease (COPD) due to the deficit.
Furthermore, augmentation therapy has been found to decrease the progression of lung illness and minimize the number of exacerbations in patients with Alpha-1 Antitrypsin Deficiency. Also, research suggests that this deficiency may have benefits in terms of liver disease
Alpha-1 Antitrypsin Deficiency (AATD) is a hereditary disorder that can cause severe lung and liver illnesses. The growing awareness of AATD among healthcare professionals and the general public is resulting in earlier and more frequent diagnoses. This, combined with the global increase in genetic screening methods, is broadening the patient pool eligible for augmentation therapy, resulting in market growth.
The advancement of sophisticated biotechnological approaches and genetic therapies is a major driver of the AATD augmentation therapy market. These developments not only improve the efficacy and safety of existing medications but also make it easier to discover new treatment choices. Innovations in recombinant DNA technology, for example, offer the potential to provide more stable and effective versions of alpha-1 antitrypsin, allowing patients to better manage their AATD symptoms and enhance their overall health.
Furthermore, government attempts to improve healthcare infrastructure and facilitate the research and registration of orphan pharmaceuticals are also propelling the AATD augmentation therapy market. In many areas, AATD medicines are backed by advantageous reimbursement rules and incentives for rare illness research, making them more affordable to patients. These policies have the potential to dramatically lower patients' financial burdens while also increasing the adoption rate of augmentation therapy.
Augmentation therapy for Alpha-1 Antitrypsin Deficiency (AATD) is frequently prohibitively expensive, limiting their availability, particularly in locations with underdeveloped healthcare systems or in nations where insurance coverage for such medicines is inadequate. The high cost is due in part to the difficult manufacturing methods involved in making the alpha-1 antitrypsin protein, as well as the medicines' classification as orphan pharmaceuticals, which are created for disorders that afflict only a small fraction of the population. This financial barrier stops patients from accessing needed therapies, limiting the market's growth potential.
Furthermore, currently, AATD augmentation therapy options are limited, and there is continuous disagreement over their usefulness and cost-effectiveness. Some individuals do not respond well to conventional medications, and the effectiveness of augmentation therapy in halting the course of lung disease varies. Also, the requirement for lifelong treatment is a difficulty in terms of patient compliance and satisfaction. The limited number of treatment alternatives, as well as the varied efficacy of medicines, pose challenges to market expansion and patient outcomes.
According to VMR analyst, the prolastin C segment is expected to dominate the segment within the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. Prolastin-C, one of the first augmentation medicines available for AATD, has a long track record of success and safety. Several studies and real-world evidence have supported its usage in AATD patients to reduce the progression of emphysema. This robust evidence foundation has made it a popular choice among healthcare providers and people, adding to its market dominance.
Prolastin-C, as a market pioneer in AATD augmentation therapy, enjoys strong brand recognition and loyalty from physicians and patients. Years of great experiences and belief in the product's effectiveness and safety are common reasons for loyalty. Many patients and healthcare providers prefer to continue with a tried-and-true therapy rather than transition to newer alternatives with which they are unfamiliar.
Furthermore, the manufacturer of Prolastin-C has created comprehensive patient support initiatives, which have greatly improved its market position. These initiatives may include financial help, instructional materials, and individualized patient support services. Such assistance is critical for individuals suffering from chronic illnesses such as AATD, as it can enhance medication adherence, access to therapy, and overall patient happiness. These programs transform Prolastin-C from a medicine into a holistic treatment solution, which can be a deciding factor for many patients and caregivers when selecting an augmentation therapy.
The hospital segment is estimated to hold the largest share of the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. Hospitals provide a wide range of medical treatments, including integrated care for patients with complicated illnesses such as AATD. They house interdisciplinary teams made up of pulmonologists, hepatologists, genetic counselors, and other specialists who can work together to provide tailored therapy options. Hospitals' capacity to provide comprehensive care from diagnosis to treatment and follow-up in one location makes them the preferred choice for many patients, driving market share.
Hospitals are often outfitted with advanced diagnostic equipment and treatment facilities required for the proper care of AATD. This includes specialist equipment for genetic testing, lung function tests, and access to the most recent augmentation medicines. The availability of cutting-edge healthcare technology, as well as the ability to perform sophisticated treatments such as intravenous infusion of alpha-1 antitrypsin, contribute to their market dominance.
Furthermore, hospitals have more strong systems in place to handle insurance and payment policies, making it easier for patients to receive covered services. Because of the intricacy and expensive cost of AATD augmentation therapy, many patients rely heavily on insurance. Hospitals' ability to properly manage these financial issues can remove considerable hurdles to treatment, increasing patient appeal and, as a result, market share.
The North American region is estimated to dominate the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. North America, particularly the United States, has a sophisticated healthcare infrastructure that provides extensive access to cutting-edge medical technologies, facilities, and specialized healthcare experts. This region has one of the highest healthcare expenditures per capita internationally, allowing for major investment in uncommon diseases such as AATD. The availability of specialized treatment centers, as well as an emphasis on early detection and management of genetic diseases, are critical for effective AATD treatment, leading to the region's market domination.
Furthermore, North America is home to some of the world's leading pharmaceutical and biotechnology businesses, which are pioneering research and development (R&D) in uncommon diseases such as AATD. The region's strong emphasis on innovation, combined with large R&D investments and a favorable regulatory environment for drug research and approval, has resulted in the introduction of several augmentation medicines. This ecosystem not only speeds up the availability of new medications but also ensures a consistent pipeline of innovative therapies for AATD.
Asia Pacific is estimated to exhibit the highest growth within the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. In Asia-Pacific, healthcare professionals and the general public are becoming more aware of rare disorders, such as AATD. Patient advocacy groups, healthcare providers, and government health departments have all worked to educate the public and medical community, contributing to the growth in awareness. Increased awareness leads to higher screening and diagnosis rates, which expands the patient population eligible for augmentation therapy. Early diagnosis is critical for efficiently managing AATD, which drives the demand for augmentation therapy.
Furthermore, many Asian-Pacific countries are experiencing rapid economic expansion, which is driving up healthcare spending at both the government and individual levels. As disposable incomes rise and health insurance coverage expands, more people will be able to afford modern treatments, including expensive therapy for illnesses such as AATD. Also, regional governments are increasingly realizing the necessity of tackling rare diseases, which has resulted in increased funding for healthcare services as well as support for orphan medication research and approval.
The competitive landscape of the Alpha-1 Antitrypsin deficiency augmentation therapy market is marked by ongoing research and development initiatives aimed at improving therapeutic efficacy and accessibility. Also, the introduction of biosimilar goods, as well as the prospect of generic competition, offer market participants both difficulties and opportunities.
Some of the prominent players operating in the Alpha-1 Antitrypsin deficiency augmentation therapy market include:
Takeda Pharmaceutical Company Limited
CSL Behring
Paramount Therapeutics
Vertex Pharmaceuticals Incorporated
Shire plc
Chiesi Farmaceutici SpA
Shanghai RAAS Biotechnology Co.
JCR Pharmaceuticals Co.
Instituto Butantan
Lonza Group Ltd
Samsung BioLogics
Boehringer Ingelheim
In February 2024, Grifols conducted Cohort 1 in a clinical investigation of Alpha-1 15, a first-in-human subcutaneous dosage alternative for AATD, to provide patients with a more comfortable home administration option. This development has substantial implications for patient convenience and autonomy.
In November 2024, Intellia Therapeutics intended to focus on NTLA-3001, a CRISPR-mediated in vivo gene insertion candidate for AATD-associated lung illness. This novel technique seeks to permanently restore functional A1AT protein expression with a single dose, potentially removing the need for weekly intravenous infusions or lung transplants in extreme cases. A Clinical Trial Application (CTA) submission for a Phase 1 study of NTLA-3001 is anticipated for the first quarter of 2024.
In June 2022, Takeda and Arrowhead Pharmaceuticals announced the findings of a Phase 2 study of Fazirsiran in AATD patients in the New England Journal of Medicine. The study found significant decreases in liver globule burden and improvements in indicators of liver damage. This collaboration seeks to co-develop and maybe co-commercialize Fazirsiran, which offers a promising novel therapy for AATD.