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市場調查報告書
商品編碼
1733396
2026 年至 2032 年 Alpha-1 抗胰蛋白酶缺乏症增強療法市場(按產品類型、最終用戶和地區)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斷上升以及人們對增強療法在控制肺部疾病方面的益處的認知不斷提高是推動 α-1 抗胰蛋白酶缺乏症增強療法市場成長的主要力量。根據 Verified Market Research 分析師的預測,α-1 抗胰蛋白酶缺乏症增強療法市場在 2024 年的價值約為 15.9 億美元,並在預測期內達到 26.9 億美元的估值。
為了滿足 α-1 抗胰蛋白酶缺乏症患者未滿足的醫療需求,對增強治療方案的需求日益成長,持續的研究和開發工作正在推動 α-1 抗胰蛋白酶缺乏症增強治療市場向前發展。這將有助於市場在 2024 年至 2032 年期間以 6.8% 的複合年成長率成長。
Alpha-1 抗胰蛋白酶缺乏症增強療法市場定義/概述
Alpha-1 抗胰蛋白酶缺乏症增強療法是治療方法,即身體無法產生足夠的 Alpha-1 抗胰蛋白酶 (AAT) 蛋白,從而增加肺部和肝損傷的風險。這種治療方法包括定期靜脈注射由人體血漿製成的純 AAT 蛋白,以增加血液中的 AAT 水平。這種治療方法試圖保護肺部免受白血球產生的酵素造成的損害,特別是對於易患肺氣腫或慢性阻塞性肺病(COPD)等肺部疾病的人。
此外,增強療法已被證明可以減緩肺部疾病的進展,並最大限度地減少 α-1 抗胰蛋白酶缺乏症患者的病情惡化次數。研究還表明,這種缺陷可能對肝病有益。
Alpha-1 抗胰蛋白酶缺乏症 (AATD) 是一種導致嚴重肺部和肝臟疾病的遺傳性疾病。醫療保健專業人員和公眾對 AATD 的認知不斷提高,從而可以更早、更頻繁地進行診斷。再加上全球基因篩檢方法的興起,適合接受增強療法的患者群體正在不斷擴大,從而推動市場成長。
先進的生物技術方法和基因治療的進步是 AATD 增強療法市場的主要驅動力。這些發展不僅提高了現有治療方法的有效性和安全性,而且促進了新治療方法的發現。例如,重組 DNA 技術的創新有可能提供更穩定、更有效的 α-1 抗胰蛋白酶,使患者能夠更好地控制 AATD 症狀並改善他們的整體健康。
此外,政府試圖改善醫療保健基礎設施並鼓勵孤兒藥的研究和註冊也推動了 AATD 增強療法市場的發展。在許多地區,AATD 治療都受到優惠的報銷規定和罕見疾病研究激勵措施的支持,使患者更能負擔得起。這些措施有可能大幅減輕患者的經濟負擔,同時提高增強療法的接受度。
α1-抗胰蛋白酶缺乏症 (AATD) 的增強療法通常非常昂貴,限制了其普及,特別是在醫療體係不發達的地區和保險覆蓋率較差的國家。成本高昂是由於生產 α-1 抗胰蛋白酶蛋白的難度,以及該藥物被歸類為孤兒藥物。這些經濟障礙阻礙了患者獲得所需的治療方法,從而限制了市場的成長潛力。
此外,目前 AATD 增強治療選擇有限,其效用和成本效益仍有爭議。有些患者對常規藥物治療沒有反應,而透過增強療法來阻止肺部疾病的進展,其成功率也各不相同。此外,終生治療的需要對患者的依從性和滿意度提出了挑戰。治療選擇有限,加上藥物療效參差不齊,對市場擴張和病患治療效果構成了挑戰。
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.