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市場調査レポート
商品コード
1643921
酵素補充療法の世界市場(2025年~2032年)Global Enzyme Replacement Therapy Market - 2025-2032 |
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カスタマイズ可能
適宜更新あり
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酵素補充療法の世界市場(2025年~2032年) |
出版日: 2025年01月27日
発行: DataM Intelligence
ページ情報: 英文 176 Pages
納期: 即日から翌営業日
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世界の酵素補充療法の市場規模は、2024年に110億5,000万米ドルに達し、2032年には216億7,000万米ドルに達すると予測され、予測期間2025年~2032年のCAGRは8.7%で成長する見込みです。
酵素補充療法(ERT)は、精製されたヒト、動物、または組換え酵素を投与することにより、先天性酵素欠損症に対処することを目的とした治療法です。この治療法は主に、重要な代謝過程に必要な特定の酵素が欠乏しているために重篤な障害や早死にを引き起こす可能性のある、まれな遺伝性疾患に用いられます。
ERTでは、ヒトまたは動物の組織由来の酵素、あるいは組換えDNA技術によって生産された酵素を静脈内に注入します。その目的は、遺伝性酵素欠乏症候群の患者において、欠乏または欠損した酵素を補充することです。酵素活性を回復させることにより、ERTは細胞内に有毒基質が蓄積するのを防ぎ、症状を緩和し、疾患の進行を遅らせます。
ERTは、グルコセレブロシダーゼという酵素が欠損しているゴーシェ病などのライソゾーム貯蔵疾患によく用いられます。この疾患では、ERTは酵素欠損により細胞内に蓄積する脂肪分子であるグルコセレブロシドの分解を補助します。ERTで治療される他の疾患には、ファブリー病やα-1-アンチトリプシン欠乏症があります。これらの要因が、世界の酵素補充療法市場拡大の原動力となっています。
促進要因と抑制要因
遺伝性疾患の有病率の増加
遺伝性疾患の有病率の増加は、酵素補充療法(ERT)市場の重要な促進要因です。これらの疾患の罹患率が上昇するにつれて、ERTを含む効果的な治療に対する需要も相応に拡大すると予想されます。ERTは、酵素の欠乏によって引き起こされる遺伝性疾患であるリソソーム貯蔵疾患に特に関連しています。
遺伝子疾患は人口のかなりの部分に影響を及ぼしています。2022年12月の米国立衛生研究所(NIH)のデータによると、ゴーシェ病(GD)は最も有病率の高いライソゾーム貯蔵障害(LSD)の1つで、70以上の遺伝性代謝疾患を包含し、その頻度は合わせて出生5,000人に1人程度です。一般集団におけるGDの発症率は、出生10万人あたり0.39~5.80人と推定されており、より正確には出生10万人あたり1.5人と推定されています。GDの有病率の推定値もさまざまで、人口10万人当たり0.70~1.75人、世界の有病率は人口10万人当たり約0.9人とされています。
さらに、業界の主要企業は遺伝性疾患の治療に注力し、革新的な製品の上市や承認を導入しており、これが酵素補充療法の世界市場成長の原動力となっています。例えば、2023年9月、Amicus Therapeuticsは、米国食品医薬品局(FDA)がPombiliti(cipaglucosidase alfa-atga)とOpfolda(miglustat)65mgカプセルの併用を承認したと発表しました。この革新的な2剤併用療法は、特に体重40kg以上の後期発症ポンペ病(LOPD)で、現在使用しているERTで改善が認められない成人に適応があります。
また、アミカス・セラピューティクスは2023年6月、欧州委員会(EC)が後期発症ポンペ病(LOPD)成人患者に対する2成分療法の一環として、酵素安定化剤カプセル65mgのオプフォルダ(一般名:ミグルスタット)を承認したと発表しました。この承認は、オプフォルダをポンビリチ(シパグルコシダーゼ・アルファ)と併用することを可能にするものであり、意義深いものです。これらすべての要因が、世界の酵素補充療法市場に需要をもたらしています。
さらに、バイオテクノロジーの進歩に対する需要の高まりも、世界の酵素補充療法市場の拡大に寄与しています。
高い治療費
酵素補充療法(ERT)に関連する高額な治療費が市場の大きな抑制要因となっており、患者のアクセスに影響を与え、ヘルスケアシステムに大きな圧力をかけています。こうした経済的障壁に対処することは、ERTへのアクセスを改善し、希少遺伝性疾患の患者がその状態を効果的に管理するべく必要な治療を受けられるようにするために極めて重要です。
このように、上記の要因は世界の酵素補充療法市場の潜在的成長を制限している可能性があります。
The global enzyme replacement therapy market reached US$11.05 billion in 2024 and is expected to reach US$21.67 billion by 2032, growing at a CAGR of 8.7 % during the forecast period 2025-2032.
Enzyme replacement therapy (ERT) is a medical treatment aimed at addressing congenital enzyme deficiencies by administering purified human, animal, or recombinant enzymes. This therapy is primarily utilized for rare genetic disorders that can lead to severe disability and premature death due to the lack of specific enzymes necessary for vital metabolic processes.
ERT involves the intravenous infusion of enzymes derived from human or animal tissues or produced through recombinant DNA technology. The goal is to replace the missing or deficient enzyme in patients with inherited enzyme deficiency syndromes. By restoring the enzyme's activity, ERT helps prevent the accumulation of toxic substrates within cells, thereby alleviating symptoms and slowing disease progression.
ERT is commonly used for lysosomal storage diseases, such as Gaucher disease, where there is a deficiency in the enzyme glucocerebrosidase. In this condition, ERT aids in breaking down glucocerebroside, a fatty molecule that accumulates in cells due to enzyme deficiency. Other conditions treated with ERT include Fabry disease and alpha-1-antitrypsin deficiency. These factors have driven the global enzyme replacement therapy market expansion.
Market Dynamics: Drivers & Restraints
Increasing Prevalence of Genetic Disorders
The increasing prevalence of genetic disorders is a significant driver in the enzyme replacement therapy (ERT) market. As the incidence of these disorders rises, the demand for effective treatments, including ERT, is expected to grow correspondingly. ERT is particularly relevant for lysosomal storage diseases, which are genetic disorders caused by enzyme deficiencies.
Genetic disorders affect a substantial portion of the population. According to the National Institutes of Health (NIH) data in December 2022, Gaucher disease (GD) is one of the most prevalent lysosomal storage disorders (LSDs), which encompasses over 70 inherited metabolic diseases with a combined frequency of approximately 1 in 5,000 live births. The incidence of GD in the general population has been estimated to range from 0.39 to 5.80 per 100,000 live births, with a more precise estimate of 1.5 cases per 100,000 live births. Prevalence estimates for GD vary as well, with figures ranging from 0.70 to 1.75 per 100,000 population and a global prevalence of approximately 0.9 cases per 100,000 inhabitants.
Furthermore, key players in the industry more focus on the treatment of genetic disorders & introducing innovative product launches and approvals that would drive this global enzyme replacement therapy market growth. For instance, in September 2023, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted approval for Pombiliti (cipaglucosidase alfa-atga) in combination with Opfolda (miglustat), 65 mg capsules. This innovative two-component therapy is specifically indicated for adults with late-onset Pompe disease (LOPD) who weigh 40 kg or more and have not shown improvement with their current enzyme replacement therapy (ERT).
Also, in June 2023, Amicus Therapeutics announced that the European Commission (EC) has approved Opfolda (miglustat), a 65 mg enzyme stabilizer capsule, as part of a two-component therapy for adults with late-onset Pompe disease (LOPD). This approval is significant because it allows Opfolda to be used in conjunction with Pombiliti (cipaglucosidase alfa). All these factors demand the global enzyme replacement therapy market.
Moreover, the rising demand for the advancements in biotechnology contributes to the global enzyme replacement therapy market expansion.
High Treatment Costs
The high treatment costs associated with enzyme replacement therapy (ERT) pose a significant restraint in the market, impacting patient access and placing considerable pressure on healthcare systems. Addressing these financial barriers is crucial for improving access to ERT and ensuring that individuals with rare genetic disorders receive the necessary treatments to manage their conditions effectively.
Thus, the above factors could be limiting the global enzyme replacement therapy market's potential growth.
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The global enzyme replacement therapy market is segmented based on enzyme type, indication type, route of administration, end-user, and region.
The agalsidase beta segment is expected to dominate the global enzyme replacement therapy market share
The agalsidase beta segment holds a major portion of the global enzyme replacement therapy market share and is expected to continue to hold a significant portion of the global enzyme replacement therapy market share during the forecast period.
Fabrazyme (agalsidase beta) is an approved enzyme replacement therapy (ERT) specifically developed for the treatment of Fabry disease, a rare genetic disorder resulting from a deficiency in the enzyme alpha-galactosidase A (a-Gal A). This enzyme deficiency leads to the accumulation of toxic molecules, particularly globotriaosylceramide (Gb3), within cells, causing various health complications, including damage to the heart, kidneys, and nervous system.
Fabry disease is caused by a mutation in the GLA gene, which provides the instructions for producing the enzyme alpha-galactosidase A (a-Gal A). This enzyme is responsible for breaking down the fatty molecule globotriaosylceramide (Gb3), also known as GL-3. When individuals have Fabry disease, they cannot produce sufficient functional a-Gal A, leading to an inability to effectively break down Gb3. Consequently, this fatty molecule accumulates to toxic levels in various tissues and organs, particularly affecting the kidneys and heart, which causes damage and results in the symptoms associated with the disease.
To address this deficiency, Fabrazyme is an enzyme replacement therapy that contains agalsidase beta, a functional version of the missing a-Gal A enzyme. This lab-produced enzyme has the same amino acid sequence as the natural enzyme and is created using recombinant DNA technology. In this process, cells are modified to include the GLA gene, enabling them to produce the enzyme.
The replacement enzyme provided by Fabrazyme can effectively break down Gb3, helping to prevent its toxic buildup in cells and thereby mitigating the harmful effects of Fabry disease. Furthermore, key players in the industry more focus on clinical trials and getting positive results that would propel this segment's growth in the global enzyme replacement therapy market.
For instance, in October 2024, Chiesi Global Rare Diseases announced the results of their BRIGHT trial, which evaluated the efficacy of the enzyme-replacement therapy drug ELFABRIO (pegunigalsidase alfa-iwxj) administered at twice the approved dose in patients who have previously received treatment with agalsidase alfa or agalsidase beta. The standard approved dosage for ELFABRIO is 1 mg per kilogram (kg), given every two weeks. These factors have solidified the segment's position in the global enzyme replacement therapy market.
North America is expected to hold a significant position in the global enzyme replacement therapy market share
North America holds a substantial position in the global enzyme replacement therapy market and is expected to hold most of the market share.
The North America enzyme replacement therapy (ERT) market is experiencing robust growth, driven by several key factors that enhance patient access to effective treatments for rare genetic disorders. Approximately 25 to 35 million Americans are affected by rare diseases, significantly increasing the demand for effective treatments such as ERT. The growing recognition and diagnosis of these conditions contribute to the expansion of the enzyme replacement therapy market.
North America boasts a well-established healthcare system that supports advanced medical technologies and treatment options. This infrastructure facilitates patient access to enzyme replacement therapies and enhances the overall treatment experience.
The regulatory environment in North America is favorable for ERT, particularly for drugs with Orphan Drug Designation, which accelerates the approval process and allows patients quicker access to new therapies. Significant investments from pharmaceutical companies in R&D are driving innovation in ERT products. The introduction of new recombinant enzymes has expanded treatment options, improving efficacy and patient outcomes.
Furthermore, a major number of key players' presence, innovative product launches, and approvals would drive this global enzyme replacement therapy market growth. For instance, in November 2023, Takeda announced that the U.S. Food and Drug Administration (FDA) has approved ADZYNMA (ADAMTS13, recombinant-krhn), marking it as the first and only recombinant enzyme replacement therapy specifically indicated for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP).
Also, in February 2023, Chiesi Global Rare Diseases, a division of Chiesi Farmaceutici S.p.A., announced that the U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) to treat non-central-nervous-system-manifestations of alpha-mannosidosis (AM) in both adult and pediatric patients. Alpha-mannosidosis is an ultra-rare, progressive lysosomal storage disorder resulting from a deficiency in the enzyme a-mannosidase. Thus, the above factors are consolidating the region's position as a dominant force in the global enzyme replacement therapy market.
Asia Pacific is growing at the fastest pace in the global enzyme replacement therapy market share
Asia Pacific holds the fastest pace in the global enzyme replacement therapy market and is expected to hold most of the market share.
The Asia-Pacific enzyme replacement therapy (ERT) market is anticipated to witness substantial growth, propelled by several key factors that enhance the demand for effective treatments for rare genetic disorders. The region is experiencing a notable rise in the diagnosis of rare genetic disorders, with approximately 258 million individuals in Asia and the Pacific affected. This growing patient population significantly heightens the demand for effective ERT options.
Increased investments in healthcare infrastructure across countries such as China and India are contributing to market growth. These investments improve access to advanced medical technologies and treatment options, facilitating the availability of enzyme replacement therapies. Technological advancements in biotechnology have led to improved formulations and delivery systems for ERT products. These innovations enhance the efficacy and safety profiles of therapies, making them more appealing to both healthcare providers and patients.
Supportive government policies aimed at improving healthcare access play a crucial role in advancing ERT. Regulatory support for new ERT approvals in countries like Japan has enhanced treatment options for patients with rare diseases. Increased awareness and education about rare diseases among healthcare providers lead to earlier diagnosis and intervention, driving demand for ERT. Educational initiatives ensure that practitioners are well-informed about available therapies and their benefits.
Furthermore, key players' strategies such as investments & agreements and new product launches & approvals would drive this global enzyme replacement therapy market growth. For instance, in January 2024, JCR Pharmaceuticals Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to JR-441, an experimental enzyme replacement therapy for Sanfilippo syndrome type A (MPS IIIA). This designation is particularly important as it applies to therapies intended for conditions affecting fewer than 200,000 individuals in the U.S. It provides several incentives aimed at accelerating clinical development, including tax credits for clinical trials and the potential for seven years of market exclusivity upon approval.
Also, in May 2024, Japan's Shionogi & Co. announced a significant $150 million deal to develop the first oral therapy for Pompe disease, a rare genetic disorder. This agreement involves an exclusive worldwide licensing arrangement with Maze Therapeutics for their investigational drug, MZE001, which functions as an oral glycogen synthase 1 (GYS1) inhibitor. Thus, the above factors are consolidating the region's position as the fastest-growing force in the global enzyme replacement therapy market.
The major global players in the enzyme replacement therapy market include Sanofi, Pfizer Inc., BioMarin Pharmaceutical Inc., Protalix Biotherapeutics Inc., Takeda Pharmaceuticals Inc., AbbVie., CHIESI Farmaceutici S.p.A., Alexion Pharmaceuticals, Inc., and Amicus Therapeutics, Inc. among others.
The global enzyme replacement therapy market report delivers a detailed analysis with 60+ key tables, more than 50 visually impactful figures, and 176 pages of expert insights, providing a complete view of the market landscape.
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