世界のリソソーム蓄積障害治療薬市場 – 業界動向と2030年までの予測

リソソーム蓄積障害治療薬市場分析と規模

リソソーム蓄積症は、酵素不足により体内の細胞に多数の有害な化学物質が異常に蓄積する遺伝性の代謝障害です。この病気はおよそ 50 種類あり、骨格、脳、皮膚、心臓、中枢神経系など、体のさまざまな部位に影響を及ぼします。リソソーム蓄積症は、まだ発見されつつあります。これらの病気の一部には、潜在的な治療法の臨床試験が進行中ですが、多くのリソソーム蓄積症には承認された薬がありません。                           

Data Bridge Market Researchは、2022年に97億3,863万米ドルだったリソソーム蓄積障害治療薬は、2030年までに216億4,692万米ドルに急増し、予測期間中に10.50％のCAGRを達成すると分析しています。リソソーム疾患の有病率の上昇により、「酵素補充療法」がリソソーム蓄積障害治療薬の治療セグメントを支配しています。市場価値、成長率、セグメンテーション、地理的範囲、主要プレーヤーなどの市場シナリオに関する洞察に加えて、Data Bridge Market Researchがまとめた市場レポートには、詳細な専門家分析、患者の疫学、パイプライン分析、価格分析、規制の枠組みも含まれています。

リソソーム蓄積障害治療薬市場の範囲とセグメンテーション

Market Definition

Lysosomal or lysosomal storage disease is a rare hereditary metabolic disease. It is caused by faulty or mutant metabolism, which results in the lack or deficiency of enzymes, resulting in an abnormal material build-up in various body cells. If done before age two, bone marrow transplantation (BMT) successfully prevents progressive mental retardation in children with MPS IH (Hurler disease). Enzyme Replacement Therapy (ERT) is successful for those with Gaucher disease type I. Anemia and low platelet counts have improved, liver and spleen enlargement has decreased significantly, and skeletal abnormalities have improved. Individuals with Gaucher disease types II and III who get ERT also see improvements in these systemic signs.

Lysosomal Storage Disorder Drugs Market Dynamics

Drivers

• Prevalence and Awareness:

The increasing recognition and diagnosis of lysosomal storage disorders worldwide have driven the demand for effective treatment options. As awareness about these rare genetic disorders grows, more patients are being identified, leading to a greater need for drug development.

• Unmet Medical Need

Lysosomal storage disorders are a group of rare genetic diseases characterized by the deficiency of specific enzymes within lysosomes, resulting in the accumulation of undigested materials in cells. These disorders often lead to severe, progressive symptoms and can be life-threatening. Due to the limited treatment options available, there is a significant unmet medical need for effective therapies, driving the development of drugs in this field.

• Orphan Drug Incentives

Governments and regulatory agencies worldwide have implemented various incentives to promote the development of drugs for rare diseases, including lysosomal storage disorders. These incentives may include extended market exclusivity, tax credits, research grants, and accelerated regulatory pathways. Such incentives encourage pharmaceutical companies to invest in research and development efforts for these rare conditions.

• Advances in Biotechnology

The advancements in biotechnology and drug discovery techniques have significantly contributed to developing novel therapies for lysosomal storage disorders. Innovative approaches such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors have shown promising results in preclinical and clinical studies, fueling further investment in this field.

Opportunities

• Large Addressable Market

While individual LSDs are rare, there are over 50 known lysosomal storage disorders, each with unique genetic defects and clinical manifestations. Collectively, these disorders affect a substantial number of patients worldwide. The combined prevalence of LSDs makes for a sizable addressable market, presenting an opportunity for drug developers and manufacturers.

• Limited Treatment Options

Historically, treatment options for LSDs were limited, primarily focusing on symptomatic management. However, recent advancements in understanding the underlying biology of these disorders have led to the development of innovative therapies. The availability of disease-modifying treatments, such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors, has expanded the treatment landscape, providing better options for patients and driving market growth.

Restraints/Challenges

• Limited Patient Population

Lysosomal storage disorders are rare genetic diseases, and each specific disorder affects a relatively small number of patients. The limited patient population poses challenges for drug developers and manufacturers in achieving economies of scale and generating substantial revenue. The small market size may also deter some companies from investing in the research and development of LSD drugs.

• High Development Cost

Developing drugs for rare diseases, including LSDs, can be expensive and time-consuming. The cost of conducting clinical trials, obtaining regulatory approvals, and ensuring safety and efficacy can be substantial. The relatively small patient population and the complex nature of LSDs may result in higher per-patient costs, making the development of LSD drugs financially challenging for some companies.

This   Hospice  Market report provides details of new recent developments, trade regulations, import-export analysis, production analysis, value chain optimization, market share, the impact of domestic and localized market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on the Hospice  Market Contact Data Bridge Market Research for an Analyst Brief, our team will help you take an informed market decision to achieve market growth.

Recent Development

• In January 2021, M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced that the U.S. Food and Drug Administration (FDA) granted six rare pediatric disease designations (RPDDs) for various programs within its development pipeline for LSDs, including four recombinant enzyme and two gene therapy programs
• In July 2019, Takeda Pharmaceutical Company launched an enzyme replacement therapy portfolio for lysosomal storage disorders (LSD) in India, expanding its range of rare disease therapies nationwide

Global Lysosomal Storage Disorder Drugs Scope

The lysosomal storage disorder drugs are segmented on the basis of treatment, indication, and end User. The growth amongst these segments will help you analyze meagre growth segments in the industries and provide the users with a valuable market overview and market insights to help them make strategic decisions for identifying core market applications.

Treatment

• Enzyme Replacement Therapy
• Stem Cell Therapy
•  Substrate Reduction Therapy
•  Others

Indication  

• Gaucher's Disease
• Fabry Disease
•  Pompe’s Syndrome
•  Mucopolysaccharidosis,
• Others

End user

• Hospitals,
• Clinics
• Others

Lysosomal Storage Disorder Drugs Regional Analysis/Insights

The lysosomal storage disorder drugs is analysed, and market size insights and trends are provided by country, treatment, indication, and end user as referenced above.

The countries covered in the lysosomal storage disorder drugs report are U.S., Canada and Mexico, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific , Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa, Brazil, Argentina and Rest of South America

North America dominates the lysosomal storage disorder drugs because of the strong base of healthcare facilities, the strong presence of major players in the market, the rising prevalence of lysosomal disease, and the rising number of research activities in this region.   

Asia-Pacific is expected to witness significant growth during the forecast period of 2023 to 2030 due to the increase in government initiatives to promote awareness, rise in medical tourism, growing research activities in the region, availability of massive untapped markets, large population pool, and the growing demand for quality healthcare in the region.

レポートの国別セクションでは、市場の現在および将来の傾向に影響を与える国内市場における個別の市場影響要因と規制の変更も提供しています。下流および上流のバリュー チェーン分析、技術動向、ポーターの 5 つの力の分析、ケース スタディなどのデータ ポイントは、各国の市場シナリオを予測するために使用される指標の一部です。また、国別データの予測分析を提供する際には、グローバル ブランドの存在と可用性、および地元および国内ブランドとの競争が激しいか少ないために直面​​する課題、国内関税と貿易ルートの影響も考慮されます。   

ヘルスケアインフラの成長 導入基盤と新技術の浸透

リソソーム蓄積障害薬は、各国の医療設備支出の増加、リソソーム蓄積障害薬のさまざまな種類の製品のインストールベース、ライフライン曲線を使用したテクノロジーの影響、医療規制シナリオの変化とそれらがリソソーム蓄積障害薬に与える影響に関する詳細な市場分析も提供します。データは、2010年から2020年の履歴期間について利用可能です。

競争環境とリソソーム蓄積障害治療薬のシェア分析

リソソーム蓄積障害薬の競争状況では、競合他社ごとに詳細が提供されます。含まれる詳細には、会社概要、会社の財務状況、収益、市場の可能性、研究開発への投資、新しい市場への取り組み、世界的なプレゼンス、生産拠点と施設、生産能力、会社の強みと弱み、製品の発売、製品の幅と広さ、アプリケーションの優位性などがあります。提供されている上記のデータ ポイントは、リソソーム蓄積障害薬に関連する会社の焦点にのみ関連しています。

リソソーム蓄積障害治療薬を扱っている主な企業は次のとおりです。

• ノバルティスAG（スイス）
• ジョンソン・エンド・ジョンソン プライベート・リミテッド（米国）
• テバ製薬工業株式会社（アイルランド）
• メルク社（米国）
• アラガン（アイルランド）
• ファイザー社（米国）
• グラクソ・スミスクライン（英国）
• サノフィ（フランス）
• メルクKGaA（ドイツ）
• アボット（米国）
• ベーリンガーインゲルハイムインターナショナルGmbH（ドイツ）
• 武田薬品工業株式会社（日本）
• アミカス・セラピューティクス社（米国）
• モデナ社（米国）
• Greenovation Biotech GmbH（ドイツ）
• バイオマリン（米国）
• JCRファーマ株式会社（日本）
• ISU ABXIS（韓国）
• イドルシア・ファーマシューティカルズ社（スイス）
• AVROBIO社（米国）
• Resverlogix Corp.（カナダ）
• エンザイバント（米国）
• CHIESI Farmaceutici SpA (イタリア) 

SKU-59872