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表紙：ヘルマンスキー・パドラック症候群の世界市場-2023年～2030年
市場調査レポート
商品コード
1345435

ヘルマンスキー・パドラック症候群の世界市場-2023年～2030年

Global Hermansky Pudlak Syndrome Market - 2023-2030
出版日: | 発行: DataM Intelligence | ページ情報: 英文 186 Pages | 納期: 約2営業日

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ヘルマンスキー・パドラック症候群の世界市場-2023年～2030年
出版日: 2023年09月06日
発行: DataM Intelligence
ページ情報: 英文 186 Pages
納期: 約2営業日
ご注意事項 :
本レポートは最新情報反映のため適宜更新し、内容構成変更を行う場合があります。ご検討の際はお問い合わせください。
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  • 概要
  • 目次
概要

概要

ヘルマンスキー・パドラック症候群（HPS）は、アダプター蛋白質3（AP-3）とリソソーム関連小器官複合体1、2、3（BLOC-1、BLOC-2、BLOC-3）の4つの蛋白質複合体の構成要素をコードする11の遺伝子のうち1つの遺伝子のホモ接合体または複合ヘテロ接合体変異によって引き起こされます。この4つの複合体はすべて、リソソームとリソソーム関連オルガネラ（LROS）の細胞内での生合成と輸送をサポートしています。LROSには、メラノソーム、血小板緻密体（デルタ顆粒とも呼ばれる）、II型肺細胞のラメラ小体、細胞傷害性T細胞およびサプレッサーT細胞、ナチュラルキラー（NK）細胞の顆粒タンパク質などが含まれます。

HPSの患者では、血小板凝集能の低下に伴う出血傾向がみられます。本症候群患者の血小板は、コラーゲン、トロンビン、エピネフリン、アデノシン二リン酸（ADP）による凝集に異常があります。

さらに、ヘルマンスキー・パドラック症候群市場は、広大な消費者基盤、出血問題や利用しやすい治療法に対する意識の高まり、有利な補償政策などの様々な要因によって牽引されており、Ferring Pharmaceuticals、Dr. Reddy's、Sagent Pharma、Ubi Pharmなどの主要企業が業界を牽引しています。

ダイナミクス

希少疾患の有病率の増加

希少疾患の有病率の増加は、予測期間中の市場を後押しする重要な要因の一つです。希少疾患は医療分野全体に存在します。嚢胞性線維症のように名前が広く知られているものもあれば、猫目症候群のようにあまり知られていないものもあります。ほとんどのがん（数種類を除く）は希少です。神経・筋疾患、代謝疾患、染色体異常、皮膚疾患、骨・骨格疾患、心臓、血液、肺、腎臓、その他の臓器・器官に影響を及ぼすまれな疾患もあります。

例えば、国立衛生研究所（NIH）によると、アメリカ人の2,500万人から3,000万人が罹患している希少疾患は約7,000種類あります。これはアメリカ人の10人に1人に相当し、エレベーターに1人、バスに4人乗っていることになります。

ヘルマンスキー・パドラック症候群（HPS）の有病率の上昇

ヘルマンスキー・パドラック症候群の有病率の上昇は、予測期間中に市場が成長するための重要な要因の1つです。例えば、NCBIで発表された最新の論文によると、ヘルマンスキー・パドラック症候群（HPS）はまれであり、有病率は全世界で100万人に1～9人ですが、特定の集団ではより一般的です。一例として、プエルトリコ北西部では1,800人に1人の有病率であり、約22人に1人がこの変異の保因者です。プエルトリコのHPS1型罹患者のほとんどは、HPS1のエクソン15に同じ16塩基対の重複を有しています。

規制遵守率の低さが市場成長を阻害する

貧しい国々では、国家機関やその他の機関が特定の病気に対する認識を高めることに失敗しているため、検査の普及率が低いです。この問題は、早期診断と患者の適切な治療を妨げることにより、市場の成長を阻害します。HPS患者は、術前に肺の評価を受けるべきです。麻酔チームは、本症の一部として出血傾向や肺合併症の可能性を認識しておく必要があります。HPS患者は全身麻酔後に肺合併症を起こす可能性があります。さらに、この症候群の患者には、術後の慎重なモニタリングが必要です。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

  • 影響要因
    • 促進要因
      • 希少疾患の有病率の増加
      • ヘルマンスキー・パドラック症候群（HPS）の有病率の上昇
    • 抑制要因
      • 低い規制遵守が市場の成長を阻害する
    • 機会
      • 長期的な治療アプローチ
    • 影響分析

第5章 産業分析

  • ポーターのファイブフォース分析
  • サプライチェーン分析
  • 価格分析
  • 規制分析
  • ロシア・ウクライナ戦争の影響分析
  • DMIの見解

第6章 COVID-19分析

第7章 タイプ別

  • HPS1
  • AP3B1
  • HPS3
  • HPS4
  • HPS5
  • HPS6
  • その他

第8章 治療タイプ別

  • 酢酸デスモプレシン
  • 経口避妊薬
  • 正常血小板の輸血

第9章 投与経路別

  • 経口
  • 静脈内

第10章 流通経路別

  • 病院薬局
  • 小売薬局
  • オンライン薬局

第11章 地域別

  • 北米
    • 米国
    • カナダ
    • メキシコ
  • 欧州
    • ドイツ
    • 英国
    • フランス
    • イタリア
    • スペイン
    • その他欧州
  • 南米
    • ブラジル
    • アルゼンチン
    • その他南米
  • アジア太平洋
    • 中国
    • インド
    • 日本
    • オーストラリア
    • その他アジア太平洋
  • 中東・アフリカ

第12章 競合情勢

  • 競合シナリオ
  • 市況/シェア分析
  • M&A分析

第13章 企業プロファイル

  • Ferring Pharmaceuticals
    • 企業概要
    • 製品ポートフォリオと説明
    • 財務概要
    • 主な動向
  • Dr. Reddy's
  • Sagent Pharma
  • UbiPharm
  • Amring Pharmaceuticals
  • Avet Pharmaceuticals
  • McKesson
  • Hybio Pharmaceuticals
  • Midas Pharma
  • SG Pharma

第14章 付録

目次
Product Code: PH6786

Overview

Hermansky-Pudlak syndrome (HPS) is caused by homozygous or compound heterozygous mutations in 1 of 11 genes that encode components in one of four protein complexes: adapter protein 3 (AP-3) and biogenesis of lysosome-related organelles complex 1, 2, and 3 (BLOC-1, BLOC-2, and BLOC-3). All four complexes support intracellular biogenesis and trafficking of lysosome and lysosome-related organelles (LROS). LROS include melanosomes, platelet dense bodies (also called delta granules), lamellar bodies of type II pneumocytes, and granule proteins of cytotoxic and suppressor T cells and natural killer (NK) cells.

Patients with HPS have a bleeding tendency associated with poor platelet aggregation. Platelets in patients with the syndrome have abnormal aggregation with collagen, thrombin, epinephrine, and adenosine diphosphate (ADP).

Furthermore, the Hermansky pudlak syndrome market is driven by various factors the vast consumer base increased awareness of bleeding problems and accessible remedies, and advantageous compensation policies drive the industry have significant key players like Ferring Pharmaceuticals, Dr. Reddy's, Sagent Pharma, Ubi Pharm and others.

Dynamics

Increasing Prevalence of Rare Diseases

The rise in the prevalence of rare diseases is one of the key factors that help the market during the forecast period. Rare diseases are present across the medical spectrum. Some are widely recognized by name, such as cystic fibrosis, while others are less known, such as cat eye syndrome. Most cancers (all but a few types) are rare. There are rare neurological and neuromuscular diseases, metabolic diseases, chromosomal disorders, skin diseases, bone and skeletal disorders, and rare diseases affecting the heart, blood, lungs, kidneys, and other body organs and systems.

For instance, according to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans. This equates to 1 in 10 Americans, or one on every elevator and four on every bus.

Rise in the Prevalence of Hermansky Pudlak Syndrome(HPS)

The rise in the prevalence of Hermansky Pudlak syndrome is one of the key factors that help the market to grow during the forecast period. For instance, according to an updated article published at NCBI Hermansky-Pudlak syndrome (HPS) is rare, with a prevalence of 1 to 9 in 1,000,000 individuals worldwide but is more common in certain populations. As an example, the prevalence is 1 in 1800 individuals in the northwestern region of Puerto Rico, where approximately 1 in 22 individuals are carriers of the mutation. Most HPS type 1-affected individuals in Puerto Rico have the same founder 16-base-pair duplication in exon 15 of HPS1.

Low Regulatory Compliance Will Stifle Market Growth

The failure of national organizations and other institutions in poor countries to raise awareness about certain ailments resulted in low testing penetration. This issue stifles market growth by hindering early diagnosis and appropriate patient care. Patients with HPS should obtain a preoperative pulmonary evaluation. The anesthesia team should be aware of bleeding tendencies and potential pulmonary complications as part of the syndrome. Patients with HPS may have pulmonary complications following general anaesthesia. Further, patients with the syndrome need careful post-operative monitoring.

Segment Analysis

The global Hermansky Pudlak Syndrome market is segmented based on type, therapy type, route of administration, distribution channel and region.

The Desmopressin Segment From the Therapy Type Segment Accounted For Approximately 41.7% of Hermansky Pudlak Syndrome Share

The desmopressin segment from the therapy type segment accounted for 41.7% and it is expected to be dominated during the The effectiveness of desmopressin acetate in HPS would likely depend on the individual's specific bleeding symptoms and the extent of platelet dysfunction. In some cases, it might provide modest relief for certain bleeding issues.

Excessive bleeding in individuals with HPS may be treated with medications that promote blood coagulation such as desmopressin (DDAVP), which has been shown to be effective for some patients. Other agents include aminocaproic acid, tranexamic acid and recombinant factor seven. For more serious bleeding, platelet transfusions can also be used and if possible, tissue-matched platelets are recommended.

Geographical Penetration

North America Accounted for Approximately 38.4% of the Market Share

North America is estimated to hold about 38.4 % of the total market share throughout the forecast period, due to its cost-effectiveness and access to healthcare facilities, improved treatment options, Patient awareness of bleeding disorders, as well as private-sector and government Therapeutics initiatives, assisting in market development. Also due to the preliminary stage acceptance of proven medical technology, the market in the area is the rapid ongoing expansion of firms operating in the drug sector.

For instance, in April 2023, Biopharma company Ocean Biomedical, Inc. announced its commitment to developing viable treatment options for Hermansky-Pudlak Syndrome (HPS). The company disclosed its plans to target OCF-203, its pulmonary fibrosis treatment candidate, as a therapeutic for fatal pulmonary fibrotic conditions caused by HPS.

Ocean's anti-fibrosis platform aims to target significant unmet needs for conditions like Idiopathic Pulmonary Fibrosis (IPF) and Hermansky-Pudlak Syndrome, and it has the potential to expand further into other areas like scleroderma, alcoholic liver disease, NASH, and kidney failure.

Competitive Landscape

The major global players in the market include: Ferring Pharmaceuticals, Dr Reddy's, Sagent Pharma, Ubi Pharm, Amring Pharmaceuticals, Avet Pharmaceuticals, McKesson, Hybio Pharmaceuticals, Midas Pharma, SG Pharma and among others.

COVID-19 Impact Analysis

The overall impact of COVID-19 had a substantial impact on the global Hermansky Pudlak syndrome market, as this syndrome is a rare disease that requires specialized medical care and treatment options but COVID-19 has disrupted the overall healthcare systems in which people having Hermansky Pudlak syndrome treatment options became unaffordable which indeed impacted the overall market.

By Type

  • HPS1
  • AP3B1
  • HPS3
  • HPS4
  • HPS5
  • HPS6
  • Others

By Therapy Type

  • Desmopressin Acetate
  • Oral Contraceptives
  • Transfusions of Normal Blood Platelets

By Route of Administration

  • Oral
  • Intravenous

By Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

By Region

  • North America
    • U.S.
    • Canada
    • Mexico
  • Europe
    • Germany
    • UK
    • France
    • Italy
    • Russia
    • Rest of Europe
  • South America
    • Brazil
    • Argentina
    • Rest of South America
  • Asia-Pacific
    • China
    • India
    • Japan
    • Australia
    • Rest of Asia-Pacific
  • Middle East and Africa

Key Developments

  • In October 2022, Ferring Pharmaceuticals and Blackstone Life Sciences announced a restructured cooperation in nadofaragene firadonavec, an experimental new gene therapy in late-stage research for patients with high-grade, Bacillus Calmette-Guerin refractory, non-muscle invasive bladder cancer.
  • In August 2022, American Regent, Inc., a California-based maker of medicines for human and veterinary use with production facilities in New York and Ohio, announced the acquisition of HBT Labs, Inc. HBT Labs offers a pipeline of generic and 505(b)(2) pharmaceutical medicines that target a variety of therapeutic indications, including cancer and CNS diseases.

Why Purchase the Report?

  • To visualize the global Hermansky Pudlak Syndrome market segmentation based on type, therapy type, route of administration, distribution channel and region as well as understand key commercial assets and players.
  • Identify commercial opportunities by analyzing trends and co-development.
  • Excel data sheet with numerous data points of global Hermansky Pudlak Syndrome market level with all segments.
  • PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
  • Product mapping available as excel consisting of key products of all the major players.

The Global Hermansky Pudlak Syndrome Market report would provide approximately 61 tables, 58 figures and 186 Pages.

Target Audience 2023

  • Manufacturers/ Buyers
  • Industry Investors/Investment Bankers
  • Research Professionals
  • Emerging Companies

Table of Contents

1. Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

  • 3.1. Snippet by Type
  • 3.2. Snippet by Therapy Type
  • 3.3. Snippet by Route of Administration
  • 3.4. Snippet by Distribution Channel
  • 3.5. Snippet by Region

4. Dynamics

  • 4.1. Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Increasing Prevalence of Rare Diseases
      • 4.1.1.2. Rise in the prevalence of Hermansky Pudlak Syndrome(HPS)
    • 4.1.2. Restraints
      • 4.1.2.1. Low Regulatory Compliance Will Stifle Market Growth
    • 4.1.3. Opportunity
      • 4.1.3.1. Long-Term Treatment Approach
    • 4.1.4. Impact Analysis

5. Industry Analysis

  • 5.1. Porter's Five Force Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Pricing Analysis
  • 5.4. Regulatory Analysis
  • 5.5. Russia-Ukraine War Impact Analysis
  • 5.6. DMI Opinion

6. COVID-19 Analysis

  • 6.1. Analysis of COVID-19
    • 6.1.1. Scenario Before COVID
    • 6.1.2. Scenario During COVID
    • 6.1.3. Scenario Post COVID
  • 6.2. Pricing Dynamics Amid COVID-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During the Pandemic
  • 6.5. Manufacturers Strategic Initiatives
  • 6.6. Conclusion

7. By Type

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
    • 7.1.2. Market Attractiveness Index, By Type
  • 7.2. HPS1 *
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.3. AP3B1
  • 7.4. HPS3
  • 7.5. HPS4
  • 7.6. HPS5
  • 7.7. HPS6
  • 7.8. Others

8. By Therapy Type

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
    • 8.1.2. Market Attractiveness Index, By Therapy Type
  • 8.2. Desmopressin Acetate *
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.3. Oral Contraceptives
  • 8.4. Transfusions of Normal Blood Platelets

9. By Route of Administration

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 9.1.2. Market Attractiveness Index, By Route of Administration
  • 9.2. Oral *
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 9.3. Intravenous

10. By Distribution Channel

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 10.1.2. Market Attractiveness Index, By Distribution Channel
  • 10.2. Hospital Pharmacies *
    • 10.2.1. Introduction
    • 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 10.3. Retail Pharmacies
  • 10.4. Online Pharmacies

11. By Region

  • 11.1. Introduction
    • 11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
    • 11.1.2. Market Attractiveness Index, By Region
  • 11.2. North America
    • 11.2.1. Introduction
    • 11.2.2. Key Region-Specific Dynamics
    • 11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
    • 11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
    • 11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.2.7.1. U.S.
      • 11.2.7.2. Canada
      • 11.2.7.3. Mexico
  • 11.3. Europe
    • 11.3.1. Introduction
    • 11.3.2. Key Region-Specific Dynamics
    • 11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
    • 11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
    • 11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.3.7.1. Germany
      • 11.3.7.2. UK
      • 11.3.7.3. France
      • 11.3.7.4. Italy
      • 11.3.7.5. Spain
      • 11.3.7.6. Rest of Europe
  • 11.4. South America
    • 11.4.1. Introduction
    • 11.4.2. Key Region-Specific Dynamics
    • 11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
    • 11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
    • 11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.4.7.1. Brazil
      • 11.4.7.2. Argentina
      • 11.4.7.3. Rest of South America
  • 11.5. Asia-Pacific
    • 11.5.1. Introduction
    • 11.5.2. Key Region-Specific Dynamics
    • 11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
    • 11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
    • 11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.5.7.1. China
      • 11.5.7.2. India
      • 11.5.7.3. Japan
      • 11.5.7.4. Australia
      • 11.5.7.5. Rest of Asia-Pacific
  • 11.6. Middle East and Africa
    • 11.6.1. Introduction
    • 11.6.2. Key Region-Specific Dynamics
    • 11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
    • 11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Type
    • 11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel

12. Competitive Landscape

  • 12.1. Competitive Scenario
  • 12.2. Market Positioning/Share Analysis
  • 12.3. Mergers and Acquisitions Analysis

13. Company Profiles

  • 13.1. Ferring Pharmaceuticals *
    • 13.1.1. Company Overview
    • 13.1.2. Product Portfolio and Description
    • 13.1.3. Financial Overview
    • 13.1.4. Key Developments
  • 13.2. Dr. Reddy's
  • 13.3. Sagent Pharma
  • 13.4. UbiPharm
  • 13.5. Amring Pharmaceuticals
  • 13.6. Avet Pharmaceuticals
  • 13.7. McKesson
  • 13.8. Hybio Pharmaceuticals
  • 13.9. Midas Pharma
  • 13.10. SG Pharma

LIST NOT EXHAUSTIVE

14. Appendix

  • 14.1. About Us and Services
  • 14.2. Contact Us