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全球赫曼斯基普德拉克綜合症市場 - 2023-2030
市場調查報告書
商品編碼
1345435

全球赫曼斯基普德拉克綜合症市場 - 2023-2030

Global Hermansky Pudlak Syndrome Market - 2023-2030
出版日期: | 出版商: DataM Intelligence | 英文 186 Pages | 商品交期: 約2個工作天內

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簡介目錄

概述

全球赫曼斯基普德拉克綜合症市場於 2022 年達到 XX 億美元，預計到 2030 年將達到 XX 億美元，在 2023-2030 年預測期間年複合成長率為 XX%。

Hermansky-Pudlak 症候群(HPS) 是由11 個基因中的1 個基因的純合或複合雜合突變引起，這些基因編碼四種蛋白質複合物之一的成分：銜接蛋白3 (AP-3) 和溶酶體相關細胞器複合物的生物發生1、2、和 3（BLOC-1、BLOC-2 和 BLOC-3）。所有四種複合物都支持細胞內生物發生以及溶酶體和溶酶體相關細胞器 (LROS) 的運輸。 LROS 包括黑素體、血小板緻密體（也稱為 delta 顆粒）、II 型肺細胞的層狀體以及細胞毒性和抑制性 T 細胞以及自然殺傷 (NK) 細胞的顆粒蛋白。

HPS 患者有與血小板聚集不良相關的出血傾向。該症候群患者的血小板中膠原蛋白、凝血酶、腎上腺素和二磷酸腺苷 (ADP) 異常聚集。

此外，赫曼斯基普德拉克綜合症市場受到多種因素的推動，廣大消費者基礎提高了對出血問題和可及治療的認知，有利的補償政策推動該行業擁有重要的關鍵參與者，如Ferring Pharmaceuticals、Dr. Reddy's、Sagent Pharma、Ubi Pharm 和其他。

動力學

罕見病患病率增加

罕見疾病患病率的上升是預測期內幫助市場的關鍵因素之一。罕見疾病存在於整個醫學領域。有些疾病的名稱已廣為人知，例如囊性纖維化，而另一些疾病則鮮為人知，例如貓眼綜合症。大多數癌症（除少數類型外的所有癌症）都很罕見。有罕見的神經和神經肌肉疾病、代謝疾病、染色體疾病、皮膚病、骨骼和骨骼疾病，以及影響心臟、血液、肺、腎臟和其他身體器官和系統的罕見疾病。

例如，根據美國國立衛生研究院 (NIH) 的數據，大約有 7,000 種罕見疾病影響著 25 至 3000 萬美國人。這相當於十分之一的美國人，或者每部電梯上有一個人，每輛公共汽車上有四個人。

赫曼斯基普德拉克綜合症（HPS）患病率上升

赫曼斯基普德拉克綜合症患病率的上升是預測期內幫助市場成長的關鍵因素之一。例如，根據NCBI 發表的一篇最新文章，赫曼斯基-普德拉克症候群(HPS) 很罕見，全世界每1,000,000 個人中有1 至9 人患有這種疾病，但在某些人群中更為常見。例如，在波多黎各西北部地區，患病率為每 1800 人中就有 1 人，其中大約每 22 人中就有 1 人是該突變的攜帶者。波多黎各大多數受 HPS 1 型影響的個體在 HPS1 的外顯子 15 中具有相同的 16 鹼基對重複。

低監管合規性將抑制市場成長

貧窮國家的國家組織和其他機構未能提高人們對某些疾病的認知，導致檢測普及率較低。這個問題阻礙了早期診斷和適當的患者護理，從而抑制了市場成長。 HPS 患者應進行術前肺部評估。麻醉團隊應該意識到該症候群的出血傾向和潛在的肺部併發症。 HPS 患者在全身麻醉後可能會出現肺部併發症。此外，患有該症候群的患者需要仔細的術後監測。

目錄

第 1 章：方法和範圍

  • 研究方法論
  • 報告的研究目的和範圍

第 2 章：定義和概述

第 3 章：執行摘要

  • 按類型分類
  • 按治療類型分類
  • 按給藥途徑分類
  • 按配銷通路分類
  • 按地區分類

第 4 章：動力學

  • 影響因素
    • 動力
      • 罕見病患病率增加
      • 赫曼斯基普德拉克綜合症（HPS）患病率上升
    • 限制
      • 低監管合規性將抑制市場成長
    • 機會
      • 長期治療方法
    • 影響分析

第 5 章：行業分析

  • 波特五力分析
  • 供應鏈分析
  • 定價分析
  • 監管分析
  • 俄烏戰爭影響分析
  • DMI 意見

第 6 章：COVID-19 分析

  • COVID-19 分析
    • 新冠疫情爆發前的情景
    • 新冠疫情期間的情景
    • 新冠疫情后的情景
  • COVID-19 期間的定價動態
  • 供需譜
  • 疫情期間政府與市場相關的舉措
  • 製造商戰略舉措
  • 結論

第 7 章：按類型

  • 高壓鈉燈1
  • AP3B1
  • 高壓鈉燈3
  • 高壓鈉燈4
  • 高壓鈉燈5
  • 高壓鈉燈6
  • 其他

第 8 章：按治療類型

  • 醋酸去氨加壓素
  • 口服避孕藥
  • 正常血小板的輸注

第 9 章：按給藥途徑分類

  • 口服
  • 靜脈

第 10 章：按配銷通路

  • 醫院藥房
  • 零售藥店
  • 網上藥店

第 11 章：按地區

  • 北美
    • 美國
    • 加拿大
    • 墨西哥
  • 歐洲
    • 德國
    • 英國
    • 法國
    • 義大利
    • 西班牙
    • 歐洲其他地區
  • 南美洲
    • 巴西
    • 阿根廷
    • 南美洲其他地區
  • 亞太
    • 中國
    • 印度
    • 日本
    • 澳大利亞
    • 亞太其他地區
  • 中東和非洲

第 12 章：競爭格局

  • 競爭場景
  • 市場定位/佔有率分析
  • 併購分析

第 13 章：公司簡介

  • Ferring Pharmaceuticals
    • 公司簡介
    • 產品組合和描述
    • 財務概覽
    • 主要進展
  • Dr. Reddy's
  • Sagent Pharma
  • UbiPharm
  • Amring Pharmaceuticals
  • Avet Pharmaceuticals
  • McKesson
  • Hybio Pharmaceuticals
  • Midas Pharma
  • SG Pharma

第 14 章：附錄

簡介目錄
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