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全球赫曼斯基普德拉克综合症市场 - 2023-2030
市场调查报告书
商品编码
1345435

全球赫曼斯基普德拉克综合症市场 - 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: 约2个工作天内

价格

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简介目录

概述

全球赫曼斯基普德拉克综合症市场于 2022 年达到 XX 亿美元，预计到 2030 年将达到 XX 亿美元，在 2023-2030 年预测期间复合年增长率为 XX%。

Hermansky-Pudlak 综合征(HPS) 是由11 个基因中的1 个基因的纯合或复合杂合突变引起，这些基因编码四种蛋白质复合物之一的成分：衔接蛋白3 (AP-3) 和溶酶体相关细胞器复合物的生物发生1、2、和 3（BLOC-1、BLOC-2 和 BLOC-3）。所有四种复合物都支持细胞内生物发生以及溶酶体和溶酶体相关细胞器 (LROS) 的运输。 LROS 包括黑素体、血小板緻密体（也称为 delta 颗粒）、II 型肺细胞的层状体以及细胞毒性和抑制性 T 细胞以及自然杀伤 (NK) 细胞的颗粒蛋白。

HPS 患者有与血小板聚集不良相关的出血倾向。该综合征患者的血小板中胶原蛋白、凝血酶、肾上腺素和二磷酸腺苷 (ADP) 异常聚集。

此外，赫曼斯基普德拉克综合症市场受到多种因素的推动，广大消费者基础提高了对出血问题和可及治疗的认识，有利的补偿政策推动该行业拥有重要的关键参与者，如Ferring Pharmaceuticals、Dr. Reddy's、Sagent Pharma、Ubi Pharm 和其他的。

动力学

罕见病患病率增加

罕见疾病患病率的上升是预测期内帮助市场的关键因素之一。罕见疾病存在于整个医学领域。有些疾病的名称已广为人知，例如囊性纤维化，而另一些疾病则鲜为人知，例如猫眼综合症。大多数癌症（除少数类型外的所有癌症）都很罕见。有罕见的神经和神经肌肉疾病、代谢疾病、染色体疾病、皮肤病、骨骼和骨骼疾病，以及影响心臟、血液、肺、肾臟和其他身体器官和系统的罕见疾病。

例如，根据美国国立卫生研究院 (NIH) 的数据，大约有 7,000 种罕见疾病影响着 25 至 3000 万美国人。这相当于十分之一的美国人，或者每部电梯上有一个人，每辆公共汽车上有四个人。

赫曼斯基普德拉克综合症（HPS）患病率上升

赫曼斯基普德拉克综合症患病率的上升是预测期内帮助市场增长的关键因素之一。例如，根据NCBI 发表的一篇最新文章，赫曼斯基-普德拉克综合征(HPS) 很罕见，全世界每1,000,000 个人中有1 至9 人患有这种疾病，但在某些人群中更为常见。例如，在波多黎各西北部地区，患病率为每 1800 人中就有 1 人，其中大约每 22 人中就有 1 人是该突变的携带者。波多黎各大多数受 HPS 1 型影响的个体在 HPS1 的外显子 15 中具有相同的 16 碱基对重复。

低监管合规性将抑制市场增长

贫穷国家的国家组织和其他机构未能提高人们对某些疾病的认识，导致检测普及率较低。这个问题阻碍了早期诊断和适当的患者护理，从而抑制了市场增长。 HPS 患者应进行术前肺部评估。麻醉团队应该意识到该综合征的出血倾向和潜在的肺部并发症。 HPS 患者在全身麻醉后可能会出现肺部并发症。此外，患有该综合征的患者需要仔细的术后监测。

目录

第 1 章：方法和范围

  • 研究方法论
  • 报告的研究目的和范围

第 2 章：定义和概述

第 3 章：执行摘要

  • 按类型分類的片段
  • 按治疗类型分類的片段
  • 按给药途径分類的片段
  • 按分销渠道分類的片段
  • 按地区分類的片段

第 4 章：动力学

  • 影响因素
    • 司机
      • 罕见病患病率增加
      • 赫曼斯基普德拉克综合症（HPS）患病率上升
    • 限制
      • 低监管合规性将抑制市场增长
    • 机会
      • 长期治疗方法
    • 影响分析

第 5 章：行业分析

  • 波特五力分析
  • 供应链分析
  • 定价分析
  • 监管分析
  • 俄乌战争影响分析
  • DMI 意见

第 6 章：COVID-19 分析

  • COVID-19 分析
    • 新冠疫情爆发前的情景
    • 新冠疫情期间的情景
    • 新冠疫情后的情景
  • COVID-19 期间的定价动态
  • 供需谱
  • 疫情期间政府与市场相关的倡议
  • 製造商战略倡议
  • 结论

第 7 章：按类型

  • 高压钠灯1
  • AP3B1
  • 高压钠灯3
  • 高压钠灯4
  • 高压钠灯5
  • 高压钠灯6
  • 其他的

第 8 章：按治疗类型

  • 醋酸去氨加压素
  • 口服避孕药
  • 正常血小板的输注

第 9 章：按给药途径分类

  • 口服
  • 静脉

第 10 章：按分销渠道

  • 医院药房
  • 零售药店
  • 网上药店

第 11 章：按地区

  • 北美
    • 我们
    • 加拿大
    • 墨西哥
  • 欧洲
    • 德国
    • 英国
    • 法国
    • 意大利
    • 西班牙
    • 欧洲其他地区
  • 南美洲
    • 巴西
    • 阿根廷
    • 南美洲其他地区
  • 亚太
    • 中国
    • 印度
    • 日本
    • 澳大利亚
    • 亚太其他地区
  • 中东和非洲

第 12 章：竞争格局

  • 竞争场景
  • 市场定位/份额分析
  • 併购分析

第 13 章：公司简介

  • Ferring Pharmaceuticals
    • 公司简介
    • 产品组合和描述
    • 财务概览
    • 主要进展
  • Dr. Reddy's
  • Sagent Pharma
  • UbiPharm
  • Amring Pharmaceuticals
  • Avet Pharmaceuticals
  • McKesson
  • Hybio Pharmaceuticals
  • Midas Pharma
  • SG Pharma

第 14 章：附录

简介目录
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