세계의 헤르만스키-푸들라크 증후군 시장(2023-2030년)

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