united kingdom hypophosphatasia therapeutics market

United Kingdom Hypophosphatasia Therapeutics Market: Segmented by Type, Treatment, Route of Administration, Distribution Channels, and End Users Size, Share, Impact, Growth, Trends, and Forecasts (2020 2028)

  • Published Date: 2022-12-28
  • Report ID: 139901
  • Pages: 200
  • Format: prudent report format

Report Overview

The UK Hypophosphatasia (HPP) Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Hypophosphatasia (HPP) is also known as Rathbun disease is an ultra-rare metabolic genetic disorder characterized by the abnormal development of bones and teeth due to defective mineralization, a process by which bones and teeth use minerals such as calcium and phosphorus. It caused by mutations in the tissue nonspecific alkaline phosphatase (TNSALP) gene, also called the ALPL gene. Such mutations lead to low activity of this enzyme that should be breaking down a chemical called inorganic pyrophosphate that blocks mineralization, profound muscle weakness, seizures, respiratory failure, and premature death.

According to a report, the disease is more common in older children and adults, affecting 1 per 6,370 of the population. HPP affects males and females in equal numbers. Juvenile and adult-onset forms of HPP have much lower mortality rates than those appearing in infancy, where 100% of those diagnosed in the first six months of their lives die before they reach one year. The total cost per person per year of treatment is a whopping 366,912 (assuming an average weight of 19.3 kg and 100% adherence). It seems to be rare in individuals with black ancestry.



Market Growth Drivers

The HPP community is helping to bring programs that will bring new and treatment options to people with rare diseases. Currently, clinical trials are conducted, wherein a large number of organizations are actively participating in several research programs to develop novel therapies for infantile and juvenile-onset hypophosphatasia treatment.

Introduction of several novel therapies, breakthrough therapy designation, orphan drug designation for rare diseases, financial incentives and grants by the government, clinical

studies on the safety and efficacy of products are expected to be the biggest hit in the UK infantile and juvenile-onset hypophosphatasia market over the forecast period.

Market Restraints

Gaps in making hypophosphatasia (HPP) health priority, patients non-adherence to therapy regime, high therapy cost, a smaller number of approved therapies, failure in a clinical trial at the late phase are some of the factors restraining the infantile and juvenile-onset hypophosphatasia treatment market.


TABLE OF CONTENTS

1. UK Hypophosphatasia Therapeutics Market Overview........................
A. Market Size
2. Market Growth Drivers and Restraints.
Market Growth Drivers
A. High Demand of Disease-Specific Novel Treatments
B. Huge Financial Support to the Researchers for Developing Novel Intervention
C. Increase in Special Designation from the Regulatory Authorities
Market Restraints
A. High Cost of Treatment
B. Failure of Clinical Trials at the Late Phase
3. Major Clinical Forms of Hypophosphatasia
A. Perinatal Hypophosphatasia
B. Infantile Hypophosphatasia
C. Juvenile Hypophosphatasia
D. Adult Hypophosphatasia
E. Odonto-hypophosphatasia
4. Hypophosphatasia Therapeutics Market Segmentation.....................
A. By Type
I. Odonto-hypophosphatasia
II. Pseudo-hypophosphatasia
III. Others
B. By Treatment
I. Asfotase alfa
II. Non-steroidal Anti-inflammatory Drugs
III. Thiazide Diuretics
IV. Surgery
V. Others
C. By Route of Administration
I. Oral
II. Injectables
III. Others
D. By Distribution Channels
I. Retail Pharmacies
II. Hospital Pharmacies
III. Online Pharmacies
IV. Others
E. By End Users
I. Academic and Research Organisations
II. Diagnostic Centres
III. Hospitals and Clinics
5. Hypophosphatasia Major Drugs Market Share
A. Market Analysis, Insights and Forecast By Revenue Type
6. Competitive Landscape...
A. Major Players
B. Products in Pipeline
7. Key Company Profiles.
A. Alexion Company overview, Product & Services, Strategies & Financials
B. AM-Pharma Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth
A. New Trends and Development of Hypophosphatasia Therapeutics Market
B. Future Opportunities
10. Conclusion
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