UK Hypophosphatasia (HPP) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2573

  • Format: PPT, PDF

UK 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: June 2021

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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

Market Segmentation

By Type:

Based on type, the hypophosphatasia treatment market can be segmented into

  • Odonto-hypophosphatasia
  • Pseudo-hypophosphatasia
  • Others

The odonto-hypophosphatasia segment accounted for the highest share of the hypophosphatasia treatment market in 2020. This segment is expected to dominate the hypophosphatasia treatment market during the forecast period due to the high number of patients with odonto-hypophosphatasia, which is characterized by loss of premature teeth in childhood or loss of teeth in adulthood.

By Treatment:

On the basis of treatment, the hypophosphatasia treatment market can be divided into

  • Strensiq (asfotase alfa
  • Non-steroidal anti-inflammatory drugs (NSAIDs)
  • Thiazide diuretics
  • Surgery
  • Others

The medications generated the highest revenue in 2020 and are anticipated to witness growth during the forecast period. This can be attributed to the launch of products for the treatment of hypophosphatasia from major manufacturers. In the UK, the European Medicines Agency (EUA) also approved Strensiq (asfotase alfa) as the first approved medical treatment for infantile, perinatal, and juvenile-onset HPP.

By Route of Administration:

Based on the route of administration, the hypophosphatasia treatment of the market can be segmented into

  • Oral
  • Injectables
  • Others

The injectables witness huge market growth potential during the forecast period. As asfotase alfa and recombinant human alkaline phosphatase is given as an enzyme replacement therapy by the subcutaneous route of administration that targets the underlying cause of the disease.

By Distribution Channels:

In terms of distribution channels, the hypophosphatasia treatment market can be divided into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

The hospital pharmacies segment generated the highest revenue in 2020 and is anticipated to witness growth during the forecast period due to most of the major products are prescription products, which are available in hospital pharmacies.

By End Users:

As per the end-user, the hypophosphatasia treatment market has been segmented into

  • Academic and research organizations
  • Diagnostic centers
  • Hospitals and clinics

Currently, there are around 250+ hospital admissions for hypophosphatasia in the UK. Thus, the hospitals and clinics segment commands the largest market share in terms of value during the forecast period.

Top Major Players

The major key players operating in United Kingdom Hypophosphatasia (HPP) treatment markets are Alexion Pharmaceuticals Inc., and AM-Pharma BV primarily. There is currently no permanent solution for hypophosphatasia. Medical management is aimed at monitoring and alleviating symptoms and decreasing morbidity. Vitamin B6 may also be given to reduce seizures. On the other hand, asfotase alfa by Alexion Pharmaceuticals Inc. and recombinant human alkaline phosphatase by AM-Pharma BV considered as an effective enzyme replacement therapy to treat all forms of hypophosphatasia, prevailing in the UK market and expected to develop novel therapies for infantile and juvenile-onset hypophosphatasia treatment during the forecast period.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

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The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

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