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The global primary hypophosphatasia treatment market is expected to grow at a CAGR of 5.10% during the forecast period of 2023-2031. The market is driven due to the growing awareness of the rare disease, a strong product pipeline, and increased financial support for researchers developing new innovative drugs for hypophosphatasia.
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Hypophosphatasia (HPP) is a rare genetic disorder characterised by impaired bone and tooth mineralization (calcification). Mineralization is the process by which bones and teeth absorb the calcium and phosphorus required for proper hardness and strength.
The six major clinical forms are distinguished primarily by the age at which symptoms appear and a diagnosis is made like perinatal HPP, infantile HPP, childhood HPP, adult HPP, odontohypophosphatasia, and pseudohypophosphatasia. These forms are referred to by their decreasing severity.
Mutations in the ALPL gene can cause HPP. Genes encode instructions for the production of proteins that serve vital functions in the body. When a protein is mutated, it can become faulty, inefficient, or even absent, as in HPP. Depending on the function of the protein, one or more organ systems of the body may be jeopardised.
HPP is diagnosed by identifying its symptoms and complications, which begins with a thorough patient history. A thorough clinical examination reveals HPP signs, which are supported by routine x-rays and a variety of laboratory tests, including biochemical studies. For those who are familiar or experienced with this disorder, HPP is frequently easy to identify.
Most physicians, understandably, have little or no knowledge of HPP which leads to a frustrating delay in diagnosis. Commercial laboratories now offer genetic mutation analysis of the ALPL gene to support a diagnosis of HPP.
According to the market research report, the hypophosphatasia treatment market can be categorised into the following segments:
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Market Breakup by Type
Market Breakup by Diagnosis Method
Market Breakup by Treatment Method
Market Breakup by Treatment Channel
Market Breakup by Region
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HPP affects both men and women equally. Severe HPP is estimated to affect one in every 100,000 live births in Canada. The overall incidence and prevalence of the various forms of HPP are unknown or poorly understood. Milder cases may go undetected or be misdiagnosed. HPP is most common in the Mennonite population of Canada, is relatively common in Japan, and appears to be rare in people of Black ancestry.
Asfotase alfa (Strensiq) was approved by the US Food and Drug Administration (FDA) as the first medical treatment for perinatal, infantile, and juvenile-onset HPP. In the United States, patients of any age with pediatric-onset HPP are eligible for this bone-targeted tissue-nonspecific alkaline phosphatase (TNSALP) replacement therapy administered subcutaneously.
The growing number of hypophosphatasia patients, a family history of hypophosphatasia, a strong product pipeline, and increased financial support for researchers developing new innovative drugs for hypophosphatasia treatment are expected to drive the hypophosphatasia market during the forecast period. Furthermore, an increase in special designation from regulatory authorities to provide treatment to patients as soon as possible is boosting the market.
Teriparatide, a type of parathyroid hormone, has been given "off-label" to several adults with HPP complicated by metatarsal stress fractures or femoral pseudo fractures, resulting in fracture healing. Children are not permitted to use the drug. More research is required to determine teriparatide's long-term safety and effectiveness in the treatment of HPP. To treat severe HPP, bone marrow cell transplantation has been used.
Preliminary short-term results from the use of an anti-sclerostin antibody for HPP have also been reported. Sclerostin is a protein found in osteocytes, which are cells embedded in bone. Sclerostin aids in the reduction (downregulation) of osteoblasts. Sclerostin-specific antibodies have been shown to increase bone mass in osteoporosis patients.
The report gives an in-depth analysis of the key players involved in the global hypophosphatasia treatment market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows:
REPORT FEATURES | DETAILS |
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Base Year | 2022 |
Historical Period | 2016-2022 |
Forecast Period | 2023-2031 |
Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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Breakup by Type |
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Breakup by Diagnosis Method |
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Breakup by Treatment Method |
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Breakup by Treatment Channel |
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Breakup by Region |
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Market Dynamics |
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Supplier Landscape |
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Companies Covered |
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*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
2 Research Methodology
3 Executive Summary
4 Hypophosphatasia Overview
4.1 Guidelines and Stages
4.2 Pathophysiology
4.3 Screening and Diagnosis
4.4 Treatment Pathway
5 Patient Profile
5.1 Patient Profile Model
5.2 Patient Psychology and Emotional Impact Factors
5.3 Risk Assessment and Treatment Success Rate
6 Current Scenario Evaluation
6.1 Emerging Therapies and Clinical Trials Synopsis
6.2 Patent Landscape and Impact
6.3 Cost of Treatment
6.4 Others
7 Challenges and Unmet Needs
7.1 Treatment Pathway Challenges
7.2 Compliance and Drop- out Analysis
7.3 Awareness and Prevention Gaps
8 Global Hypophosphatasia Treatment Market
8.1 Global Hypophosphatasia Treatment Market Overview
8.2 Global Hypophosphatasia Treatment Market Analysis
8.2.1 Market Overview
8.2.1.1 Global Historical Hypophosphatasia Treatment Market Value (2016-2022)
8.2.1.2 Global Forecast Hypophosphatasia Treatment Market Value (2023-2031)
8.2.2 Global Hypophosphatasia Treatment Market by Type
8.2.2.1 Market Overview
8.2.2.1.1 Perinatal
8.2.2.1.2 Infantile
8.2.2.1.3 Childhood
8.2.2.1.4 Adult
8.2.2.1.5 Odonto-hypophosphatasia
8.2.2.1.6 Pseudo-hypophosphatasia
8.2.3 Global Hypophosphatasia Treatment Market by Diagnosis Method
8.2.3.1 Market Overview
8.2.3.1.1 X-rays
8.2.3.1.2 Biochemical Tests
8.2.3.1.3 Chorionic Villus Sampling (CVS)
8.2.3.1.4 Ultrasounds
8.2.3.1.5 Vitamin B6 Test
8.2.4 Global Hypophosphatasia Treatment Market by Treatment Method
8.2.4.1 Market Overview
8.2.4.1.1 Medications
8.2.4.1.1.1 Nonsteroidal anti-inflammatory drugs (NSAIDs)
8.2.4.1.1.2 Vitamin B6
8.2.4.1.1.3 Others
8.2.4.1.2 Dental Care
8.2.4.1.3 Physical Therapy
8.2.4.1.4 Occupational Therapy
8.2.5 Global Hypophosphatasia Treatment Market by Treatment Channel
8.2.5.1 Market Overview
8.2.5.1.1 Public
8.2.5.1.2 Private
8.2.6 Global Hypophosphatasia Treatment Market by Region
8.2.6.1 Market Overview
8.2.6.1.1 North America
8.2.6.1.2 Europe
8.2.6.1.3 Asia Pacific
8.2.6.1.4 Latin America
8.2.6.1.5 Middle East and Africa
9 North America Hypophosphatasia Treatment Market
9.1 Market Share by Country
9.2 United States of America
9.2.1 Historical Trend (2016-2022)
9.2.2 Forecast Trend (2023-2031)
9.3 Canada
9.3.1 Historical Trend (2016-2022)
9.3.2 Forecast Trend (2023-2031)
10 Europe Hypophosphatasia Treatment Market
10.1 Market Share by Country
10.2 United Kingdom
10.2.1 Historical Trend (2016-2022)
10.2.2 Forecast Trend (2023-2031)
10.3 Germany
10.3.1 Historical Trend (2016-2022)
10.3.2 Forecast Trend (2023-2031)
10.4 France
10.4.1 Historical Trend (2016-2022)
10.4.2 Forecast Trend (2023-2031)
10.5 Italy
10.5.1 Historical Trend (2016-2022)
10.5.2 Forecast Trend (2023-2031)
10.6 Others
11 Asia Pacific Hypophosphatasia Treatment Market
11.1 Market Share by Country
11.2 China
11.2.1 Historical Trend (2016-2022)
11.2.2 Forecast Trend (2023-2031)
11.3 Japan
11.3.1 Historical Trend (2016-2022)
11.3.2 Forecast Trend (2023-2031)
11.4 India
11.4.1 Historical Trend (2016-2022)
11.4.2 Forecast Trend (2023-2031)
11.5 ASEAN
11.5.1 Historical Trend (2016-2022)
11.5.2 Forecast Trend (2023-2031)
11.6 Australia
11.6.1 Historical Trend (2016-2022)
11.6.2 Forecast Trend (2023-2031)
11.7 Others
12 Latin America Hypophosphatasia Treatment Market
12.1 Market Share by Country
12.2 Brazil
12.2.1 Historical Trend (2016-2022)
12.2.2 Forecast Trend (2023-2031)
12.3 Argentina
12.3.1 Historical Trend (2016-2022)
12.3.2 Forecast Trend (2023-2031)
12.4 Mexico
12.4.1 Historical Trend (2016-2022)
12.4.2 Forecast Trend (2023-2031)
12.5 Others
13 Middle East and Africa Hypophosphatasia Treatment Market
13.1 Market Share by Country
13.2 Saudi Arabia
13.2.1 Historical Trend (2016-2022)
13.2.2 Forecast Trend (2023-2031)
13.3 United Arab Emirates
13.3.1 Historical Trend (2016-2022)
13.3.2 Forecast Trend (2023-2031)
13.4 Nigeria
13.4.1 Historical Trend (2016-2022)
13.4.2 Forecast Trend (2023-2031)
13.5 South Africa
13.5.1 Historical Trend (2016-2022)
13.5.2 Forecast Trend (2023-2031)
13.6 Others
14 Global Hypophosphatasia Treatment Market Dynamics
14.1 Market Drivers and Constraints
14.2 SWOT Analysis
14.3 Porter’s Five Forces Model
14.4 Key Demand Indicators
14.5 Key Price Indicators
14.6 Industry Events, Initiatives, and Trends
14.7 Value Chain Analysis
15 Supplier Landscape
15.1 Kirin Holdings Company
15.1.1 Company Overview
15.1.2 Product Portfolio
15.1.3 Demographic Reach and Achievements
15.1.4 Mergers and Acquisitions
15.1.5 Certifications
15.2 Vericel Corporation
15.2.1 Company Overview
15.2.2 Product Portfolio
15.2.3 Demographic Reach and Achievements
15.2.4 Mergers and Acquisitions
15.2.5 Certifications
15.3 Mereo BioPharma Group plc
15.3.1 Company Overview
15.3.2 Product Portfolio
15.3.3 Demographic Reach and Achievements
15.3.4 Mergers and Acquisitions
15.3.5 Certifications
15.4 Novartis AG
15.4.1 Company Overview
15.4.2 Product Portfolio
15.4.3 Demographic Reach and Achievements
15.4.4 Mergers and Acquisitions
15.4.5 Certifications
15.5 Bayer AG
15.5.1 Company Overview
15.5.2 Product Portfolio
15.5.3 Demographic Reach and Achievements
15.5.4 Mergers and Acquisitions
15.5.5 Certifications
15.6 AM-Pharma B.V.
15.6.1 Company Overview
15.6.2 Product Portfolio
15.6.3 Demographic Reach and Achievements
15.6.4 Mergers and Acquisitions
15.6.5 Certifications
15.7 Alexion
15.7.1 Company Overview
15.7.2 Product Portfolio
15.7.3 Demographic Reach and Achievements
15.7.4 Mergers and Acquisitions
15.7.5 Certifications
15.8 Pfizer, Inc.
15.8.1 Company Overview
15.8.2 Product Portfolio
15.8.3 Demographic Reach and Achievements
15.8.4 Mergers and Acquisitions
15.8.5 Certifications
16 Hypophosphatasia Treatment Market- Distribution Model (Additional Insight)
16.1 Overview
16.2 Potential Distributors
16.3 Key Parameters for Distribution Partner Assessment
17 Payment Methods (Additional Insight)
17.1 Government Funded
17.2 Private Insurance
17.3 Out-of-Pocket
*Additional insights provided are customisable as per client requirements.
qThe market is likely to grow at a CAGR of 5.10% during the forecast period of 2023-2031.
The market growth is driven by the growing awareness of the rare disease, a strong product pipeline, and increased financial support for researchers developing new innovative drugs for hypophosphatasia.
qNorth America is the dominating region in the global market owing to the rising demand for the treatment with the increased awareness about the disorder.
Kirin Holdings Company, Vericel Corporation, Mereo BioPharma Group plc, Novartis AG, Bayer AG, AM-Pharma B.V., and Alexion, Pfizer, Inc., among others are the leading companies in the market.
Lack of awareness of the treatment and a rise in the absence of information about the treatments will restrain the growth of hypophosphatasia treatment market.
Within the first year of life, mortality rates among patients with perinatal or infantile HPP ranged from 58 to 100 percent.
Adults with HPP may be diagnosed when routine laboratory testing reveals low serum ALP levels or when being evaluated for osteoporosis or fractures.
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