DelveInsight’s “Hypophosphatasia Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Hypophosphatasia market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Hypophosphatasia drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Hypophosphatasia treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Hypophosphatasia: An Overview
Hypophosphatasia is defined as a rare, genetic metabolic disorder characterized by impaired mineralization of bones and teeth, according to the National Organization for Rare Disorders (NORD). Defective mineralization results in bones that are soft and prone to fracture and deformity and it also leads to tooth loss. Depending on the specific form, it can be inherited in an autosomal recessive or dominant manner.
It is characterized by defective bone and dental mineralization, leading to skeletal abnormalities with complications resulting in significant morbidity and mortality. The phenotypes range from the complete absence of bone mineralization and fetal death, mainly due to respiratory problems associated with thoracic deformities and pulmonary hypoplasia, to spontaneous fractures, premature tooth loss, seizures, or even nephrocalcinosis.
Based on various manifestations and severity, it is subdivided from the most severe to the mildest forms as perinatal, infantile, childhood-onset, adult, odonto Hypophosphatasia, and benign perinatal Hypophosphatasia.
Until recently, the treatment was mostly symptomatic and supportive, depending upon clinical manifestation. However, almost a demi decade ago, the first line of therapy for Hypophosphatasia, STRENSIQ (asfotase alfa), developed by AstraZeneca (Alexion AstraZeneca Rare Disease), was approved by the US FDA (2015) which is an enzyme replacement therapy. Pharmacological therapies like NSAIDs, glucocorticoids, potassium binders, vitamin D and B6 supplementation, and others; nonpharmacological therapies like mechanical ventilation and surgical intervention are also recommended.
Hypophosphatasia Market Key Facts
The total diagnosed prevalent cases of Hypophosphatasia in the 7MM were found to be approximately 6,540 cases in 2022, which are expected to increase by 2032 at a CAGR of 0.6% during the study period (2019 ─2032).
As per DelveInsight analysis, in the US, mild, moderate, and severe cases of Hypophosphatasia were approximately 4,950, 1000, and 120, respectively, in 2022.
In EU4 and the UK, mild, moderate, and severe cases of Hypophosphatasia were approximately 270, 55, and 7, respectively, in 2022.
Japan accounted for approximately 140 diagnosed prevalent cases of Hypophosphatasia in 2022.
In EU4 and the UK, Germany accounted for the highest cases of the diagnosed prevalent Hypophosphatasia, followed by France and the UK.
The US accounted for nearly 93% of the total diagnosed prevalent cases of Hypophosphatasia in the 7MM in the year 2022.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Hypophosphatasia pipeline therapies. It also thoroughly assesses the Hypophosphatasia market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete details of the market trend for each marketed Hypophosphatasia drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Hypophosphatasia Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Hypophosphatasia epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Hypophosphatasia epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Hypophosphatasia Epidemiology, Segmented as –
Diagnosed Prevalent Cases of Hypophosphatasia in the 7MM [2019–2032]
Severity-specific Diagnosed Prevalent Cases of Hypophosphatasia in the 7MM [2019–2032]
Hypophosphatasia Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Hypophosphatasia market or expected to be launched during the study period. The analysis covers the Hypophosphatasia market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Hypophosphatasia drugs based on their sale and market share.
The report also covers the Hypophosphatasia pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Hypophosphatasia companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Hypophosphatasia Therapeutics Analysis
Until recently, the treatment for Hypophosphatasia was mostly symptomatic and supportive, depending upon clinical manifestation. NSAIDs or glucocorticoids were majorly recommended to treat bone and joint pain due to the deposition of calcium pyrophosphate or hydroxyapatite crystals. They are still recommended to improve bone, joint or persistent pain secondary to fractures. Vitamin B6 and vitamin D are recommended for seizure control and supplementation. Potassium and phosphate binders and ACE inhibitors are also recommended.
Additional supportive therapies include ventilatory support, physiotherapy, occupational therapy, chronic pain management, and fracture care. Some cases, like nonhealing fractures or craniosynostosis, require surgical intervention. However, with the approval, the treatment paradigm for Hypophosphatasia has changed, making ERT the mainstay treatment for at least all severe forms of the disease.
Hypophosphatasia Companies Actively Working in the Therapeutics Market Include
And Many More
Emerging and Marketed Hypophosphatasia Therapies Covered in the Report Include:
ENPP1 inhibitor: Rallybio/ Exscientia
STRENSIQ (asfotase alfa): AstraZeneca
And Many More
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Hypophosphatasia Competitive Intelligence Analysis
4. Hypophosphatasia Market Overview at a Glance
5. Hypophosphatasia Disease Background and Overview
6. Hypophosphatasia Patient Journey
7. Hypophosphatasia Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Hypophosphatasia Treatment Algorithm, Current Treatment, and Medical Practices
9. Hypophosphatasia Unmet Needs
10. Key Endpoints of Hypophosphatasia Treatment
11. Hypophosphatasia Marketed Therapies
12. Hypophosphatasia Emerging Drugs and Latest Therapeutic Advances
13. Hypophosphatasia Seven Major Market Analysis
14. Attribute Analysis
15. Hypophosphatasia Market Outlook (In US, EU5, and Japan)
16. Hypophosphatasia Companies Active in the Market
17. Hypophosphatasia Access and Reimbursement Overview
18. KOL Views on the Hypophosphatasia Market
19. Hypophosphatasia Market Drivers
20. Hypophosphatasia Market Barriers
22. DelveInsight Capabilities
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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