Hypophosphatasia Market Size in the 7MM was approximately USD 677.8 Million in 2021

"The increase in market size is a direct consequence of increasing awareness and increasing diagnosed prevalent population of Hypophosphatasia in the 7MM"

The Hypophosphatasia market report provides current treatment practices, emerging drugs, market share of individual therapies, and the current and forecasted 7MM Hypophosphatasia market size from 2019 to 2032. The Report also covers current Hypophosphatasia treatment practice, SWOT analysis, reimbursement, market access, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Key takeaways from the Hypophosphatasia Market Research Report

• According to DelveInsight estimates, mild Hypophosphatasia was most prevalent in the US, followed by moderate and severe Hypophosphatasia.

• According to DelveInsight's estimates, there were approximately 6,497 total diagnosed prevalent cases of Hypophosphatasia in the seven major markets, in 2021. These cases are expected to increase by 2032 at a CAGR of 0.6% during the study period (2019-2032).

• According to DelveInsight's estimates, with approximately 6,030 cases, the US had the highest (92.8%) diagnosed prevalence of Hypophosphatasia in the 7MM in the year 2021.

• The leading Hypophosphatasia Market Companies includes PuREC, AstraZeneca, Aruvant Sciences, AM-Pharma, Rallybio, Exscientia, and others.

• Promising Hypophosphatasia Pipeline Therapies includes STRENSIQ (asfotase alfa), REC-01, ALXN1850, ARU-2801, recap, ENPP1 inhibitor, and others.

To discover which therapies are expected to grab the major Hypophosphatasia Market share, click here for Hypophosphatasia Market Research Report @ https://www.delveinsight.com/sample-request/hypophosphatasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Hypophosphatasia Overview

Hypophosphatasia is defined as a rare, inherited metabolic disorder characterized by poor mineralization of bones and teeth, according to National Organization for Rare Disorders (NORD). Defective mineralization causes fragile, fracture-prone, and deformed bones, which also result in tooth loss. It can be inherited autosomally recessively or dominantly, depending on the specific form. It is a multi-systemic metabolic disorder caused by loss-of-function mutations in the ALPL gene that encodes the mineralization-associated enzyme, tissue-nonspecific alkaline phosphatase (TNSALP). TNSALP regulates mitochondrial function and ATP levels. Defects in TNSALP are linked to mitochondrial dysfunction, cell respiration, and an increase in reactive oxygen species, which results in metabolic abnormalities in the group.

Hypophosphatasia Epidemiology Segmentation in the 7MM

• Hypophosphatasia Diagnosed Prevalent cases

• Hypophosphatasia Severity-specific Diagnosed Prevalent cases

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Hypophosphatasia Marketed Drugs

• STRENSIQ (asfotase alfa): AstraZeneca

STRENSIQ (asfotase alfa), developed by Alexion Pharma (now acquired by AstraZeneca), is a TNSALP indicated for the treatment of patients with perinatal, infantile- and juvenile-onset Hypophosphatasia.

It is a soluble glycoprotein composed of two identical polypeptide chains, which consists of the catalytic domain of human TNSALP, the human immunoglobulin G1 Fc domain, and a deca-aspartate peptide used as a bone targeting domain. It is an enzyme replacement therapy that replaces the TNSALP enzyme, the deficiency of which leads to elevations in several TNSALP substrates, including inorganic pyrophosphate (PPi). Elevated extracellular levels of PPi block hydroxyapatite crystal growth that inhibits bone mineralization and causes an accumulation of unmineralized bone matrix which manifests as rickets and bone deformation in infants and children and as osteomalacia. Replacement of the enzyme upon STRENSIQ treatment reduces the enzyme-substrate levels.

Hypophosphatasia Emerging Drugs

• REC-01: PuREC
Human bone marrow-derived mesenchymal stem cells (MSCs) developed by PuREC Co., Ltd., clarified the use of antibodies that recognize two types of cell surface markers, LNGFR (CD271) and Thy1 (CD90), enabled extremely efficient sorting of human MSCs. PuREC specializes in regenerative cell therapy using MSCs. Advancements in human bone marrow cell isolation have led to the extraction of an extremely pure and rapidly expanding MSC population. REC (rapidly expanding cells) exhibit improved self-renewal and multilineage differentiation, which is highly relevant to cell therapy. In parallel, an initial clinical study infusing MSCs in patients with severe Hypophosphatasia has shown improvement in bone mineralization and bone function. Currently, Phase I/II clinical study in Japan is being led by Shimane University with a Japanese government grant (AMED (Japan Agency for Medical Research and Development)) and key hospitals using PuREC's REC.

• ALXN1850: AstraZeneca
ALXN1850 developed by AstraZeneca is an ERT replacing deficient ALP activity and targets ALP substrates to improve bone mineralization and ameliorate systemic manifestations of the disease. It is a next-generation Hypophosphatasia therapy designed to provide higher activity, higher bioavailability, and longer half-life than STRENSIQ (asfotase alfa). These improvements may result in significant benefits for Hypophosphatasia patients, including potentially lower, less frequent doses, improved efficacy, and lower injection volumes compared to STRENSIQ. Recently ALXN1850 has completed its Phase I trial, and Alexion, AstraZeneca Rare Disease is planning to initiate a Phase III trial of ALXN1850 in 2023.

• ARU-2801: Aruvant Sciences
ARU-2801 is a one-time, adeno-associated virus (AAV) gene therapy designed to deliver potentially curative efficacy to patients with Hypophosphatasia without the limitations of chronic administration. Data from preclinical studies with ARU-2801 showed improved disease biomarkers and increased survival. Preclinical research showed that treatment with ARU-2801 resulted in sustained elevation of tissue nonspecific alkaline phosphatase (TNAP), the missing enzyme in Hypophosphatasia, at levels that ameliorate disease symptoms. Manufacturing process development and Investigational New Drug (IND) application-enabling studies are currently underway.

Hypophosphatasia Market Outlook

Hypophosphatasia is a rare, genetic, metabolic disorder caused by autosomal recessive mutations or a single dominant-negative mutation in the ALPL gene found on chromosome 1 encoding TNSALP. It is a heterogeneous disease with high variability in disease presentation and severity, ranging from perinatal-onset with mortality as high as 100% to adult-onset with little mortality but a high disease burden. Until recently, the treatment 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.

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Scope of the Hypophosphatasia Market Research Report

• Coverage- 7MM

• Hypophosphatasia Companies- PuREC, AstraZeneca, Aruvant Sciences, AM-Pharma, Rallybio, Exscientia, and others.

• Hypophosphatasia Pipeline Therapies- STRENSIQ (asfotase alfa), REC-01, ALXN1850, ARU-2801, recap, ENPP1 inhibitor, and others.

• Hypophosphatasia Market Dynamics: Hypophosphatasia Market Drivers and Barriers

• Hypophosphatasia Market Access and Reimbursement, Unmet Needs

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Table of Content
1. Key Insights
2. Report Introduction
3. Hypophosphatasia Market Overview at a Glance
4. Methodology of Hypophosphatasia Epidemiology and Market
5. Executive Summary of Hypophosphatasia
6. Key Events
7. Hypophosphatasia Disease Background and Overview
8. Hypophosphatasia Epidemiology and Patient Population
9. Hypophosphatasia Patient Journey
10. Hypophosphatasia Marketed Drugs
11. Hypophosphatasia Emerging Drugs
12. Hypophosphatasia: Market Analysis
13. Key Opinion Leaders' Views
14. SWOT Analysis
15. Hypophosphatasia Unmet Need
16. Hypophosphatasia Market Access and Reimbursement
17. Appendix
18. DelveInsight Capabilities
19. Disclaimer
20. About DelveInsight

To read more about the Hypophosphatasia Market Dynamics of the report, click here for Hypophosphatasia Market Barriers and Drivers @ https://www.delveinsight.com/sample-request/hypophosphatasia-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

About Us

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