“The Hypophosphatasia market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period (2023-2032). Furthermore, launching various multiple- Hypophosphatasia pipeline products will significantly revolutionize the Hypophosphatasia market dynamics”
The Hypophosphatasia market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Hypophosphatasia market size from 2019 to 2032. The report also covers current Hypophosphatasia treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.
Key Takeaways from the Hypophosphatasia Market Report
- According to DelveInsight’s estimates, among the 7MM, the US is predicted to have the largest market for Hypophosphatasia, with approximately USD 619.2 million in 2021, followed by Japan and Germany.
- Although the advance-stage pipeline for Hypophosphatasia is ineffective, many therapies exist in the initial stages of development. ENPP1 inhibitors, next-generation ERTs, regenerative cell therapies, and gene therapies are anticipated to change the treatment paradigm for Hypophosphatasia in the distant future, addressing the unmet need for curative therapy.
- The leading Hypophosphatasia Companies include PuREC, AstraZeneca, Aruvant Sciences, AM-Pharma, Rallybio, Exscientia, and Others
- Promising Hypophosphatasia Pipeline Therapies include ALXN1850, asfotase alfa, REC-01, ALXN1850, ARU-2801, and others.
- ALXN1850 (Alexion Pharmaceuticals) is an investigational, next-generation asfotase alfa asset. Currently, it is being investigated in Phase I clinical development for the treatment of hypophosphatasia to assess safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and immunogenicity of ALXN1850 when given intravenous (IV) and subcutaneous (SC) to adults with HPP by dividing the participants into three experimental cohorts, each of which will be administered with 3 dosages (low, medium, and high) of ALXN1850, respectively, via IV infusion and/or SC over multiple administration intervals.
- REC-MSC engraftment (PuREC) are rapidly expanding cells that enables the synthesis of 10^12 ordered pure cells within 12 weeks without requiring complicated methodologies such as gene-modification. It exhibits improved self-renewal and multilineage differentiation, highly relevant to cell therapy that can be used for many types of applications including cell therapy field for orphan diseases. In 2021, the company planned to file IND for hypophosphatasia. Additionally, it is prepared for the first in-human trial.
Discover more about therapies set to grab major Hypophosphatasia Market Share @ Hypophosphatasia Market Size
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.
Hypophosphatasia Epidemiology Segmentation in the 7MM
- Hypophosphatasia Diagnosed Prevalent cases
- Hypophosphatasia Severity-specific Diagnosed Prevalent cases
Download the report to understand which factors are driving Hypophosphatasia Epidemiology Trends @ Hypophosphatasia Epidemiological Insights
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.
To know more about Hypophosphatasia Treatment options, visit @ Hypophosphatasia Drugs
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.
Hypophosphatasia Market Landscape
The Hypophosphatasia Market Value is forecasted to steadily during the study period, 2019-2032. The increase in disease burden prevalence in almost all 7MM, rise in hypophosphatasia awareness programs (both from public governments and patient associations), a robust pipeline of novel therapeutic options (pharmacological and non-pharmacological) are the major growth drivers of HPP Market. However, huge investments in R&D and complicated clinical trials to develop novel drugs and medical devices increases the ROI risk involved, which hampers the HPP market. Growing awareness, increased genetic screening, need for novel treatment options are glittering opportunities in HPP market.
Learn more about the Hypophosphatasia Pipeline Therapies in clinical trials @ Hypophosphatasia Market Landscape
Scope of the Hypophosphatasia Market Report
- Coverage- 7MM
- Study Period- 2019-2032
- Hypophosphatasia Companies- PuREC, AstraZeneca (NYSE: AZN), Aruvant Sciences, AM-Pharma, Rallybio (NYSE: RLYB), Exscientia (NYSE: EXAI), and Others
- Hypophosphatasia Pipeline Therapies- ALXN1850, asfotase alfa, REC-01, ALXN1850, ARU-2801, and others.
- Hypophosphatasia Market Dynamics: Hypophosphatasia Market Drivers and Barriers
Discover more about Hypophosphatasia Drugs in development @ Hypophosphatasia Ongoing Clinical Trials Analysis
Table of Content
- Key Insights
- Hypophosphatasia Report Introduction
- Hypophosphatasia Market Overview at a Glance
- Methodology of Hypophosphatasia Epidemiology and Market
- Executive Summary of Hypophosphatasia
- Key Events
- Disease Background and Overview
- Hypophosphatasia Epidemiology and Patient Population
- Patient Journey
- Hypophosphatasia Marketed Drugs
- Hypophosphatasia Emerging Drugs
- Hypophosphatasia: Market Analysis
- Key Opinion Leaders’ Views
- Hypophosphatasia SWOT Analysis
- Hypophosphatasia Unmet Need
- Hypophosphatasia Market Access and Reimbursement
- Appendix
- DelveInsight Capabilities
- Disclaimer
- About DelveInsight
Get in touch with our Business executive @ Hypophosphatasia Market Drivers and Barriers
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