X-linked hypophosphatemia is a rare inherited disorder that prevents the body from properly processing phosphate and is estimated to affect 1 in 20,000 individuals. This genetic bone disease is characterized by low phosphate levels in the blood, with bone pain, dental problems, and hearing loss as the common symptoms. The rising advancements in genetic and molecular research including a better understanding of the genetic mutations that cause X-linked hypophosphatemia are likely to stimulate the development of targeted therapies in the coming years.
Report Coverage
The X-linked Hypophosphatemia (XLH) Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into X-linked hypophosphatemia (XLH) drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for X-linked hypophosphatemia (XLH). The report includes the analysis of over 100 pipeline drugs and 50+ companies. The X-linked hypophosphatemia (XLH) pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from X-linked hypophosphatemia (XLH).
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to X-linked hypophosphatemia (XLH).
X-linked Hypophosphatemia (XLH) Drug Pipeline Outlook
X-linked hypophosphatemia (XLH) is considered the most common cause of inherited phosphate wasting. It is linked with serious complications including disproportionate short stature in children, rickets, hyperparathyroidism, poor mineralization of the teeth, and lower limb deformities. The characteristics and severity of this inherited disorder show significant variability between patients.
Phosphate supplements and vitamin D (calcitriol) can help in addressing the metabolic causes of the disease, reducing or even reversing the symptoms in children. Bursomab, an FDA-approved monoclonal antibody treatment for X-linked hypophosphatemia is also used for increasing the phosphate levels in the blood. With the growing focus on developing medications that target specific pathways involved in X-linked hypophosphatemia, the drug pipeline is anticipated to witness a steady expansion in the near future.
X-linked Hypophosphatemia (XLH) – Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of X-linked hypophosphatemia (XLH) drugs based on several segmentations including:
By Phase
- Late-Stage Products (Phase 3 and Phase 4)
- Mid-Stage Products (Phase 2)
- Early-Stage Products (Phase I)
- Preclinical and Discovery Stage Products
- Monoclonal Antibodies
- Enzyme Replacement Therapies
- Gene Therapies
- Small Molecules
- Oral
- Parenteral
- Others
The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis , phase I and II cover a major share of the total clinical trials for X-linked hypophosphatemia (XLH).
X-linked Hypophosphatemia (XLH) – Pipeline Assessment Segmentation, By Drug Classes
The drug molecule categories covered under the X-linked hypophosphatemia (XLH) pipeline analysis include monoclonal antibodies, enzyme replacement therapies, gene therapies, and small molecules. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for X-linked hypophosphatemia (XLH).
X-linked Hypophosphatemia (XLH) Clinical Trials Therapeutic Assessment – Competitive Dynamics
The EMR report for the X-linked hypophosphatemia (XLH) drug pipeline covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in X-linked hypophosphatemia (XLH) clinical trials:
- Kyowa Kirin, Inc.
- Ultragenyx Pharmaceutical Inc
- Inozyme Pharma
- Medialis Ltd.
- Zeria Pharmaceutical
Major drugs currently in the drug pipeline are as follows:
Drug: INZ-701
Sponsored by Inozyme Pharma, the objective of this open-label Phase II interventional study is to investigate the long-term safety of investigational drug candidate INZ-701 in an estimated 200 subjects with ENPP1 deficiency or ABCC6 deficiency. The study is expected to be completed by December 2030 and has the potential to treat autosomal recessive hypophosphatemic rickets type 2, a form of X-linked hypophosphatemia.
Drug: KK8123
This Phase 1/2 dose-escalation study (with an optional safety extension period) is aimed at evaluating the safety, tolerability, and efficacy of KK8123 in adults with X-linked hypophosphatemia. The interventional study has enrolled an estimated 24 subjects and is expected to be completed by October 2028.
Reasons To Buy This Report
The X-linked hypophosphatemia (XLH) Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for X-linked hypophosphatemia (XLH). It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within X-linked hypophosphatemia (XLH) pipeline insights.
Key Questions Answered in the X-linked Hypophosphatemia (XLH) – Pipeline Insight Report
- What is the current landscape of X-linked hypophosphatemia (XLH) pipeline drugs?
- How many companies are developing X-linked hypophosphatemia (XLH) drugs?
- How many phase III and phase IV drugs are currently present in X-linked hypophosphatemia (XLH) pipeline drugs?
- Which companies/institutions are leading the X-linked hypophosphatemia (XLH) drug development?
- What is the efficacy and safety profile of X-linked hypophosphatemia (XLH) pipeline drugs?
- What are the opportunities and challenges present in the X-linked hypophosphatemia (XLH) drug pipeline landscape?
- Which company is conducting major trials for X-linked hypophosphatemia (XLH) drugs?
- What geographies are covered for X-linked hypophosphatemia (XLH) clinical trials?
- What are emerging trends in X-linked hypophosphatemia (XLH) clinical trials?
Global Clinical Trials Market
X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline Insight Report
- 1 Preface
- 1.1 Introduction
- 1.2 Objectives of the Study
- 1.3 Research Methodology & Assumptions
- 2 Executive Summary
- 3 Overview of X-linked Hypophosphatemia (XLH)
- 3.1 Signs and Symptoms
- 3.2 Causes
- 3.3 Risk Factors
- 3.4 Types of X-linked Hypophosphatemia (XLH)
- 3.5 Diagnosis
- 3.6 Treatment
- 4 Patient Profile: X-linked Hypophosphatemia (XLH)
- 4.1 Patient Profile Overview
- 4.2 Patient Psychology and Emotional Impact Factors
- 4.3 Risk Assessment and Treatment Success Rate
- 5 X-linked Hypophosphatemia (XLH): Epidemiology Snapshot
- 5.1 X-linked Hypophosphatemia (XLH) Incidence by Key Markets
- 5.2 X-linked Hypophosphatemia (XLH) – Patients Seeking Treatment in Key Markets
- 6 X-linked Hypophosphatemia (XLH): Market Dynamics
- 6.1 Market Drivers and Constraints
- 6.2 SWOT Analysis
- 7 X-linked Hypophosphatemia (XLH): Key Facts Covered
- 7.1 Top Countries Contributing to Clinical Trials in Asia-Pacific
- 7.2 Top Countries Contributing to Clinical Trials in Europe
- 7.3 Top Countries Contributing to Clinical Trials in North America
- 7.4 Top Countries Contributing to Clinical Trials in Other Regions
- 8 X-linked Hypophosphatemia (XLH), Drug Pipeline Assessment
- 8.1 Assessment by Treatment Type
- 8.2 Assessment by Route of Administration
- 8.3 Assessment by Drug Class
- 9 EMR Drug Pipeline Comparative Analysis
- 9.1 List of X-linked Hypophosphatemia (XLH) Pipeline Drugs
- 9.1.1 By Company
- 9.1.2 By Phase
- 9.1.3 By Indication
- 9.1.4 By Trial Status
- 9.1.5 By Funder Type
- 9.2 EMR Attribute Scoring Analysis of Pipeline Drugs (Top Drugs)
- 10 X-linked Hypophosphatemia (XLH) Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
- 10.1 Comparative Analysis for Late-Stage Drugs
- 10.1.1 Study Type
- 10.1.2 Recruitment Status
- 10.1.3 Company
- 10.1.4 Funder Type
- 10.2 Product Level Analysis*
- 10.2.1 Drug 1
- 10.2.1.1 Product Description
- 10.2.1.2 Trial ID
- 10.2.1.3 Sponsor Name
- 10.2.1.4 Study Type
- 10.2.1.5 Drug Class
- 10.2.1.6 Eligibility Criteria
- 10.2.1.7 Study Record Dates
- 10.2.1.7.1 First Submitted
- 10.2.1.7.2 First Posted
- 10.2.1.7.3 Last Update Posted
- 10.2.1.7.4 Last Verified
- 10.2.1.8 Indication
- 10.2.1.9 Study Design
- 10.2.1.10 Recruitment Status
- 10.2.1.11 Enrollment (Estimated)
- 10.2.1.12 Location Countries
- 10.2.1.13 Recent Results
- 10.2.2 Other Drugs
- 11 X-linked Hypophosphatemia (XLH) Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
- 11.1 Comparative Analysis for Mid-Stage Drugs
- 11.1.1 Study Type
- 11.1.2 Recruitment Status
- 11.1.3 Company
- 11.1.4 Funder Type
- 11.2 Product Level Analysis*
- 11.2.1 Drug: INZ-701
- 11.2.1.1 Product Description
- 11.2.1.2 Trial ID
- 11.2.1.3 Sponsor Name
- 11.2.1.4 Study Type
- 11.2.1.5 Drug Class
- 11.2.1.6 Eligibility Criteria
- 11.2.1.7 Study Record Dates
- 11.2.1.7.1 First Submitted
- 11.2.1.7.2 First Posted
- 11.2.1.7.3 Last Update Posted
- 11.2.1.7.4 Last Verified
- 11.2.1.8 Indication
- 11.2.1.9 Study Design
- 11.2.1.10 Recruitment Status
- 11.2.1.11 Enrollment (Estimated)
- 11.2.1.12 Location Countries
- 11.2.1.13 Recent Results
- 11.2.2 Drug: KK8123
- 11.2.3 Other Drugs
- 12 X-linked Hypophosphatemia (XLH) Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
- 12.1 Comparative Analysis for Early-Stage Drugs
- 12.1.1 Study Type
- 12.1.2 Recruitment Status
- 12.1.3 Company
- 12.1.4 Funder Type
- 12.2 Product Level Analysis*
- 12.2.1 Drug: Calcitriol
- 12.2.1.1 Product Description
- 12.2.1.2 Trial ID
- 12.2.1.3 Sponsor Name
- 12.2.1.4 Study Type
- 12.2.1.5 Drug Class
- 12.2.1.6 Eligibility Criteria
- 12.2.1.7 Study Record Dates
- 12.2.1.7.1 First Submitted
- 12.2.1.7.2 First Posted
- 12.2.1.7.3 Last Update Posted
- 12.2.1.7.4 Last Verified
- 12.2.1.8 Indication
- 12.2.1.9 Study Design
- 12.2.1.10 Recruitment Status
- 12.2.1.11 Enrollment (Estimated)
- 12.2.1.12 Location Countries
- 12.2.2 Other Drugs
- 13 X-linked Hypophosphatemia (XLH) Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
- 13.1 Comparative Analysis for Preclinical and Discovery Stage Drugs
- 13.1.1 Study Type
- 13.1.2 Recruitment Status
- 13.1.3 Company
- 13.1.4 Funder Type
- 13.2 Product Level Analysis*
- 13.2.1 Drug 1
- 13.2.1.1 Product Description
- 13.2.1.2 Trial ID
- 13.2.1.3 Sponsor Name
- 13.2.1.4 Study Type
- 13.2.1.5 Drug Class
- 13.2.1.6 Eligibility Criteria
- 13.2.1.7 Study Record Dates
- 13.2.1.7.1 First Submitted
- 13.2.1.7.2 First Posted
- 13.2.1.7.3 Last Update Posted
- 13.2.1.7.4 Last Verified
- 13.2.1.8 Indication
- 13.2.1.9 Study Design
- 13.2.1.10 Recruitment Status
- 13.2.1.11 Enrollment (Estimated)
- 13.2.1.12 Location Countries
- 13.2.2 Other Drugs
- 14 X-linked Hypophosphatemia (XLH), Key Drug Pipeline Companies
- 14.1 Kyowa Kirin, Inc.
- 14.1.1 Company Snapshot
- 14.1.2 Pipeline Product Portfolio
- 14.1.3 Financial Analysis
- 14.1.4 Recent News and Developments
- 14.2 Inozyme Pharma
- 14.2.1 Company Snapshot
- 14.2.2 Pipeline Product Portfolio
- 14.2.3 Financial Analysis
- 14.2.4 Recent News and Developments
- 14.3 Ultragenyx Pharmaceutical Inc.
- 14.3.1 Company Snapshot
- 14.3.2 Pipeline Product Portfolio
- 14.3.3 Financial Analysis
- 14.3.4 Recent News and Developments
- 14.4 Medialis Ltd.
- 14.4.1 Company Snapshot
- 14.4.2 Pipeline Product Portfolio
- 14.4.3 Financial Analysis
- 14.4.4 Recent News and Developments
- 14.5 Zeria Pharmaceutical
- 14.5.1 Company Snapshot
- 14.5.2 Pipeline Product Portfolio
- 14.5.3 Financial Analysis
- 14.5.4 Recent News and Developments
- 15 Regulatory Framework for Drug Approval, By Region
- 16 Terminated or Suspended Pipeline Products
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