X-Linked Hypophosphatemia (XLH) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

X-Linked Hypophosphatemia (XLH) Market Outlook

Thelansis’s “X-Linked Hypophosphatemia (XLH) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential X-Linked Hypophosphatemia treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

X-Linked Hypophosphatemia (XLH) Overview

X-linked hypophosphatemia (XLH) is a hereditary disorder affecting phosphate metabolism. It arises from mutations in the Phosphate Regulating Endopeptidase Homolog, X-Linked (PHEX) gene. These mutations result in local and systemic effects, encompassing impaired growth, bone pain, osteomalacia, bone deformities, rickets, spontaneous dental abscesses, osteoarthritis, enthesopathy, hearing issues, and muscular dysfunction. XLH patients exhibit elevated levels of fibroblast growth factor 23 (FGF23). Diagnosis relies on a consistent medical history, physical examination, radiological evidence of rickets, biochemical tests, and a family history indicating the presence of XLH, whether through multiple generations or sporadic occurrences. The differential diagnosis should consider nutritional rickets, metaphyseal dysplasia, and other renal phosphate wasting disorders. Fibroblast growth factor 23 (FGF23) is primarily produced by osteocytes and osteoblasts, playing a crucial role in regulating phosphate metabolism. Mutations in the PHEX gene, which is highly expressed in osteocytes and osteoblasts, lead to elevated FGF23 levels. This occurs because abnormal cleavage of FGF23 results in hypophosphatemia due to impaired phosphate reabsorption in the renal proximal tubules (causing reduced NPT2a and 2c transporter activity) and decreased phosphate absorption in the intestines (due to decreased activity of 1α-hydroxylase, which reduces the renal synthesis of 1,25(OH)2D3).

• A humanized monoclonal antibody for FGF23, known as burosumab, has received approval for treating XLH in pediatric patients aged one year and older. Crysvita® (burosumab) was approved by the FDA and became available in the USA in March 2018. Additionally, it has gained approval from the EMA for use in 28 countries.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2021-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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