Primary Hyperoxaluria Type 1 (PH1) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034

 Primary Hyperoxaluria Type 1 (PH1) Market Outlook

Thelansis’s “Primary Hyperoxaluria Type 1 (PH1) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034" 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 Primary Hyperoxaluria Type 1 (PH1) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Primary Hyperoxaluria Type 1 (PH1) Overview

Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by mutations in the AGXT gene, inherited in an autosomal recessive manner. This condition inflicts irreversible harm on the kidneys and other vital organs, carrying the potential for life-threatening consequences. PH1 results from a deficiency of the liver peroxisomal enzyme called alanine: glyoxylate-aminotransferase (AGT). AGT plays a crucial role in converting glyoxylate into glycine. Individuals affected by PH1 are susceptible to recurrent nephrolithiasis, which is the deposition of calcium oxalate in the renal pelvis and urinary tract, nephrocalcinosis, characterized by calcium oxalate buildup in the renal parenchyma, and end-stage renal disease (ESRD). Roughly 10% of those with PH1 experience symptoms during infancy or early childhood, including nephrocalcinosis, sometimes nephrolithiasis, and growth issues due to renal failure. Most individuals with PH1 are diagnosed during childhood or early adolescence, often presenting with symptomatic nephrolithiasis and either normal or diminished kidney function. PH1 diagnosis is confirmed in individuals showing hyperoxaluria or hyperoxaluria through genetic testing that identifies biallelic pathogenic variants in AGXT. The primary approach to preventing and treating the renal and systemic complications of PH1 is to reduce the liver’s excessive oxalate production. Two main options are available: pharmacologic doses of pyridoxine (restricted to those whose AGXT genotype responds to pyridoxine) or liver transplantation, frequently combined with or sequentially performed alongside kidney transplantation in ESRD cases. In 2020, the FDA and EMA approved lumasiran, an mRNA therapeutic agent that reduces glyoxylate substrate levels available for metabolic conversion to oxalate.

 

Geography coverage:

G8 (United States, EU5 [France, Germany, Italy, Spain, U.K.], Japan, and China)

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support

*As per Thelansis’s policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2024–2034)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market’s trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?
• and more…

Read more: Primary Hyperoxaluria Type 1 (PH1) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034