Primary Hyperoxaluria Market

• According to DelveInsight’s estimates, the total Primary Hyperoxaluria Diagnosed Prevalent Cases in the 7MM were approximately ~3,500 cases in 2023. This number is expected to increase by 2034. 
• In 2023, the Primary Hyperoxaluria Market Size was highest in the US among the 7MM, accounting for approximately USD 281 million which is further expected to increase by 2034.
• Various therapies are employed to treat Primary Hyperoxaluria. Currently approved therapies include symptomatic treatment, OXLUMO (lumasiran), RIVFLOZA (nedosiran), and Others.

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DelveInsight’s “Primary Hyperoxaluria Market Insights, Epidemiology, and Market Forecast – 2034” report deliver an in-depth understanding of the Primary Hyperoxaluria, historical and forecasted epidemiology as well as the Primary Hyperoxaluria market trends in the United States, EU4 (Germany, France, Italy, Spain) and the United Kingdom, and Japan. 

The Primary Hyperoxaluria Treatment Market Report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Primary Hyperoxaluria market size from 2020 to 2034. The Report also covers current Primary Hyperoxaluria treatment practice, market drivers, market barriers, SWOT analysis, reimbursement and market access, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Primary Hyperoxaluria Treatment Market: Understanding and Algorithm

Primary Hyperoxaluria is a rare genetic disease caused by deficiencies in glyoxylate metabolism. Broadly, the Primary Hyperoxaluria is inherited in an autosomal recessive fashion and divided into three types based on the different responsible genes: type 1 (AGXT) (Primary Hyperoxaluria-I), type 2 (GRHPR) (Primary Hyperoxaluria-II), and type 3 (HOGA1) (Primary Hyperoxaluria-III). The kidney is the prime target for oxalate deposition, which leads to end-stage renal disease in a significant number of cases.

Every child with a first kidney stone and all adults who have recurrent calcium oxalate kidney stones are advised to be screened for Primary Hyperoxaluria. However, misdiagnosis and delayed diagnosis are very common in the clinical setting. If the diagnosis of Primary Hyperoxaluria is made early, disease progression can be slowed.

Primary Hyperoxaluria Diagnosis

Diagnosing primary hyperoxaluria involves a thorough approach that combines clinical evaluation, biochemical tests, and genetic analysis. Clinically, patients typically present with recurrent kidney stones, nephrocalcinosis, and impaired renal function. Biochemical tests reveal elevated oxalate levels in both urine and blood, with additional measurements of glycolate and oxalate in urine proving useful. For confirmatory diagnosis, genetic testing is essential to identify specific mutations associated with each type of hyperoxaluria, including mutations in the AGXT gene for Type 1, the GRHPR gene for Type 2, and the HOGA1 gene for Type 3.

Further details related to diagnosis are provided in the report…

Primary Hyperoxaluria Treatment

Before the approval of OXLUMO and RIVFLOZA, the treatment was mainly based on off-label, supportive therapies, and kidney and liver transplantation. In severe cases, the transplantation procedure of choice is isolated kidney transplantation in patients with Primary Hyperoxaluria-II, and usually combined liver-kidney transplantation in Primary Hyperoxaluria-I.

Treatment strategies for primary hyperoxaluria focus on managing symptoms, reducing oxalate production, and preventing kidney damage. Medical management typically involves the use of pyridoxine (vitamin B6) in Type 1 hyperoxaluria to help decrease oxalate production, while aggressive hydration and dietary modifications are recommended for all types to lower oxalate intake. Medications, such as potassium citrate, may be employed to reduce kidney stone formation and manage acidity. In cases of advanced kidney damage or failure, renal replacement therapies, including dialysis or kidney transplantation, may be necessary. For Type 1 hyperoxaluria, liver transplantation can also be considered to address the underlying enzymatic defect. Additionally, emerging research into gene therapy aims to tackle the root cause of the enzyme deficiencies, offering potential long-term solutions, particularly for Type 1 and Type 2 hyperoxaluria.

Primary Hyperoxaluria Epidemiology

As the market is derived using a patient-based model, the Primary Hyperoxaluria epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases of Primary Hyperoxaluria, Total Diagnosed Prevalent Cases of Primary Hyperoxaluria, Gender-specific Diagnosed Prevalent Cases of Primary Hyperoxaluria, Age-specific Diagnosed Prevalent Cases of Primary Hyperoxaluria, and Type-specific Diagnosed Prevalent Cases of Primary Hyperoxaluria, in the 7MM covering, the United States, EU4 countries (Germany, France, Italy, and Spain), United Kingdom, and Japan from 2020 to 2034. 

• In the assessment done by DelveInsight, the estimated total prevalent cases of Primary Hyperoxaluria in the 7MM were ~12 thousand in 2023. 
• The highest total diagnosed prevalent cases of Primary Hyperoxaluria were accounted by US in 2023 (~2 thousand), which are expected to show a rise in the future.
• Among the European countries, the UK had the highest diagnosed prevalent cases of Primary Hyperoxaluria with ~220 cases, followed by Germany, which had prevalent population of ~188 in 2023. On the other hand, Spain had the lowest prevalent population (~100 cases). 
• Japan had the fewest total diagnosed prevalent cases of Primary Hyperoxaluria in 2023, accounting for approximately 2.5% of the cases in 7MM.
• In 2023, in the US, the age-specific diagnosed prevalent cases of Primary Hyperoxaluria were highest for age group 0–4 (~780 cases), followed by 5–9 (~470 cases). The lowest number of cases was in the 15–19 years age group. 
• In 2023, in Japan, the type-specific diagnosed prevalent cases of Primary Hyperoxaluria were highest for Primary Hyperoxaluria Type 1 (~64 cases), followed by Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3 and unclassified.

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Primary Hyperoxaluria Recent Developments

• In February 2025, Arbor Biotechnologies announced that the FDA granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to ABO-101 for treating primary hyperoxaluria type 1 (PH1). These designations follow the FDA's clearance of the investigational new drug (IND) application for ABO-101 in December 2024.
• In Dec 2024, Arbor Biotechnologies announced that the FDA cleared the Investigational New Drug (IND) application for ABO-101, a gene editing therapy for primary hyperoxaluria type 1 (PH1). The redePHine Phase 1/2 study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ABO-101 in both adult and pediatric PH1 patients.
• In September 2024, YolTech Therapeutics, a clinical-stage in vivo gene editing company, announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to YOLT-203 for the treatment of primary hyperoxaluria type 1 (PH1). Earlier that month, YOLT-203 also received Rare Pediatric Disease Designation (RPDD) from the FDA.

Primary Hyperoxaluria Drug Chapters

The drug chapter segment of the Primary Hyperoxaluria Therapeutics Market Report encloses a detailed analysis of Primary Hyperoxaluria off-label drug and late-stage (Phase-III and Phase-II) pipeline drug. It also helps to understand the Primary Hyperoxaluria clinical trials details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Primary Hyperoxaluria Marketed Drugs

• OXLUMO (Lumasiran/ALN-GO1): Alnylam Pharma

Lumasiran (formerly ALN-GO1) developed by Alnylam Pharma, approved in Year 2020, is a subcutaneously administered, RNAi therapeutic targeting glycolate oxidase (GO). Lumasiran is designed to reduce hepatic levels of GO enzyme (encoded by HAO1), thereby depleting substrate necessary for oxalate production, which directly contributes to the pathophysiology of Primary Hyperoxaluria type 1. 

Lumasiran utilizes the companies’ Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. Lumasiran has received Orphan Drug Designations from both the US and EU and a Breakthrough Therapy Designation from the US FDA. The company believes lumasiran has the potential to address the full spectrum of Primary Hyperoxaluria type 1 disease severity.

Further detail in the report…

• RIVFLOZA (nedosiran/DCR-PHXC): Dicerna Pharmaceuticals, Inc.

Dicerna Pharmaceutical’s Nedosiran (formerly referred to as DCR-PHXC) is an advanced therapy utilized with the company’s GalXC technology to treat patients with Primary Hyperoxaluria. It was approved in Year 2023. It inhibits the lactate dehydrogenase (LDH) enzyme in the final common step of this pathway and thereby attempts to prevent this overproduction of oxalate. This LDH enzyme inhibition occurs specifically in the liver due to the incorporation of GalNAc targeting ligands in nedosiran that bind specifically to the asialoglycoprotein receptors (ASGPR) on hepatic cell surfaces. 

Dicerna has developed nedosiran as a once-monthly fixed-dose injection for the treatment of Primary Hyperoxaluria. This once-monthly formulation of nedosiran is designed to avoid the sudden oxalate spikes that could occur with less frequent administration or missed dosages, which could result in the formation of kidney stones and renal failure. In order to maximize patient convenience, the company is are developing prefilled syringes to enable self-administration by most Primary Hyperoxaluria patients without the need for the involvement of a health care provider for dosing. 

Further detail in the report…

Primary Hyperoxaluria Emerging Drugs

• Stiripentol (Diacomit): Biocodex

Stiripentol is an anticonvulsant drug used in treating epilepsy as an adjunct therapy along with Clobazam and Valproic acid. This drug is currently approved in the US, Canada, and European countries as oral tablets marketed as Diacomit. Unrelated to other anticonvulsants, stiripentol belongs to the group of aromatic allylic alcohols and may potentiate the effect of other antiepileptic drugs (AEDs) due to pharmacokinetic interactions. It elevates gamma-aminobutyric acid (GABA) levels, a major inhibitory neurotransmitter that regulates electrical activity in the central nervous system. 

Diacomit (stiripentol) is currently under Phase III clinical investigation for the treatment of patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.

In February 2021, the US FDA granted Orphan Drug Designation to stiripentol for the treatment of Primary Hyperoxaluria.

Further detail in the report…

Primary Hyperoxaluria Market Outlook

The global burden of Primary Hyperoxaluria has increased over the years. Among the seven major markets, the United States has reported having maximum diagnosed prevalent cases of Primary Hyperoxaluria. Though the prevalence of Primary Hyperoxaluria is high across the US, the condition is often misdiagnosed, ultimately leading to a lower treatable pool (when compared to the total prevalent population of the disease) that contributes to the market size of Primary Hyperoxaluria.

The conservative treatment of Primary Hyperoxaluria has not improved over the last 20 years, despite fundamental insights regarding molecular mechanisms and pathophysiology of the disease. However, in November 2020, a small interfering ribonucleic acid (RNAi) therapy, OXLUMO (lumasiran) got approval in the US and EU to treat Primary Hyperoxaluria1 to lower urinary oxalate levels in pediatric and adult patients. However, no therapy is approved yet in Japan. The approval was a breakthrough in the management of Primary Hyperoxaluria. Alnylam Pharmaceuticals is also planning to get approval in other countries as well.

The treatment usually started with citrates, vitamin B6, and hyperhydration. After that, iRNA therapies begin. Patients with advanced kidney failure go for kidney transplantation. Transplantation methods depend on the type of primary hyperoxaluria and the particular patient, but combined liver and kidney transplantation is the method of choice in patients with Primary Hyperoxaluria1, and isolated kidney transplantation is the preferred method with Primary Hyperoxaluria2.

According to DelveInsight, the Primary Hyperoxaluria Therapeutics Market in 7MM is expected to change in the study period 2020–2034.

• The Primary Hyperoxaluria Treatment Market Size in the 7MM was approximately USD 316 million in 2023.
• The United States accounted for the highest Primary Hyperoxaluria Treatment Market Size approximately 89% of the total market size in 7MM in 2023, in comparison to the other major markets i.e., EU4 countries (Germany, France, Italy, and Spain), and the United Kingdom, and Japan.
• Among the European countries, Germany had the highest Primary Hyperoxaluria Market Size with nearly ~USD 8 million in 2023, while Spain had the lowest market size of Primary Hyperoxaluria with ~USD 4.3 million in 2023.
• The Primary Hyperoxaluria Market Size in Japan was estimated to be ~USD 1.3 million in 2023.
• With the expected launch of upcoming therapies, such as Stiripentol the total market size of Primary Hyperoxaluria is expected to show change in the upcoming years.

 Primary Hyperoxaluria Drugs Uptake

This section focuses on the sales uptake of potential Primary Hyperoxaluria drugs that have recently been launched or are anticipated to be launched in the Primary Hyperoxaluria market between 2020 and 2034. It estimates the market penetration of Primary Hyperoxaluria drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the Primary Hyperoxaluria market.

Further detailed analysis of emerging therapies drug uptake in the report…

Primary Hyperoxaluria Pipeline Development Activities

The Primary Hyperoxaluria Therapeutics Market Report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I stage. It also analyzes key Primary Hyperoxaluria Companies involved in developing targeted therapeutics. 

Pipeline Development Activities

The Primary Hyperoxaluria Therapeutics Market Report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Primary Hyperoxaluria emerging therapies. 

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KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate the secondary research. Industry Experts were contacted for insights on Primary Hyperoxaluria evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility, including KOL from University of Alabama, Birmingham, US; Washington State University, US;  Division of Cardiology and Nephrology, US; Université de Bordeaux, France; Società Italiana di Nefrologia, Viale dell'Università, Italy; SSD Microcitemie, Italy; Hôpital Femme-Mère-Enfant, France; University of Verona, Italy, and others.

Delveinsight’s analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging therapies, treatment patterns, or Primary Hyperoxaluria market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided. 

Primary Hyperoxaluria Therapeutics Market Access and Reimbursement

The high cost of therapies for the treatment is a major factor restraining the growth of the global drug market. Because of the high cost, the economic burden is increasing, leading the patient to escape from proper treatment. The Primary Hyperoxaluria Therapeutics Market Report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Primary Hyperoxaluria Therapeutics Market Report Scope

• The Primary Hyperoxaluria Therapeutics Market Report covers a segment of key events, an executive summary, descriptive overview of Primary Hyperoxaluria, explaining its causes, signs and symptoms, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the Primary Hyperoxaluria Therapeutics Market, historical and forecasted Primary Hyperoxaluria Treatment Market Size, Primary Hyperoxaluria Drugs Market Share by therapies, detailed assumptions, and rationale behind the approach is included in the report covering the 7MM drug outreach.
• The Primary Hyperoxaluria Therapeutics Market Report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM Primary Hyperoxaluria Drugs Market.

Primary Hyperoxaluria Therapeutics Market Report Insights

• Patient-based Primary Hyperoxaluria Market Forecasting
• Therapeutic Approaches
• Primary Hyperoxaluria Pipeline Drugs Analysis
• Primary Hyperoxaluria Market Size and Trends
• Existing and Future Primary Hyperoxaluria Drugs Market Opportunity 

Primary Hyperoxaluria Therapeutics Market Report Key Strengths

• 11 years Primary Hyperoxaluria Market Forecast
• The 7MM Coverage 
• Primary Hyperoxaluria Epidemiology Segmentation
• Key Cross Competition 
• Conjoint Analysis
• Primary Hyperoxaluria Drugs Uptake
• Key Primary Hyperoxaluria Market Forecast Assumptions

Primary Hyperoxaluria Therapeutics Market Report Assessment

• Current Primary Hyperoxaluria Treatment Market Practices
• Primary Hyperoxaluria Unmet Needs
• Primary Hyperoxaluria Pipeline Drugs Analysis Profiles
• Primary Hyperoxaluria Drugs Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

Key Questions

Primary Hyperoxaluria Therapeutics Market Insights

• What was the Primary Hyperoxaluria Drugs Market Share (%) distribution in 2020 and how it would look like in 2034?
• What would be the Primary Hyperoxaluria Treatment Market Size as well as market size by therapies across the 7MM during the forecast period (2024–2034)?
• What are the key findings pertaining to the Primary Hyperoxaluria Drugs Market across the 7MM and which country will have the largest Primary Hyperoxaluria market size during the forecast period (2024–2034)?
• At what CAGR, the Primary Hyperoxaluria market is expected to grow at the 7MM level during the forecast period (2024–2034)?
• What would be the Primary Hyperoxaluria market outlook across the 7MM during the forecast period (2024–2034)?
• What would be the Primary Hyperoxaluria market growth till 2034 and what will be the resultant market size in the year 2034?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Primary Hyperoxaluria Epidemiology Insights

• What is the disease risk, burden, and Primary Hyperoxaluria Unmet Needs?
• What is the historical Primary Hyperoxaluria patient population in the United States, EU4 (Germany, France, Italy, Spain) and the UK, and Japan?
• What would be the forecasted patient population of Primary Hyperoxaluria at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Primary Hyperoxaluria?
• Out of the above-mentioned countries, which country would have the highest prevalent population of Primary Hyperoxaluria during the forecast period (2024–2034)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2024–2034)?

Current Primary Hyperoxaluria Treatment Market Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options for the Primary Hyperoxaluria treatment along with the approved therapy?
• What are the current treatment guidelines for the treatment of Primary Hyperoxaluria in the US, EU4 and the UK, And Japan?
• What are the Primary Hyperoxaluria marketed drugs and their Primary Hyperoxaluria MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for the treatment of Primary Hyperoxaluria?
• How many emerging therapies are in the mid-stage and late stages of development for the treatment of Primary Hyperoxaluria?
• What are the key collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Primary Hyperoxaluria therapies? 
• What are the recent therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies? 
• What are the clinical studies going on for Primary Hyperoxaluria and their status?
• What are the key designations that have been granted for the Primary Hyperoxaluria Emerging Therapies?
• What are the 7MM historical and forecasted Primary Hyperoxaluria Drugs Market?

Reasons to Buy

• The Primary Hyperoxaluria Therapeutics Market Report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Primary Hyperoxaluria Drugs Market.
• Insights on patient burden/disease Primary Hyperoxaluria Incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• To understand the existing Primary Hyperoxaluria Drugs Market opportunity in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identification of strong upcoming players in the Primary Hyperoxaluria Drugs Market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and potential of current and emerging therapies under the conjoint analysis section to provide visibility around leading emerging drugs.
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs.
• To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in future.
• Detailed insights on the unmet need of the existing Primary Hyperoxaluria Drugs Market so that the upcoming players can strengthen their development and launch strategy.

Stay Updated with us for Recent Articles

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