Hyperoxaluria - Market Insights, Epidemiology, and Market Forecast - 2034

Key Highlights:

• Hyperoxaluria, a clinical condition associated with excess urinary oxalate excretion, is commonly encountered and is seen in 25-45% of stone formers.
• Primary Hyperoxaluria type 1 (PH1) accounts for approximately 80% of the cases of primary hyperoxaluria and is caused by a defect in the Vitamin B6-dependent hepatic peroxisomal enzyme, Alanine Glyoxalate Aminotransferase (AGT).
• PH1 is the most severe form; overall, more than 70% of PH1 patients develop end-stage kidney disease over time; this may even occur in patients with sporadic stone disease.
• PH2 has a more benign course; no infantile oxalosis has been described, and end-stage kidney disease occurs at a relatively late age in about 20% of patients. PH3 is most benign, with so far only a few reports of renal impairment and no end-stage kidney disease.
• The US Food and Drug Administration (FDA) approved OXLUMO (Alnylam Pharmaceuticals) in 2020 and RIVFLOZA [Novo Nordisk (Dicerna Pharmaceuticals)] in 2023 for the treatment of PH1.
• The market for OXLUMO reflects a positive trajectory in both market performance and therapeutic potential for treating PH1. The drug generated approximately USD 167 million in global net product revenues for the full year of 2024, reflecting a 29% increase compared to the previous year.
• In May 2025, Arbor Biotechnologies presented IND and CTA-enabling data for its lead clinical program, ABO-101, in PH1 at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans, Louisiana.
• The hyperoxaluria pipeline is severely underdeveloped, reflecting a major unmet need and lack of innovation. Arbor Biotechnologies leads the emerging space, aiming to address both the genetic and metabolic drivers of hyperoxaluria.

DelveInsight's "Hyperoxaluria - Market Insight, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of hyperoxaluria, historical and forecasted epidemiology, as well as the hyperoxaluria market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The hyperoxaluria market report provides current treatment practices, emerging drugs, the hyperoxaluria share of individual therapies, and current and forecasted hyperoxaluria market size from 2020 to 2034, segmented by seven major markets. The report also covers current hyperoxaluria treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Hyperoxaluria Disease Understanding and Treatment Algorithm

Hyperoxaluria Overview

Hyperoxaluria is a condition characterized by excessive levels of oxalate in the urine, which can lead to the formation of calcium oxalate kidney stones, nephrocalcinosis, and progressive kidney damage. It can be classified into primary, secondary, and enteric forms. Primary hyperoxaluria (PH) is a rare genetic disorder caused by enzyme deficiencies in the liver that lead to oxalate overproduction, with three known types: PH1, PH2, and PH3. PH1 is the most severe form, often leading to early-onset kidney stones and systemic oxalosis. Secondary hyperoxaluria arises from increased dietary oxalate intake, vitamin C overuse, or conditions that cause fat malabsorption, while enteric hyperoxaluria is commonly associated with gastrointestinal disorders like inflammatory bowel disease or after bariatric surgery. Symptoms typically include recurrent kidney stones, flank pain, blood in the urine, and, in severe cases, renal failure. Diagnosis involves 24-hour urine oxalate measurements, genetic testing for PH, plasma oxalate levels in patients with reduced kidney function, and imaging studies. Management strategies focus on reducing oxalate levels and preventing stone formation through high fluid intake, dietary modifications, and medications such as vitamin B6 and citrate supplements. Recent therapeutic advances include RNA interference (RNAi) therapies like lumasiran, approved for PH1, and investigational agents like nedosiran, offering promising options for targeted treatment. In advanced cases, dialysis or liver and combined liver-kidney transplantation may be necessary. Early diagnosis and intervention are key to preventing complications and preserving kidney function.

Hyperoxaluria Diagnosis

Evaluation of acute renal colic should begin with urinalysis and a noncontrast CT scan of the abdomen and pelvis, the gold standard for detecting kidney stones, particularly in the presence of hematuria. A follow-up KUB X-ray may assist in tracking radiopaque stones like calcium oxalate if >=2-3 mm. Chemical analysis of retrieved stones can help determine their composition, which is key to identifying underlying causes such as primary hyperoxaluria (PH), where calcium oxalate monohydrate stones predominate. Suspected hyperoxaluria requires a 24-hour urine collection, ideally done at home under usual dietary conditions. Normal oxalate excretion is <=40 mg/day, with >75 mg/day suggesting PH and >100 mg/day possibly indicating enteric hyperoxaluria, often seen in patients with chronic diarrhea or post-gastric bypass. Biochemical markers such as glycolate, glycerate, and HOG/DHG help differentiate PH subtypes. Genetic testing for PH (AGXT, GRHPR, HOGA1) is recommended for patients with high urinary oxalate or recurrent stones.

Hyperoxaluria Treatment

Hyperoxaluria treatment focuses on reducing oxalate levels and preventing kidney stones. Key strategies include increasing fluid intake to produce over 3 liters of urine daily, using potassium citrate to boost urinary citrate and optimize pH, and avoiding high-oxalate foods and excess vitamin C. Calcium or iron supplements with meals help bind dietary oxalate, while pyridoxine (vitamin B6) may benefit some patients with primary hyperoxaluria type I. For enteric hyperoxaluria, cholestyramine can reduce oxalate absorption and diarrhea. In advanced PH, RNA interference therapies like lumasiran and nedosiran target oxalate-producing enzymes and have shown promising results. Dialysis may be used in severe cases, though not curative, while combined liver-kidney transplantation remains the most effective long-term solution. Emerging therapies include gene editing, oxalate-degrading enzymes, and experimental plant-based treatments. Regular monitoring and personalized care are essential.

Hyperoxaluria Epidemiology

The hyperoxaluria epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the total prevalent cases of hyperoxaluria, gender-specific cases of hyperoxaluria, age-specific cases of hyperoxaluria, and type-specific cases of hyperoxaluria in the 7MM market covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• In the US, hyperoxaluria is estimated to affect approximately 1 to 3 individuals per million people.
• In Europe, the prevalence of primary hyperoxaluria is estimated to be between 1 and 3 cases per 1,000,000 individuals.
• The highest prevalence of symptom onset and PH1 diagnosis occurs in infants and young children, with rapid disease progression in severe cases.
• PH1 accounts for approximately 80% of all hyperoxaluria cases, while PH2 and PH3 each represent about 10%.

Hyperoxaluria Drug Chapters

Currently, OXLUMO (Alnylam Pharmaceuticals) and RIVFLOZA (Novo Nordisk/Dicerna Pharmaceuticals) are the only FDA-approved medications for treating primary hyperoxaluria type 1.

Marketed Drugs

OXLUMO (lumasiran): Alnylam Pharmaceuticals

OXLUMO is a ribonucleic acid interference (RNAi) therapeutic targeting hydroxyacid oxidase 1 for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients. Hydroxyacid oxidase 1 encodes glycolate oxidase, an enzyme upstream of the disease-causing defect in PH1. In the ILLUMINATE-B pediatric Phase III study, OXLUMO demonstrated an efficacy and safety profile consistent with that observed in ILLUMINATE-A.

In November 2020, the FDA approved OXLUMO (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients.

RIVFLOZA (nedosiran): Novo Nordisk/Dicerna Pharmaceuticals

RIVFLOZA injection is a once-monthly subcutaneous RNAi therapy indicated to lower urinary oxalate levels in children 9 years of age and older and adults with PH1 and relatively preserved kidney function, e.g., eGFR >=30 mL/min/1.73 m2.

In October 2023, the FDA approved Novo Nordisk's Rivfloza injection, designed to lower urinary oxalate levels in children 9 years or older and adults with PH1 and relatively preserved kidney function.

Emerging Drugs

ABO-101: Arbor Biotechnologies

ABO-101 is a novel, investigational gene editing medicine designed to be a one-time treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 consists of a lipid nanoparticle messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene. It is currently in Phase I/II of development.

• In May 2025, Arbor Biotechnologies presented IND- and CTA-enabling data for its lead clinical program, ABO-101, in PH1 at the ASGCT 28th Annual Meeting in New Orleans, Louisiana.
• In February 2025, Arbor Biotechnologies announced that the US FDA had granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to ABO-101 for the treatment of PH1.

Drug Class Insight

In both the marketed and emerging therapies for hyperoxaluria, RNA interference (RNAi) has emerged as the dominant therapeutic class, reflecting a significant shift toward precision, gene-targeted approaches. This modality offers a promising strategy for reducing endogenous oxalate production, particularly in primary hyperoxaluria.

RNA interference (RNAi)

RNAi represents a promising therapeutic strategy aimed at reducing endogenous oxalate production, particularly in PH. RNAi utilizes small interfering RNAs (siRNAs) to selectively silence genes involved in oxalate biosynthesis. For example, lumasiran, an FDA-approved siRNA therapy, targets the HAO1 gene encoding glycolate oxidase-an upstream enzyme in the glyoxylate-oxalate pathway-thereby decreasing oxalate formation in PH type I. Similarly, nedosiran targets hepatic lactate dehydrogenase A (LDHA), a downstream enzyme common to all three PH subtypes, offering broader potential. AB-101, currently under development, is an RNAi therapeutic designed to inhibit glycolate oxidase (GO)-a key enzyme upstream in the oxalate biosynthesis pathway. By silencing the gene encoding GO, AB-101 reduces glyoxylate availability, thereby lowering downstream oxalate levels. These therapies work by delivering synthetic siRNAs that are incorporated into the RNA-induced silencing complex (RISC), guiding the complex to degrade target mRNAs and suppress protein synthesis. RNAi-based interventions have shown significant reductions in urinary and plasma oxalate levels in clinical trials, marking a shift from symptomatic management to targeted molecular correction in PH.

Hyperoxaluria Market Outlook

The therapeutic landscape for hyperoxaluria, particularly primary hyperoxaluria, has evolved significantly in recent years. Historically, management relied on conservative measures such as high fluid intake, dietary oxalate restriction, citrate supplementation, and pyridoxine in responsive cases. While these approaches aim to reduce stone formation and delay renal deterioration, they do not target the underlying metabolic defect. For advanced disease, dialysis and combined liver-kidney transplantation have long been the only definitive options, highlighting the urgent need for targeted, disease-modifying therapies.

Recent advances in RNA interference (RNAi) technology have ushered in a new era of precision treatment. OXLUMO, the first FDA- and EMA-approved RNAi therapy for PH1, inhibits glycolate oxidase upstream of oxalate production, resulting in significant reductions in urinary and plasma oxalate levels. It is administered subcutaneously and has demonstrated long-term efficacy and safety in both adult and pediatric populations. RIVFLOZA, another RNAi therapeutic, targets lactate dehydrogenase A (LDHA), a key enzyme further downstream in oxalate biosynthesis. With once-monthly dosing, it offers broader applicability for multiple PH types. AB-101, a next-generation LDHA-targeting GalNAc-siRNA currently in development, aims to improve delivery efficiency and potency while maintaining favorable tolerability.

Together, these innovations reflect a paradigm shift in hyperoxaluria treatment, moving from symptomatic management to molecularly targeted interventions. While challenges remain in treating enteric and secondary hyperoxaluria, the arrival of RNAi therapeutics like Lumasiran, Rivfloza, and investigational candidates like AB-101 marks a transformative step forward in improving outcomes and quality of life for patients with this rare but serious disorder.

Hyperoxaluria Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034. The landscape of hyperoxaluria treatment has experienced a profound transformation with the uptake of novel drugs. These innovative therapies are redefining standards of care. Furthermore, the increased uptake of these transformative drugs is a testament to the unwavering dedication of physicians, oncology professionals, and the entire healthcare community in their tireless pursuit of advancing cancer care. This momentous shift in treatment paradigms is a testament to the power of research, collaboration, and human resilience.

Hyperoxaluria Pipeline Development Activities

The report provides insights into different therapeutic candidates in the Phase I/II stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers detailed information on collaborations, acquisitions and mergers, licensing, and patent details for hyperoxaluria emerging therapies.

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 our secondary research. Some of the leaders like MD, Professor and Vice Chair of the Department of Rheumatology and Director, PhD, and others. Their opinion helps to understand and validate current and emerging therapies and treatment patterns or 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.

Delveinsight's analysts connected with 15+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as the Washington University School of Medicine, University Medical Center Hamburg-Eppendorf, and University Graduate School of Medicine, etc. were contacted. Their opinion helps understand and validate hyperoxaluria epidemiology and market trends.

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, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

The analyst analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.

In efficacy, the trial's primary and secondary outcome measures are evaluated.

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.

Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and a payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health technology assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug. In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs, including Medicare, Medicaid, Health Insurance Program (CHIP), and the state and federal health insurance marketplaces, are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs) and third-party organizations that provide services and educational programs to aid patients are also present.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used 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.

Scope of the Report:

• The report covers a descriptive overview of hyperoxaluria, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into hyperoxaluria epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for hyperoxaluria is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of the hyperoxaluria market, historical and forecasted, is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM hyperoxaluria market.

Hyperoxaluria Report Insights

Hyperoxaluria Report Insights

• Patient Population
• Therapeutic Approaches
• Hyperoxaluria Pipeline Analysis
• Hyperoxaluria Market Size and Trends
• Market Opportunities
• Impact of Upcoming Therapies

Hyperoxaluria Report Key Strengths

• Ten-Year Forecast
• 7MM Coverage
• Hyperoxaluria Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

Hyperoxaluria Report Assessment

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

FAQs:

• What was the hyperoxaluria market share (%) distribution in 2020, and what would it look like in 2034?
• What would be the hyperoxaluria total market size, as well as market size by therapies across the 7MM during the study period (2020-2034)?
• What are the key findings about the market across the 7MM, and which country will have the largest hyperoxaluria market size during the study period (2020-2034)?
• At what CAGR, the hyperoxaluria market expected to grow at the 7MM level during the study period (2020-2034)?
• What would be the hyperoxaluria market growth till 2034?
• What are the disease risks, burdens, and unmet needs of hyperoxaluria?
• What is the historical hyperoxaluria patient pool in the United States, the EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
• What will be the growth opportunities across the 7MM concerning the patient population of hyperoxaluria?
• Among the 7MM, which country would have the most prevalent cases of hyperoxaluria?
• At what CAGR is the population expected to grow across the 7MM during the study period (2020-2034)?
• How many companies are developing therapies for the treatment of hyperoxaluria?
• How many emerging therapies are in the mid-stage and late-stage of development for the treatment of hyperoxaluria?
• What are the key collaborations (industry-industry, industry-academia), Mergers and acquisitions, and licensing activities related to hyperoxaluria therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• What are the clinical studies going on for hyperoxaluria and their status?
• What are the key designations that have been granted for the emerging therapies for hyperoxaluria?
• What is the 7MM historical and forecasted market of hyperoxaluria?

Reasons to buy:

• The report will help in developing business strategies by understanding trends shaping and driving the hyperoxaluria market.
• To understand the future market competition in the hyperoxaluria market and insightful review of the SWOT analysis of hyperoxaluria.
• Organize sales and marketing efforts by identifying the best opportunities for hyperoxaluria in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• To understand the future market competition in hyperoxaluria.