primary hyperoxaluria market insight

DelveInsight’s 'Primary Hyperoxaluria - Market Insights, Epidemiology, and Market Forecast–2030’ report delivers an in-depth understanding of the Primary Hyperoxaluria historical and forecasted epidemiology as well as the Primary Hyperoxaluria market trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

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

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2018–2030

Primary Hyperoxaluria Disease Understanding and Treatment

Primary Hyperoxaluria Overview

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

Signs and symptoms may include recurrent kidney stones, hematuria, and urinary tract infections (UTIs). If the disease is left untreated, PH-I can result in end-stage renal disease, which is life-threatening. Phenotypically the disease is characterized by the recurrent formation of kidney stones and nephrocalcinosis, resulting in the gradual decline of kidney function. Once renal function deteriorates, excessive oxalate can no longer be eliminated through the urine, and thus calcium oxalate crystals precipitate in various tissues, leading to a devastating multiorgan disease called systemic oxalosis.

Primary Hyperoxaluria Diagnosis

Diagnosis of PH is based upon identification of characteristic symptoms (e.g. chronic stone formation), a detailed patient history, a thorough clinical evaluation, and a variety of specialized tests. Children with nephrocalcinosis or kidney stones should be screened for PH. PH may be suspected in individuals with a history of recurrent kidney stones and/or nephrocalcinosis. Because these conditions are rare, there may be a delay from symptom onset to diagnosis. Investigation for PH includes measuring urine and plasma oxalate levels, ruling out other causes of high oxalate levels (dietary or enteric hyperoxaluria), molecular genetic testing for mutations, and detecting the presence of kidney stones and examining their composition.

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Primary Hyperoxaluria Treatment

The goal of treatment in PH is to lower the level of oxalate in the body and prevent calcium oxalate crystals from forming in the kidneys and other body tissues. Treatment must be introduced at an early stage, and it is aimed at decreasing urinary saturation of calcium oxalate by increasing fluid intake and using urinary crystallization inhibitors. Fluid intake must be greater than 3 L/m2/day, and urinary pH must be maintained between 6.2 and 6.8. These general measurements apply to all hyperoxaluric states and their effectiveness will depend on the extent of the problem. As such, in severe cases of PH, it is usually very limited.

Oxlumo (lumasiran) got approval in the US and EU to treat PH1 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 PH.

The therapeutic options for PH are divided into crystallization inhibitor regimens, supportive measures, shock-wave therapy, hemodialysis or, even more so, peritoneal dialysis and at last transplantation. The choice of treatment depends on the individual patient’s circumstances, especially the severity of the PH and the presence of comorbidities.

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Primary Hyperoxaluria Epidemiology  

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Prevalent Cases of Primary Hyperoxaluria, 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 scenario in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2018 to 2030.

Key Findings

• The total prevalent population of PH in 7MM was 11,918 in 2020, which is expected to increase at a CAGR of 0.78%. However, the majority of the cases remain undiagnosed.
• The US accounted for the highest prevalent population of PH among 7MM with 8,773 cases in 2020.
• The estimates suggest a higher diagnosed prevalence of PH in the United States with 2,327 diagnosed cases in 2020.
• Diagnosed Prevalent population of PH in the US is expected to increase at a CAGR of 3.80% for the study period, i.e. 2018–2030.
• Age-groups 0–4 years contributed to 29.96% of the total diagnosed prevalent cases of PH in the US, in 2020, followed by age groups 5–9 years.
• Among the European countries, the UK had the highest diagnosed prevalent population of PH with 198 cases, followed by Germany which had diagnosed prevalent population of 171, in 2020. On the other hand, Spain had the lowest diagnosed prevalent population (95 cases).
• Epidemiology assessed for PH showed that Japan accounts for approximately 2.57% of the total 7MM diagnosed prevalent population, which approximates 81 cases in 2020.
• The estimates suggest a higher diagnosed prevalence of PH Type 1 throughout the 7MM. In the US, out of 2,327 diagnosed prevalent cases of PH; 1,629 were of PH Type 1 alone, in the year 2020.
• In 2020, out of total diagnosed prevalent cases in Japan, males and females contributed to 48 and 33 cases, respectively.

Country-Wise Primary Hyperoxaluria Epidemiology

The epidemiology segment also provides the Primary Hyperoxaluria epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

Primary Hyperoxaluria Drug Chapters

Primary Hyperoxaluria Marketed Drug

Oxlumo (Lumasiran/ALN-GO1): Alnylam Pharmaceuticals

Lumasiran (formerly ALN-GO1) being developed by Alnylam Pharmaceuticals, is a subcutaneously administered, investigational 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 PH1.

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. Currently, the company is evaluating lumasiran for severe PH1. The company believes lumasiran has the potential to address the full spectrum of PH1 disease severity and looks forward to reporting topline results from the important study in mid-2021.

Products detail in the report…

Primary Hyperoxaluria Emerging Drugs

Oxabact (OC5-DB-02): OxThera

Oxabact is a bimodal enteric biotherapy containing a lyophilized formulation of Oxalobacter formigenes, a nonpathogenic, oxalate-degrading bacterium. Oxabact is administered orally as a coated capsule. By promoting active and passive secretion of oxalate from the plasma into the gut, Oxabact can eliminate oxalate, thereby lowering the oxalate burden in the kidneys.

Oxabact comprises highly concentrated freeze-dried live bacteria; these commensal bacteria promote the enteric elimination of oxalate, thereby reducing the oxalate burden in the kidneys. OxThera holds worldwide rights for compositions and methods of use for the treatment of hyperoxaluria. Oxabact holds orphan drug designations in the EU and the US for the treatment of PH. The first study results are expected in June 2021. The company is planning to file for approval in the second half of 2021.

Products detail in the report…

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 PH. 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. It is an RNAi therapy in development for the treatment of all three known types of PH. The company is evaluating nedosiran in the PHYOX clinical program. Data from the PHYOX1, PHYOX2, and PHYOX4 clinical trials, the ongoing PHYOX3 open-label extension study, and PHYOX-OBX are expected to support the Nedosiran’s New Drug Application (NDA) submission. The company anticipates submitting an NDA in the third quarter of 2021.

Products detail in the report…

List to be continued 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 PH. Though the prevalence of PH 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 PH.

The treatment includes crystallization inhibitor regimens, supportive measures, shock-wave therapy, hemodialysis or, even more so, peritoneal dialysis, and at last transplantation.

The conservative treatment of PH 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 PH1 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 PH. 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 PH1, and isolated kidney transplantation is the preferred method with PH2.

The dynamics of the PH market are anticipated to change during the forecasted period owing to the expected launch of emerging therapies during the forecasted period of 2021–2030. There are presently two pipeline therapies under Phase III of the clinical trial for Oxabact (OxThera) and Nedosiran (Dicerna Pharmaceuticals). They are expected to reach the market in 2022. Both these therapies target all PH types; hence, they might hugely affect the market of Oxlumo.

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Key Findings

• The market size of PH in the 7MM is estimated to be USD 19 million in 2020, which is further expected to increase by 2030 with a CAGR of 40.40%.
• The United States holds the largest market share of the PH among the 7MM countries. In 2020, the market size of PH in the US was USD 16 million.
• In 2020, among the EU-5 countries, the UK had the highest market share, followed by Germany and France.
• Japan holds the smallest share of the 7MM PH market with a market size of USD 0.3 million in 2020 that is expected to increase by 2030.

The United States Market Outlook

This section provides the total Primary Hyperoxaluria market size and market size by therapies in the United States.

EU-5 Market Outlook

The total Primary Hyperoxaluria market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan Market Outlook

The total Primary Hyperoxaluria market size and market size by therapies in Japan are provided.

Primary Hyperoxaluria Drugs Uptake

This section focuses on the rate of uptake of the potential drugs recently launched in the Primary Hyperoxaluria market or expected to get launched in the market during the study period 2018–2030. The analysis covers the Primary Hyperoxaluria market uptake by drugs; patient uptake by therapies; and sales of each drug. 

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allows the comparison of the drugs based on market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Primary Hyperoxaluria Development Activities

The report provides insights into different therapeutic candidates in phase II and phase III stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Primary Hyperoxaluria emerging therapies.

Reimbursement Scenario in Primary Hyperoxaluria

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In the report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

Competitive Intelligence Analysis

We perform competitive market Intelligence analysis of the Primary Hyperoxaluria market by using various competitive intelligence tools that include–SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of Primary Hyperoxaluria, explaining its causes, symptoms, pathophysiology, genetic basis, diagnosis, and currently available therapies.
• Comprehensive insight has been provided into the Primary Hyperoxaluria epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for Primary 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 Primary 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 Primary Hyperoxaluria market.

Report Highlights

• The robust pipeline with novel MOA and oral ROA, increasing prevalence, and effectiveness of drugs will positively drive the Primary Hyperoxaluria market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Primary Hyperoxaluria R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for Primary Hyperoxaluria. The launch of emerging therapies will significantly impact the Primary Hyperoxaluria market.
• Our in-depth analysis of the pipeline assets across different stages of development (phase III and phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Primary Hyperoxaluria Report Insights

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

Primary Hyperoxaluria Report Key Strengths

• 10 Years Forecast
• 7MM Coverage
• Primary Hyperoxaluria Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

Primary Hyperoxaluria Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers
• SWOT analysis

Key Questions

Market Insights:

• What was the Primary Hyperoxaluria market share (%) distribution in 2018 and how it would look like in 2030?
• What would be the Primary Hyperoxaluria total market size as well as market size by therapies across the 7MM during the forecast period (2021–2030)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest Primary Hyperoxaluria market size during the forecast period (2021–2030)?
• At what CAGR, the Primary Hyperoxaluria market is expected to grow at the 7MM level during the forecast period (2021–2030)?
• What would be the Primary Hyperoxaluria market outlook across the 7MM during the forecast period (2021–2030)?
• What would be the Primary Hyperoxaluria market growth till 2030 and what will be the resultant market size in the year 2030?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What are the disease risk, burdens, and unmet needs of Primary Hyperoxaluria?
• What is the historical Primary Hyperoxaluria patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK), and Japan?
• What would be the forecasted patient pool 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 population of Primary Hyperoxaluria during the forecast period (2021–2030)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2021–2030)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current options for the treatment of Primary Hyperoxaluria along with the approved therapy?
• What are the current treatment guidelines for the treatment of Primary Hyperoxaluria?
• What are the Primary Hyperoxaluria marketed drugs and their 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 novel 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 emerging therapies for Primary Hyperoxaluria?
• What are the 7MM historical and forecasted market of Primary Hyperoxaluria?

Reasons to buy

• The report will help in developing business strategies by understanding trends shaping and driving Primary Hyperoxaluria.
• To understand the future market competition in the Primary Hyperoxaluria market and an insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Primary Hyperoxaluria in the US, Europe (Germany, Spain, Italy, France, and 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.
• Organize sales and marketing efforts by identifying the best opportunities for the Primary Hyperoxaluria market.
• To understand the future market competition in the Primary Hyperoxaluria market.