Primary Hyperoxaluria Therapeutics Market Size in the 7MM was ~USD 316 million in 2023, estimated DelveInsight

DelveInsight's "Primary Hyperoxaluria Market Insights, Epidemiology, and Market Forecast-2034 report offers 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, Spain, Italy, France) the United Kingdom and Japan.

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Key Takeaways from Primary Hyperoxaluria Market Report

* 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.
* The leading Primary Hyperoxaluria Companies such as Dicerna Pharmaceuticals, Inc., Allena Pharmaceuticals, Biocodex, Alnylam Pharmaceuticals, Biocodex, Dicerna Pharmaceuticals , and others
* Promising Primary Hyperoxaluria Therapies such as Nedosiran (DCR-PHXC), ALLN-177, Stiripentol, Lumasiran, stiripentol (Diacomit), DCR-PHXC , and others

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Primary Hyperoxaluria Epidemiology Segmentation in the 7MM

* Prevalent Cases of Primary Hyperoxaluria in the 7MM
* Diagnosed Prevalent Cases of Primary Hyperoxaluria in the 7MM
* Gender-specific Cases of Diagnosed Prevalent Cases of Primary Hyperoxaluria in the 7MM
* Age-specific Cases of Diagnosed Prevalent Cases of Primary Hyperoxaluria in the 7MM
* Type-specific Cases of Diagnosed Prevalent Cases of Primary Hyperoxaluria in the 7MM

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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.

* 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.

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.

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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.

Primary Hyperoxaluria Treatment Market

The Primary Hyperoxaluria 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.

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Scope of the Primary Hyperoxaluria Market Report

* Study Period: 2020-2034
* Coverage: 7MM
* Primary Hyperoxaluria Companies: Dicerna Pharmaceuticals, Inc., Allena Pharmaceuticals, Biocodex, Alnylam Pharmaceuticals, Biocodex, Dicerna Pharmaceuticals, and others
* Primary Hyperoxaluria Therapies: Nedosiran (DCR-PHXC), ALLN-177, Stiripentol, Lumasiran, stiripentol (Diacomit), DCR-PHXC, and others
* Primary Hyperoxaluria Therapeutic Assessment: Primary Hyperoxaluria current marketed and Primary Hyperoxaluria emerging therapies
* Primary Hyperoxaluria Market Dynamics: Primary Hyperoxaluria market drivers and Primary Hyperoxaluria market barriers
* Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
* Primary Hyperoxaluria Unmet Needs, KOL's views, Analyst's views, Primary Hyperoxaluria Market Access and Reimbursement

Table of Contents

1. Primary Hyperoxaluria Market Report Introduction

2. Executive Summary for Primary Hyperoxaluria

3. SWOT analysis of Primary Hyperoxaluria

4. Primary Hyperoxaluria Patient Share (%) Overview at a Glance

5. Primary Hyperoxaluria Market Overview at a Glance

6. Primary Hyperoxaluria Disease Background and Overview

7. Primary Hyperoxaluria Epidemiology and Patient Population

8. Country-Specific Patient Population of Primary Hyperoxaluria

9. Primary Hyperoxaluria Current Treatment and Medical Practices

10. Primary Hyperoxaluria Unmet Needs

11. Primary Hyperoxaluria Emerging Therapies

12. Primary Hyperoxaluria Market Outlook

13. Country-Wise Primary Hyperoxaluria Market Analysis (2020-2034)

14. Primary Hyperoxaluria Market Access and Reimbursement of Therapies

15. Primary Hyperoxaluria Market Drivers

16. Primary Hyperoxaluria Market Barriers

17. Primary Hyperoxaluria Appendix

18. Primary Hyperoxaluria Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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