Primary Hyperoxyaluria - Pipeline Insight, 2025

This “Primary Hyperoxyaluria - Pipeline Insight, 2025” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Primary Hyperoxyaluria pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Primary Hyperoxyaluria: Understanding

Primary Hyperoxyaluria: Overview

Primary hyperoxaluria type 1 (PH1) is a rare, autosomal recessive disorder characterized by the deficiency of the hepatic enzyme alanine-glyoxylate aminotransferase, leading to the excessive production of oxalate within the body. This condition results in the excretion of oxalate in urine and the accumulation of calcium oxalate in various organs. PH1 is the most common and severe form of primary hyperoxaluria, with an estimated prevalence of 1 to 3 cases per 1 million population and an incidence rate of approximately 1 case per 120,000 live births per year in Europe. Primary hyperoxaluria is a rare genetic condition characterized by the overproduction of oxalate in the body, leading to the formation of kidney stones and potential kidney damage. Symptoms of primary hyperoxaluria include sharp pain in the back, side, lower stomach area, or groin, discolored urine (pink, red, or brown), frequent urination with pain, difficulty urinating, and accompanying symptoms like chills, fever, upset stomach, or vomiting. If left untreated, this condition can progress to kidney stones, kidney damage, and ultimately kidney failure.

Primary hyperoxaluria is a rare genetic disorder characterized by defective glyoxylate metabolism, leading to excessive oxalate production. This excess oxalate is deposited as insoluble calcium oxalate salts in the kidneys and systemically, affecting various organs like the retina, myocardium, blood vessel walls, skin, and bones. Birefringent oxalate crystals are observed in vessel walls and connective tissues of affected organs. The condition is caused by mutations in genes like AGXT (type 1), GRHPR (type 2), and HOGA1 (type 3). Primary hyperoxaluria can result in kidney stones, renal and bladder stones, and systemic oxalosis if not managed effectively.

The diagnosis of primary hyperoxaluria involves genetic studies to definitively identify the condition. If genetic testing is inconclusive, a liver biopsy may be performed to confirm the diagnosis. In cases where the disease progresses to end-stage renal disease (ESRD), diagnosis can be made based on oxalate deposits observed on renal biopsy or recurrent oxalate nephropathy in a renal allograft. Due to the rarity of primary hyperoxaluria, diagnosis can be challenging, but tests such as genetic studies and liver biopsy play a crucial role in confirming the presence of this condition.

The treatment for primary hyperoxaluria involves a multifaceted approach to reduce oxalate accumulation and manage complications. This includes medications like lumasiran to lower oxalate production rapidly, pyridoxine to decrease urinary oxalate excretion, and potassium citrate or pyrophosphate to alkalinize urine and prevent stone formation. Procedures such as gastrostomy and IV fluids may be necessary, while dialysis can help remove excess oxalate from the blood. In cases of primary hyperoxaluria type 1, liver transplantation is often the only curative option, restoring the deficient enzyme activity. Supportive measures like high fluid intake, dietary modifications, and avoiding certain vitamins are essential components of managing this rare genetic disorder effectively.

'Primary Hyperoxyaluria- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Primary Hyperoxyaluria pipeline landscape is provided which includes the disease overview and Primary Hyperoxyaluria treatment guidelines. The assessment part of the report embraces, in depth Primary Hyperoxyaluria commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Primary Hyperoxyaluria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Primary Hyperoxyaluria R&D. The therapies under development are focused on novel approaches to treat/improve Primary Hyperoxyaluria.

Primary Hyperoxyaluria Emerging Drugs Chapters

This segment of the Primary Hyperoxyaluria report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Primary Hyperoxyaluria Emerging Drugs

Oxabact: OxThera

To address Primary Hyperoxaluria (PH), OxThera has taken a bacterial strain isolated from the human gut and developed a live biotherapeutic product candidate. Oxalobacter formigenes is an anaerobe, non-pathogenic bacterium which exclusively uses oxalate as the energy source. Administration of the bacteria at pharmacological doses to PH patients is hypothezised to promote active, incremental secretion of oxalate into the gut where it is subject to degradation and enteric elimination. This highly innovative approach reroutes oxalate elimination to the gut, thereby reducing the high oxalate burden on the kidneys. Oxabact is being evalauated in Phase III stage of development for the treatment of Primary Hyperoxaluria.

Primary Hyperoxyaluria: Therapeutic Assessment

This segment of the report provides insights about the different Primary Hyperoxyaluria drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Primary Hyperoxyaluria

• There are approx. 6+ key companies which are developing the therapies for Primary Hyperoxyaluria. The companies which have their Primary Hyperoxyaluria drug candidates in the most advanced stage, i.e. Phase III include, OxThera.

Phases

The report covers around 6+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Primary Hyperoxyaluria pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Primary Hyperoxyaluria: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Primary Hyperoxyaluria therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Primary Hyperoxyaluria drugs.

Primary Hyperoxyaluria Report Insights

• Primary Hyperoxyaluria Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Primary Hyperoxyaluria Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Primary Hyperoxyaluria drugs?
• How many Primary Hyperoxyaluria drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Primary Hyperoxyaluria?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Primary Hyperoxyaluria therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Primary Hyperoxyaluria and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Precision Biosciences
• Chinook Therapeutics
• BridgeBio
• OxThera

Key Products

• PBGENE PH1
• CHK 336
• BBP 711
• Oxabact

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