Global Primary Hyperoxaluria Treatment Market Report and Forecast 2024-2032

Global Primary Hyperoxaluria Treatment Market Outlook

The global primary hyperoxaluria treatment market size reached a value of about USD 18.91 million in 2023 and is expected to grow at a CAGR of 3.7% during the forecast period of 2024-2032. With the growing awareness of rare disease and genetic engineering advancement, the market is expected to reach a value of about USD 26.16 million by 2032.

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Primary Hyperoxaluria Insights

Primary hyperoxaluria (PH) is a rare genetic disease caused by glyoxylate metabolism deficiencies. The PH is autosomal recessive and is classified into three types based on the responsible genes: type 1 (AGXT) (PH-I), type 2 (GRHPR) (PH-II), and type 3 (HOGA1) (PH-III). The kidney is the primary site of oxalate deposition, which in many cases leads to end-stage renal disease.

End stage renal disease (ESRD) is a life-threatening condition that prevents the kidneys from effectively filtering fluids and waste products from the body.

Kidney stones in primary hyperoxaluria type 1 typically appear between childhood and early adulthood, and ESRD can develop at any age. The symptoms of primary hyperoxaluria type 2 are similar to those of type 1, but ESRD develops later in life. Primary hyperoxaluria type 3 patients frequently develop kidney stones in childhood, but few cases of this type have been described, so additional signs and symptoms are unknown.

Recurrent kidney stones, hematuria, and urinary tract infections (UTIs) are possible signs and symptoms. If left untreated, PH-I can progress to end-stage renal disease, which can be fatal. The disease is characterised phenotypically by the recurrent formation of kidney stones and nephrocalcinosis, which results in the gradual decline of kidney function. When renal function deteriorates, excess oxalate cannot be eliminated through the urine, and calcium oxalate crystals precipitate in various tissues, resulting in systemic oxalosis, a devastating multiorgan disease.

Changes (mutations) in the AGXT gene cause PH type I. Mutations in the GRHPR gene cause PH type II. Mutations in the HOGA1 gene cause PH type III. Genes encode instructions for the production of proteins, which are essential for many bodily functions. When a gene is mutated, the protein product may be faulty, inefficient, or absent. Depending on the functions of the specific protein, this can have an impact on many organ systems in the body.

According to the market research report, the primary hyperoxaluria treatment market can be categorised into the following segments:

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Market Breakup by Type

• Primary Hyperoxaluria Type I

• Primary Hyperoxaluria Type II

• Primary Hyperoxaluria Type II

Market Breakup by Diagnosis Method

• Urine Tests

• Blood Tests

• Stone Analysis

• Kidney X-rays, Ultrasound or Computerized Tomography (CT) Scan

• DNA Testing

• Kidney Biopsy

• Echocardiogram

• Eye Exam

• Bone Marrow Biopsy

• Liver Biopsy

Market Breakup by Treatment Method

• Medications
  • Potassium Citrate
  • Thiazide Diuretics
  • Vitamin Supplements
  • Lumasiran
• High Fluid Intake

• Dietary Changes

• Kidney Stone Management

• Dialysis and Transplantation

Market Breakup by Treatment Channel

• Public

• Private

Market Breakup by Region

• North America
  • United States of America
  • Canada
• Europe
  • United Kingdom
  • Germany
  • France
  • Italy
  • Others
• Asia Pacific
  • China
  • Japan
  • India
  • ASEAN
  • Australia
  • Others
• Latin America
  • Brazil
  • Argentina
  • Mexico
  • Others
• Middle East and Africa
  • Saudi Arabia
  • United Arab Emirates
  • Nigeria
  • South Africa
  • Others

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Primary Hyperoxaluria Epidemiology

Males and females are equally affected by PH. These disorder’s exact incidence and prevalence are unknown. Because some cases go undiagnosed or are misdiagnosed, it is difficult to determine the true prevalence of these conditions in the general population. Young patients with recurring kidney stone formation may benefit from molecular genetic testing. However, some patients with common PH symptoms are not found to have the known genetic mutations linked to PH, implying that the true number of people with PH is likely to be underestimated. The most common form is PH type I. 

According to one estimate, the prevalence of PH type I in the general population is 1-3 cases per 1,000,000 people, with fewer than 1,000 people affected in the United States, and the incidence is 1 case per 120,000 live births per year in Europe. PH is estimated to be 2.5 times more common in European Americans than in African Americans.

Global Primary Hyperoxaluria Treatment Market Drivers and Restraints

The population growing awareness of rare and genetic disorders is expected to drive the demand for primary hyperoxaluria treatment. During the forecast period, technological advancements in the field of genetic engineering will drive the demand for global primary hyperoxaluria treatment market.

The number of leading pharmaceutical companies with products in clinical trials for the treatment of primary hyperoxaluria will drive the market growth. In the coming decade, demand for primary hyperoxaluria treatment will rise due to an increase in diagnosis and treatment seeking. Global primary hyperoxaluria treatment market growth is hampered by a small patient population available for clinical trials.

Global Primary Hyperoxaluria Therapeutic Landscape

Treatment is determined by the type, symptoms, and severity of hyperoxaluria, and by how well the patient responds to the treatment.

Treatment may necessitate the collaborative efforts of a team of specialists. Pediatricians, surgeons, specialists who assess and treat kidney problems (nephrologists), specialists who assess and treat liver problems (hepatologists), specialists who assess and treat urinary tract problems (urologists), dieticians, and other healthcare professionals may need to plan an affected child's treatment systematically and comprehensively. Genetic counselling is advised to assist families in understanding the genetics and natural history of PH, and to provide psychosocial support. 

Lumasiran (Oxlumo) is a medication that reduces oxalate production in children and adults with primary hyperoxaluria. In some people with primary hyperoxaluria, prescription doses of vitamin B-6 can help reduce oxalate in the urine. Phosphate and citrate oral preparations aid in the prevention of calcium oxalate crystal formation. Depending on the other abnormalities in the urine, other medications, such as thiazide diuretics, may be considered.

Global Primary Hyperoxaluria Competitive Landscape

The report gives an in-depth analysis of the key players involved in the global primary hyperoxaluria treatment market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows: 

• Alnylam Pharmaceuticals

• Tecoland Corporation

• Zhejiang Tianxin Pharmaceuticals Co.

• Takeda Pharmaceuticals

• Wuxi Further Pharmaceutical Co Ltd

• Genentech

• OxThera

• Dicerna Pharmaceuticals, Inc.

• Allena Pharmaceuticals

Key Highlights of the Report