The UK Primary Hyperoxaluria Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
Primary Hyperoxaluria (PH) is an ultra-orphan disease caused by genetic mutations, which results in the build-up of overproduction and accumulation of oxalate in the body. This results in the deposition of calcium oxalate crystals in the kidneys and urinary tract, resulting in urolithiasis, nephrocalcinosis, and ultimately kidney failure. As a result of systemic oxalosis, multi-organ damage occurs which affects bones, eyes, skin, and the heart.
Affected individuals lack functional levels of a specific enzyme that normally prevents the accumulation of oxalate. In the kidneys, excess oxalate binds with calcium to form a hard compound (calcium oxalate) that is the main component of kidney and urinary stones. Common symptoms include the formation of stones throughout the urinary tract (urolithiasis) and kidneys (nephrolithiasis) and progressively increased levels of calcium in the kidneys (nephrocalcinosis).
There are three main types of PH that are inherited in an autosomal recessive pattern: PH1, PH2, and PH3. PH1 is the most severe primary hyperoxaluria and accounts for approximately 80% of cases. According to a study, primary hyperoxaluria has an estimated prevalence of 1 to 3 cases per 1 million population and an incidence rate of approximately 120,000 live births per year. It accounts for 1-2% of pediatric end-stage kidney disease but higher values are reported in specific populations with a high rate of consanguinity. The UK-based National Renal Rare Disease Registry (RaDaR) suggests there are approximately more than 100 patients across all the hospitals who have hyperoxaluria.
The increasing prevalence of primary hyperoxaluria disease is one of the major market drivers. However, with rising awareness and increased diagnostic facilities, demand for primary hyperoxaluria drugs is expected to be triggered over the forecast period. High investment in research & development by leading industry participants is expected to open a new growth window for them. With strong research and development initiatives, most of the companies are focusing on innovative and cost-effective drug development
An increase in special designation from the regulatory authority is propelling the growth of the PH market. Huge financial support to the researchers for developing novel interventions is boosting the market growth. The high demand for disease-specific novel treatment can also act as a market driver. The competitive scenario of the market and strategic collaborations may also boost the market position during the forecast period
The high cost of treatment for primary hyperoxaluria acts as a major restraint for the market growth. Additionally, limited operating revenue opportunities for research and development of targeted therapies by many market players will be considered as a challenging factor for the growth of the PH market.