The Phenylketonuria Treatment Market is estimated to be valued at US$ 1 Bn in 2023 and is expected to exhibit a CAGR of 8.6% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Market Overview:
Phenylketonuria or PKU is a genetic disease affecting protein metabolism. It causes excess levels of phenylalanine in the blood due to a deficiency in the enzyme phenylalanine hydroxylase. If left untreated, it can lead to intellectual and developmental disabilities. The treatment options available include dietary restrictions to limit phenylalanine intake and medication. Dietary treatment involves consuming special low-protein foods and avoiding high-protein foods. Medications include sapropterin tablets and powder that help reduce phenylalanine levels. Gene therapy is also being explored as a promising treatment approach.
Market Dynamics:
One of the major drivers fueling growth in the phenylketonuria treatment market is the rising cases of PKU globally. According to research, PKU affects about 1 in 10,000 to 30,000 newborns in the U.S. each year. Early diagnosis and prompt treatment is helping reduce associated health issues. Furthermore, growing awareness initiatives by patient advocacy groups and increasing research activity in gene therapies are also contributing to market expansion. However, strict dietary compliance required for lifelong treatment continues to pose a challenge. Additionally, the high treatment costs further limit broader access and adoption in developing regions.
Segment Analysis
The global Phenylketonuria Treatment Market Size is dominated by the drugs segment which has a market share of over 50%. This is because drugs remain the preferred line of treatment for phenylketonuria. Among drugs, the dominant sub-segment is Kuvan which is prescribed for the treatment of phenylketonuria in patients age 16 years and older. This drug has the largest patient pool and demand which has helped it dominate the global market.
PEST Analysis
Political:Healthcare policies favoring orphan drug development and approvals have positively impacted the phenylketonuria treatment market.
Economic: Rising healthcare spending globally is facilitating market growth. The market also benefits from tax incentives for orphan drug development.
Social: Increased awareness about rare diseases and availability of diagnosis & treatment options are driving the market.
Technological: Advancements in screening technologies for newborns help early diagnosis improving treatment outcomes. Novel drug delivery mechanisms are under development.
Key Takeaways
The global phenylketonuria treatment market is expected to witness high growth, exhibiting CAGR of 8.6% over the forecast period, due to increasing awareness about rare diseases and improving access to diagnosis and treatment. The market size for phenylketonuria treatment is US$ 1 Bn in 2023.
Europe currently dominates the market followed by North America. This is because of mandatory newborn screening programmes and favorable reimbursements. However, Asia Pacific is expected to witness fastest growth due to rising healthcare spending, increasing patient awareness and expanding healthcare facilities.
Key players operating in the phenylketonuria treatment market are Cigna, BioMarin Pharmaceuticals, Codexis, Inc., Erytech Pharma, SOM Innovation Biotech SL, Synthetic Biologics, Inc., Retrophin, Inc., Danone. Major players are focusing on expanding their drug portfolios through acquisitions and collaborative research efforts to strengthen their market position.
