Phenylketonuria (PKU) is an inherited disorder caused due to the body’s inability to break down an amino acid called phenylalanine. This amino acid is commonly found in most foods. Phenylketonuria can cause intellectual disability or other neurological problems if not treated properly. Dietary enzymes, gene therapies and drug alternatives are commonly used to treat PKU by restricting phenylalanine intake. The global Phenylketonuria Treatment Market highly relies on dietary restriction and special formula foods that are low in phenylalanine. Patients afflicted by PKU are prescribed medical foods as substitutes for normal protein foods to prevent toxic buildup of phenylalanine in their bodies. Increased focus on screening programs for newborns and better diagnosis methods is boosting the demand for such customized foods and therapeutics.
The global Phenylketonuria Treatment Market is estimated to be valued at US$ 2.62 billion 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.
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Market key trends:
Gene therapy and enzyme substitution are among the emerging trends in PKU treatments. Several clinical trials are underway to develop gene therapies that can replace dysfunctional liver enzymes and naturally reduce phenylalanine levels. For instance, phase 3 clinical trials are evaluating the efficacy and safety of DXS-5201, an investigational adeno-associated virus (AAV) gene therapy from Dimension Therapeutics, to treat PAH deficiency. Other companies like Synlogic and Homology Medicines are also developing gene therapies through bacterial enzymes that could provide safer and long-lasting treatments for PKU. These novel therapies are expected to revolutionize PKU management over the next decade.
Threat of new entrants: The threat of new entrants is moderate. High capital investment requirements and stringent regulations act as a barrier for new players. However, the potential for high growth attracts new companies.
Bargaining power of buyers: The bargaining power of buyers is high. The availability of substitutes increases the buyer options. Buyers can negotiate on price and purchase large volumes to get better deals.
Bargaining power of suppliers: The bargaining power of suppliers is moderate due to the diverse base of raw material suppliers. However, suppliers of specific ingredients have some control over pricing.
Threat of new substitutes: The threat of substitute products is moderate. Alternate therapies and new drug delivery options pose a threat. However, customer loyalty and regulatory approvals limit immediate substitution.
Competitive rivalry: The competition is high among existing players. Players compete based on product innovation, pricing, and personalized treatment programs.
The global Phenylketonuria Treatment market is expected to witness high growth. The global Phenylketonuria Treatment Market is estimated to be valued at US$ 2.62 billion in 2023 and is expected to exhibit a CAGR of 8.6% over the forecast period 2023 to 2030.
Regional analysis suggests North America will dominate the market during the forecast period due to increasing diagnosis rate, advanced healthcare infrastructure, and presence of leading market players. Europe is also expected to grow significantly due to growing adoption of screening programs and availability of PKU treatment options.
Key players comprised: Key players operating in the Phenylketonuria Treatment market are BioMarin Pharmaceutical Inc., Cambrooke Therapeutics, Codexis Inc., Homology Medicines, Inc., and SOM Biotech SL.
- Source: Coherent Market Insights, Public sources, Desk research
- We have leveraged AI tools to mine information and compile it