by The global 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 (PKU) is a genetic disorder characterized by the body’s inability to break down an essential amino acid called phenylalanine. This inability to metabolize phenylalanine leads to accumulation of high levels of phenylalanine and its byproducts in the blood. If left untreated, it can cause intellectual disability, seizures, behavioral problems, and other issues. The standard treatment involves restricting dietary intake of phenylalanine and supplementing with a phenylalanine-free medical formula. Products in this market include medical foods like formulas and supplements to help manage phenylalanine levels as well as drug therapies to treat PKU symptoms and complications. Early diagnosis and lifelong treatment are imperative to prevent intellectual disabilities associated with high phenylalanine levels.
Market Key Trends:
One of the key trends in the PKU treatment market is rising research and development activities for new therapeutic products. Several biopharmaceutical companies are conducting clinical trials for new drug formulations that could potentially overcome limitations of existing treatments. For instance, BioMarin Pharmaceuticals is developing an investigational gene therapy called valoctocogene roxaparvovec which aims to restore the PAH enzyme activity and reduce dietary phenylalanine restrictions. Companies are also developing novel drug delivery methods like oral pills and extended-release injections to enhance patient compliance over traditional medical foods. Additionally, advancing newborn screening programs globally are facilitating early diagnosis of PKU, thereby driving the adoption of treatment options. However, high cost of therapies continues to hamper widespread access in developing regions.
Porter’s Analysis
Threat of new entrants: The risk of new entrants entering the phenylketonuria treatment market is low due to high capital requirements for R&D and manufacturing.
Bargaining power of buyers: The bargaining power of buyers is high due to availability of substitutes and concentrated customer base of patients.
Bargaining power of suppliers: The bargaining power of suppliers is moderate as raw material suppliers have a limited control over pricing but can impact if they collectively bargain.
Threat of new substitutes: The threat of substitutes is moderate as new novel treatment alternatives constantly being researched but proving efficacy and safety takes significant time.
Competitive rivalry: High due to presence of major players focusing on new product launches and expansion.
Key Takeaways
The global Phenylketonuria Treatment Market Size is expected to witness high growth, exhibiting CAGR of 8.6% over the forecast period, due to increasing awareness initiatives for rare diseases by key players and government bodies.
The market size for 2023 is US$ 1 Bn. By 2030, the market is expected to reach US$ 1.8 Bn, increasing at a CAGR of 8.6% during the forecast period.
North America dominates the global phenylketonuria treatment market and is expected to maintain its dominance over the forecast period. This is attributed to growing research on novel treatment therapies and supportive government policies for rare disease treatments in the US.
Key players operating in the phenylketonuria treatment market are Cigna, BioMarin Pharmaceuticals, Codexis, Inc., Erytech Pharma, SOM Innovation Biotech SL, Synthetic Biologics, Inc., and Retrophin, Inc. Major players are focusing on new product launches and expansion strategies to strengthen their market position.
