Phenylketonuria Treatment Market to Flourish Owing to Rising Awareness Regarding Phenylketonuria Disorders
Phenylketonuria or PKU is a genetic disorder characterized by the body’s inability to breakdown phenylalanine, an essential amino acid obtained from diet. If left untreated, the accumulated phenylalanine and its byproducts can cause intellectual disabilities and neurological dysfunction. Treatment involves adhering to a low-protein, phenylalanine-restricted diet throughout life and medication supplements. The global phenylketonuria treatment market includes drugs for reducing or replacing dietary phenylalanine intake such as Kuvan and Palynziq. These specialty medications help in the management of phenylketonuria and improve patients’ quality of life.
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 Dynamics:
Rising awareness regarding phenylketonuria disorders and availability of effective treatment options are driving the growth of the global phenylketonuria treatment market. According to the National Institutes of Health (NIH), an estimated 1 in every 15,000 babies in the United States is born with phenylketonuria. Growing patient population with phenylketonuria and innovation in treatment therapies such as gene therapy are further fueling the market growth. For instance, in October 2020, Homology Medicines Inc. received orphan drug designation from the US FDA for its PKU gene therapy HMI-102. In addition, favorable reimbursement policies in developed economies for phenylketonuria treatment drugs are positively impacting the market growth. However, high costs associated with phenylketonuria treatments may hamper the market growth over the forecast period.
SWOT Analysis
Strength:
– Increasing awareness about PKU treatment and management is driving the market growth. Regular dietary treatment and medications aids in avoiding long term complications of PKU.
– Availability of expanded newborn screening programs for early diagnosis of PKU is helping patients receive timely treatment.
– Research and development activities are leading to the development of novel drugs and treatment options for better management of PKU.
Weakness:
– High cost associated with PKU treatment poses a major limitation particularly in low and middle-income countries.
– Lack of disease-modifying drugs and options for adults with PKU. Existing therapies only control symptoms but do not cure the condition.
Opportunity:
– Growing healthcare expenditure in emerging nations has opened up opportunities for market players to offer affordable treatment options.
– Development of gene therapies holds potential to provide a complete cure for PKU in the future.
Threats:
– Stringent regulatory processes for the approval of new drug entities increases the time and resources required to bring treatments to market.
– Challenge of developing cost-effective drug formulations suitable for all age groups of PKU patients.
Key Takeaways
The global Phenylketonuria Treatment market is expected to witness high growth during the forecast period of 2023 to 2030. The market size for 2023 is estimated to be US$ 1 Bn and is anticipated to grow at a CAGR of 8.6% during the forecast period.
Regional analysis: North America currently dominates the global market due to large patient population, high adoption of innovative therapies, and rapid uptake of advanced treatment options in the region. However, Asia Pacific is expected to grow at the fastest pace over the forecast period owing to rising healthcare investment, expanding healthcare infrastructure and increasing medical needs of large patient population in China and India.
Key players: Key players operating in the Phenylketonuria Treatment market are BioMarin Pharmaceuticals Inc., Censa Pharmaceutical, Synlogic Inc., Erytech Pharma SA, Codexis Inc., SOM Innovation Biotech SL, Homology Medicines Inc., and Ultragenyx (Dimension Therapeutics). These companies are involved in continual research and development activities to introduce novel treatment options for better management of PKU.
