The global phenylketonuria therapeutics market is expected to garner a market value of USD 1 billion in 2023 and is expected to accumulate a market value of USD 2.28 billion by registering a CAGR of 8.6% in the forecast period 2023 to 2033. The market for Phenylketonuria Therapeutics registered a CAGR of 5.2% in the historical period 2018 to 2022.
Phenylketonuria (PKU) is a rare inherited metabolic disorder that results in the inability of the body to break down an amino acid called phenylalanine. This leads to a buildup of phenylalanine in the blood, which can cause severe brain damage and other neurological problems if left untreated.
The current treatment for PKU involves a strict low-phenylalanine diet, which can be difficult to adhere to and often requires the use of specialized medical foods and supplements. In addition to dietary management, there are also pharmacological treatments available for PKU, including sapropterin dihydrochloride, which is a synthetic form of tetrahydrobiopterin (BH4), a co-factor required for phenylalanine metabolism.
The global market for PKU therapeutics is projected to grow in the coming years, driven by an increasing prevalence of PKU and a growing awareness of the disorder among healthcare professionals and patients.
| Report Attribute | Details |
|---|---|
| Expected Market Value (2023) | USD 1 billion |
| Anticipated Forecast Value (2033) | USD 2.28 billion |
| Projected Growth Rate (2023 to 2033) | 8.6% CAGR |
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According to market research and competitive intelligence provider, Future Market Insights- the market for phenylketonuria therapeutics reflected a value of 5.2% during the historical period, 2018 to 2022. One of the key drivers of market growth is the increasing prevalence of PKU worldwide. According to a report by the National Organization for Rare Disorders, PKU affects approximately one in every 10,000 to 15,000 newborns worldwide. This number is expected to increase due to factors such as increased genetic testing and newborn screening.
Another factor contributing to market growth is the increasing awareness of PKU among healthcare professionals and patients. As awareness of the disorder grows, there is a growing demand for effective treatments that can improve patient outcomes and quality of life.
In addition, the market is driven by the introduction of novel therapies and enhancements to existing products. Pharmaceutical companies are investing heavily in research and development to introduce new treatments and improve existing ones. Overall, the market for phenylketonuria therapeutics is expected to register a CAGR of 8.6% in the forecast period 2023 to 2033.
Increasing Prevalence of Phenylketonuria to Push Market Growth
Phenylketonuria (PKU) is a rare genetic disorder that affects the body's ability to process the amino acid phenylalanine. If left untreated, it can cause intellectual disability and other health problems. As a result, the demand for effective treatments for the disorder is growing, and this is one of the most prominent drivers of the Phenylketonuria therapeutics market.
Another key driver is the technological advancements in PKU diagnostics. These advancements have led to earlier and more accurate diagnosis of the disorder, which has increased demand for PKU therapeutics. Early diagnosis can also help prevent the development of more severe symptoms of the disorder.
In addition to this, governments around the world are taking initiatives to raise awareness about PKU and to fund research and development of effective therapeutics for the disorder. This has helped to create a more favorable regulatory environment, as well as increase funding for research and development. The growing support for PKU research and development is also driving the demand for new and innovative treatments for the disorder.
Finally, the increasing prevalence of PKU is also a driver of the Phenylketonuria therapeutics market. The disorder is becoming more common worldwide, which has led to an increase in the number of patients seeking treatment for the condition. As more patients are diagnosed with PKU, the demand for effective therapeutics will continue to grow.
In conclusion, the Phenylketonuria therapeutics market is being driven by a combination of factors, including the increasing prevalence of PKU, technological advancements in PKU diagnostics, government initiatives and funding, and growing demand for effective treatments for the disorder. These drivers are expected to continue to fuel growth in the PKU therapeutics market in the coming years.
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Expensive Cost of Treatment to restrict Market Growth
One of the significant challenges faced by the PKU therapeutics market is the lack of awareness and underdiagnosis of the disorder. Many patients with PKU are undiagnosed or misdiagnosed, which can lead to delayed treatment and poor health outcomes.
Moreover, the cost of PKU treatment can be high, making it difficult for patients and families to afford. The cost of specialized diets, medical foods, and supplements can add up quickly, and many insurance plans do not cover the full cost of treatment. Currently, there are limited treatment options available for PKU, and many patients do not respond well to existing treatments. This has led to a growing need for new and more effective treatments.
The phenylketonuria therapeutics market faces several challenges, including limited awareness and diagnosis, high cost of treatment, limited treatment options, compliance issues, and social stigma and isolation. Addressing these challenges will require collaboration among patients, healthcare providers, researchers, and policymakers to improve diagnosis, develop more affordable and effective treatments, and provide support and resources for patients and their families.
Increasing awareness and diagnosis of the condition Shaping Landscape for Phenylketonuria Therapeutics in South & East Asia
The phenylketonuria (PKU) therapeutics market in the South & East Asia region is still developing, but there is a growing demand for effective treatments for PKU. PKU is a rare disorder, and the prevalence of the condition varies widely across the region. However, with the increasing availability of advanced diagnostic tools and increasing awareness about PKU, the market for PKU therapeutics is expected to grow in the region.
One of the key factors driving the growth of the PKU therapeutics market in the South & East Asia region is the increasing number of diagnosed patients with PKU. As healthcare infrastructure in the region continues to improve, more patients are being diagnosed with the condition, creating a growing demand for PKU therapeutics.
The cost of PKU treatment is also a significant factor in the South & East Asia region. In many countries, the cost of PKU treatment can be high, and access to specialized medical foods and supplements can be limited. However, with increasing awareness about the disorder, more government and private organizations are coming forward to support patients and improve access to treatment.
Another factor driving the growth of the PKU therapeutics market in the region is the increasing number of research studies and clinical trials focused on PKU. These studies are contributing to the development of new and innovative treatments for PKU, which is expected to expand the treatment options available for patients in the region. In conclusion, the PKU therapeutics market in the South & East Asia region is still developing, but there is a growing demand for effective treatments.
Increasing Focus on Technological Advancements shaping the landscape for Phenylketonuria Therapeutics in North America
The Phenylketonuria Therapeutics market in North America is expected to experience significant growth in the coming years, driven by several factors. One of the primary drivers of market growth is the high prevalence of underlying medical conditions that can lead to Phenylketonuria. These conditions are becoming increasingly common in North America, particularly among the aging population, and are contributing to a growing demand for innovative and effective treatments for Phenylketonuria.
In addition to the increasing prevalence of underlying medical conditions, the market is also being fueled by advances in technology and research. New diagnostic tools and treatment options are being developed, which are expected to improve outcomes for patients with Phenylketonuria.
Another key factor driving the phenylketonuria therapeutics market in North America is the high level of awareness and understanding of the condition among healthcare providers and the general public. This has led to increased diagnosis rates and better management of the condition, which is contributing to improved patient outcomes.
Finally, the presence of well-established healthcare infrastructure and a high level of investment in research and development in North America are also expected to boost market growth. This has created a favorable environment for the development and commercialization of new and innovative products and treatments for Phenylketonuria.
Overall, the phenylketonuria therapeutics market in North America is poised for significant growth in the coming years, expected to accumulate 40% of global revenues in 2023. This growth is likely to be driven by a range of factors including increasing prevalence of underlying medical conditions, advances in technology and research, high levels of awareness and understanding, and a favorable healthcare infrastructure and investment environment.
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Parenteral route of administration to hold a significant share and push market growth
The injectable route of administration is likely to account for a significant share of the Phenylketonuria Therapeutics market. This is due to the effectiveness of parenteral treatments, which can be administered directly into the bloodstream or affected area for faster and more targeted delivery of medication.
Parenteral treatments are also preferred in cases where oral medications may not be suitable, such as for patients with difficulty swallowing or those who require higher doses that may not be feasible with oral medications. In addition, several established parenteral treatments for Phenylketonuria are already available on the market, which further contributes to the dominance of the parenteral segment.
Hospital pharmacies segment to hold a significant share and push market growth
Hospitals and specialty clinics are often the first point of contact for patients with Phenylketonuria, as they provide specialized care and access to diagnostic tools and treatments. These facilities also have the expertise and resources to provide comprehensive care to patients with complex or severe cases of Phenylketonuria.
In addition, many of the treatments for Phenylketonuria require specialized administration or monitoring, such as intravitreal injections or electroretinography testing, which are typically performed in hospitals or specialty clinics. This further contributes to the dominance of these distribution channels in the Phenylketonuria Therapeutics market.
Overall, hospitals and specialty clinics are likely to continue to account for a significant share of the Phenylketonuria Therapeutics market, driven by the specialized care and expertise they provide. However, as patient preferences and healthcare delivery models continue to evolve, the market share of the various distribution channels may shift over time.
Key players in the market include companies such as Competition Deep Dive, Cigna, BioMarin Pharmaceuticals, Codexis, Inc., Erytech Pharma, SOM Innovation Biotech SL, Synthetic Biologics, Inc., Retrophin, Inc., Danone, Censa Pharmaceuticals, Homology Medicines, Inc., along with healthcare providers and technology companies among other global players.
| Report Attribute | Details |
|---|---|
| Market Value in 2023 | USD 1 billion |
| Market Value in 2033 | USD 2.28 billion |
| Growth Rate | CAGR of 8.6% from 2023 to 2033 |
| Base Year for Estimation | 2022 |
| Historical Data | 2018 to 2022 |
| Forecast Period | 2023 to 2033 |
| Quantitative Units | Revenue in USD Billion and CAGR from 2023 to 2033 |
| Report Coverage | Revenue Forecast, Volume Forecast, Company Ranking, Competitive Landscape, Growth Factors, Trends, and Pricing Analysis |
| Segments Covered | Route of Administration, Distribution Channel, Drug Class, Region |
| Regions Covered | North America; Latin America; Europe; South Asia; East Asia; Oceania; Middle East & Africa |
| Key Countries Profiled |
United States, Canada, Brazil, Mexico, Germany, United Kingdom, France, Spain, Italy, India, Malaysia, Singapore, Thailand, China, Japan, South Korea, Australia, New Zealand, GCC Countries, South Africa, Israel |
| Key Companies Profiled | The Cigna Group; BioMarin Pharmaceutical Inc.; Codexis Inc.; Erytech Pharma S.A; SOM Innovation Biotech S.A; Synthetic Biologics, Inc. (Theriva); Retrophin Inc. (Travere Therapeutics); Danone S.A.; Censa Pharmaceuticals (PTC Therapeutics); Homology Medicines Inc. |
| Customization | Available Upon Request |
In 2023, the overall market is estimated to generate a revenue sum of nearly USD 1 billion.
The injectable route of administration segment generates higher revenue for the global market.
The region is expected to account for 40% global revenue due to the presence of many leading market players.
The overall regional market has a limited share but is expected to grow at a faster rate through 2033.
Cigna, BioMarin, and Codexis are known to be the leading market players.
1. Executive Summary 2. Market Overview 3. Market Background 4. Global Market Analysis 2018 to 2022 and Forecast, 2023 to 2033 5. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Drug Type 5.1. Kuvan 5.2. Palynziq 6. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Route of Administration 6.1. Oral 6.2. Parenteral 6.3. Intravenous 6.4. Subcutaneous 7. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Distribution Channel 7.1. Hospitals Pharmacies 7.2. Retail Pharmacies 7.3. Online Pharmacies 8. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Region 8.1. North America 8.2. Latin America 8.3. Europe 8.4. South Asia 8.5. East Asia 8.6. Oceania 8.7. Middle East & Africa 9. North America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 10. Latin America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 11. Europe Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 12. South Asia Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 13. East Asia Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 14. Oceania Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 15. Middle East & Africa Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 16. Key Countries Market Analysis 17. Market Structure Analysis 18. Competition Analysis 18.1. Cigna 18.2. BioMarin Pharmaceutical Inc. 18.3. Codexis, Inc. 18.4. Erytech Pharma S.A 18.5. SOM Innovation Biotech S.A 18.6. Synthetic Biologics, Inc. (Theriva) 18.7. Retrophin, Inc. 18.8. Danone S.A 18.9. Censa Pharmaceuticals (PTC Therapeutics) 18.10. Homology Medicines, Inc. 19. Assumptions & Acronyms Used 20. Research Methodology
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Phenylketonuria Therapeutics Market
