Phenylketonuria Treatment Market by Treatment Type (Gene Therapy & Enzyme Substitution Therapies, Nutritional Supplements, Pharmacological Treatment), Route of Administration (Oral, Parenteral), Distribution Channel - Global Forecast 2025-2032
Description
The Phenylketonuria Treatment Market was valued at USD 929.31 million in 2024 and is projected to grow to USD 966.08 million in 2025, with a CAGR of 5.60%, reaching USD 1,437.78 million by 2032.
Comprehensive introduction to phenylketonuria treatment evolution contextualized within contemporary clinical pathways and patient-centered management considerations
Phenylketonuria (PKU) stands as a clinically and commercially significant inherited metabolic condition characterized by impaired metabolism of the amino acid phenylalanine. Advances in newborn screening, biochemical diagnostics, and lifelong management pathways have reshaped clinical trajectories, enabling earlier intervention and more precise therapeutic decision-making. Over recent years, therapeutic options have diversified beyond strict dietary regimens to include pharmacological agents, enzyme substitution approaches, and genetic modalities, each carrying distinct clinical profiles, administration requirements, and patient adherence implications.
Clinicians, caregivers, and health systems now must navigate an evolving evidence base that links biochemical control with neurocognitive and quality-of-life outcomes across pediatric and adult populations. This report provides an integrated perspective on current standards of care, emerging interventional strategies, and the complex interplay between clinical efficacy, delivery mechanisms, and real-world uptake. By framing treatment innovation within the operational realities of specialty supply chains, payer frameworks, and patient-centered care pathways, the analysis equips decision-makers with a coherent foundation for strategic planning and clinical program development.
Strategic overview of how scientific innovation, care-delivery modernization, and payer evolution are reshaping the future of phenylketonuria treatment access
The therapeutic landscape for phenylketonuria has entered a period of transformative change driven by converging scientific, regulatory, and care-delivery trends. Gene-centric approaches and enzyme substitution technologies are progressing through clinical development, promising mechanisms that target the root metabolic defect rather than solely mitigating downstream consequences. At the same time, pharmacological innovations have expanded treatment options for patients who do not achieve optimal biochemical control with dietary measures alone, and nutritional supplement formulations continue to improve tolerability and adherence.
These shifts are reinforced by technology-enabled care models, including digital adherence tools, telehealth-supported dietary management, and specialty pharmacy coordination, which collectively reduce barriers to consistent long-term management. Payer engagement has evolved in parallel, with value-based contracting discussions increasingly focused on clinical outcomes, patient-reported measures, and long-term cost offsets associated with improved neurocognitive trajectories. Consequently, stakeholders must align development strategies, commercial models, and care delivery designs to capture the clinical promise of innovation while addressing operational complexity and equitable access.
Practical analysis of how the United States tariff changes anticipated in 2025 could alter supply chain resilience, access pathways, and procurement strategies for PKU-related therapies
Anticipated tariff adjustments implemented in the United States in 2025 introduce a new layer of complexity for supply chains that support specialty therapies, medical foods, and laboratory reagents essential to phenylketonuria care. Many therapy components, including specialized proteins, reagents for diagnostic testing, and niche nutritional ingredients, rely on a global manufacturing footprint. Changes to import costs can therefore influence procurement strategies, push sourcing diversification, and catalyze nearshoring conversations among manufacturers seeking to preserve price stability and supply continuity.
Clinically, any reconfiguration of supply lines has the potential to create disparities in access if cost pressures translate into constrained distribution of high-cost therapies or interruptions in availability of specialized dietary products. Payers and health systems are likely to emphasize formulary resilience and contract language that guards against supply volatility. Strategically, biopharmaceutical developers and specialty manufacturers should accelerate supply chain mapping, including alternative sourcing, domestic manufacturing options, and inventory-buffer strategies, while engaging with policymakers to highlight the clinical ramifications of tariff-driven disruptions. In parallel, clinicians and patient advocacy groups will need proactive communication plans to manage expectations and support continuity of care during transitional periods.
Granular segmentation insights that connect treatment modalities, administration routes, age-based clinical pathways, and distribution channels to real-world access and adherence dynamics
A segmentation-focused lens clarifies how therapeutic uptake and care delivery differ across treatment modalities, routes of administration, age cohorts, and distribution pathways. Treatments that target the underlying defect, including gene therapy and enzyme substitution approaches, attract distinct development and commercialization pathways compared with nutritional supplements and pharmacological treatments, the latter category of which includes therapies such as pegvaliase and sapropterin dihydrochloride that address metabolic control through enzymatic replacement or cofactor modulation. Route of administration further differentiates patient and provider preferences, with oral therapies typically favored for ease of use and parenteral administrations requiring specialized clinic-based coordination or supportive home infusion models.
Age is a central determinant of clinical strategy and adherence design; pediatric populations face developmental and nutritional considerations that shape device selection, dosing strategies, and caregiver education, whereas adults contend with long-term adherence, comorbidity management, and transitions from pediatric to adult specialty care. Distribution channels likewise influence access and patient experience: hospital pharmacies and specialty clinics offer integrated clinical oversight for complex parenteral therapies and enzymatic treatments, while retail and online pharmacy models provide convenient refills for oral agents and nutraceuticals. Understanding these intersecting segments enables targeted clinical pathways, payer engagement strategies, and commercial models that reflect the operational realities of each patient cohort and product archetype.
Regional analysis highlighting how distinct regulatory environments, care infrastructures, and distribution capabilities shape phenylketonuria treatment pathways across major global territories
Regional dynamics exert a profound influence on regulatory pathways, reimbursement architectures, clinical practice norms, and supply chain logistics across the global phenylketonuria landscape. In the Americas, established newborn screening programs, concentrated specialty care centers, and an evolving payer environment create opportunities and constraints that shape therapy adoption patterns and patient management infrastructures. Stakeholders in this region must navigate a complex interplay of private and public payer requirements alongside robust patient advocacy networks that influence guideline development and access programs.
The Europe, Middle East & Africa region presents diverse regulatory frameworks and heterogenous health system capacities; centralized regulatory mechanisms coexist with country-level reimbursement heterogeneity, creating fragmentation that developers must address through phased approvals, localized evidence generation, and tailored access strategies. In the Asia-Pacific region, increasing investment in genetic and rare disease capabilities, expanding newborn screening coverage, and growing local manufacturing capacity are altering the supply and demand equation. Each regional bloc requires bespoke engagement plans that account for regulatory timelines, local clinical practice patterns, procurement norms, and the maturity of specialty distribution infrastructures.
Insightful synthesis of competitive strategies, partnership models, and operational enablers that define company positioning and program execution in the PKU treatment ecosystem
The competitive and collaborative landscape features a blend of established specialty pharmaceutical organizations, nimble biotechnology companies, diagnostic firms, and manufacturers of medical foods and nutritional supplements. Strategic activity increasingly centers on partnerships that combine clinical development expertise with manufacturing scale, as well as collaborations that integrate diagnostics with therapeutic delivery to enable precision-tailored interventions. Across the continuum, stakeholders prioritize evidence generation that demonstrates durable biochemical control, neurocognitive benefit, and meaningful patient-reported outcomes to support clinical adoption and payer engagement.
Operationally, companies investing in vertically integrated capabilities-spanning clinical manufacturing, cold-chain logistics for parenteral products, and specialized distribution-are better positioned to manage complexity and ensure continuity of supply. In parallel, commercial leaders emphasize robust patient support programs, specialty pharmacy relationships, and digital tools to optimize adherence and outcomes. Mergers, licensing deals, and strategic alliances continue to shape the innovation pipeline and route-to-market strategies, with an emphasis on technologies that reduce treatment burden and expand the pool of patients who can sustain long-term metabolic control.
Actionable and pragmatic recommendations for companies to strengthen evidence generation, supply resilience, distribution strategies, and patient-centric commercial models in PKU care
Industry leaders should adopt a multipronged approach that aligns clinical development priorities with pragmatic commercial and operational design. First, invest in evidence-generation frameworks that link biochemical endpoints to functional and cognitive outcomes, thereby strengthening value propositions for payers and clinicians. Next, prioritize supply chain resilience by diversifying sourcing, validating alternate suppliers for critical inputs, and investing in manufacturing redundancies or regional production capacities to mitigate tariff and geopolitical risks.
Additionally, companies should tailor product and service delivery to segmented patient needs, optimizing formulations and administration modalities for pediatric versus adult populations and coordinating distribution strategies that leverage both specialty clinic integration and patient-centric retail or online fulfillment. Engage proactively with payers through outcomes-based contracting pilots and real-world evidence programs that demonstrate clinical and economic impact. Finally, cultivate robust patient support ecosystems that combine clinical education, digital adherence tools, and coordinated specialty pharmacy services to reduce attrition and improve long-term outcomes. Executing these priorities will require cross-functional alignment across R&D, regulatory, commercial, and supply chain teams to translate strategic intent into operational reality.
Robust and transparent research methodology combining literature synthesis, expert interviews, and scenario-based supply chain mapping to validate strategic conclusions
The research underpinning this analysis integrates a disciplined blend of primary and secondary methodologies designed to ensure validity, reproducibility, and relevance for decision-makers. Secondary evidence was synthesized from peer-reviewed clinical literature, regulatory documents, treatment guidelines, and public policy materials to construct a baseline understanding of disease biology, therapeutic mechanisms, and care standards. Primary input was obtained through structured interviews with clinical experts, specialty pharmacists, supply chain leaders, and patient advocates to capture real-world operational challenges, access barriers, and emergent best practices.
These qualitative insights were triangulated across multiple sources to validate thematic conclusions and to identify actionable implications for stakeholders. Supply chain mapping exercises and scenario analyses informed the assessment of tariff-related impacts and operational mitigations. Throughout the study, rigorous editorial and methodological controls were applied to ensure that conclusions reflect consensus where present and transparently highlight areas of uncertainty where evidence remains emergent. The result is a pragmatic, stakeholder-oriented evidence base intended to inform clinical program design, commercial strategy, and operational planning.
Concluding synthesis of clinical promise, operational complexity, and stakeholder responsibilities to translate therapeutic advances into improved long-term outcomes for people with PKU
The evolving therapeutic and care-delivery environment for phenylketonuria presents both significant promise and practical complexity. Scientific advances are expanding the range of interventions that can alter disease trajectory, yet each innovation brings distinct operational demands related to manufacturing, distribution, clinical administration, and payer engagement. Concurrently, policy shifts and macroeconomic factors, including trade and tariff dynamics, introduce supply-side uncertainties that can influence access and cost management strategies.
To translate therapeutic potential into consistent clinical benefit, stakeholders must coordinate across clinical, commercial, and policy domains. Clinicians and researchers should continue to prioritize outcomes that matter to patients and payers, while manufacturers and distributors should build resilient, patient-centered delivery systems. Payers and policymakers play a critical role by creating frameworks that reward durable health outcomes and support equitable access. Together, these aligned efforts can ensure that advances in treatment meaningfully improve long-term cognitive, functional, and quality-of-life outcomes for people living with phenylketonuria.
Please Note: PDF & Excel + Online Access - 1 Year
Comprehensive introduction to phenylketonuria treatment evolution contextualized within contemporary clinical pathways and patient-centered management considerations
Phenylketonuria (PKU) stands as a clinically and commercially significant inherited metabolic condition characterized by impaired metabolism of the amino acid phenylalanine. Advances in newborn screening, biochemical diagnostics, and lifelong management pathways have reshaped clinical trajectories, enabling earlier intervention and more precise therapeutic decision-making. Over recent years, therapeutic options have diversified beyond strict dietary regimens to include pharmacological agents, enzyme substitution approaches, and genetic modalities, each carrying distinct clinical profiles, administration requirements, and patient adherence implications.
Clinicians, caregivers, and health systems now must navigate an evolving evidence base that links biochemical control with neurocognitive and quality-of-life outcomes across pediatric and adult populations. This report provides an integrated perspective on current standards of care, emerging interventional strategies, and the complex interplay between clinical efficacy, delivery mechanisms, and real-world uptake. By framing treatment innovation within the operational realities of specialty supply chains, payer frameworks, and patient-centered care pathways, the analysis equips decision-makers with a coherent foundation for strategic planning and clinical program development.
Strategic overview of how scientific innovation, care-delivery modernization, and payer evolution are reshaping the future of phenylketonuria treatment access
The therapeutic landscape for phenylketonuria has entered a period of transformative change driven by converging scientific, regulatory, and care-delivery trends. Gene-centric approaches and enzyme substitution technologies are progressing through clinical development, promising mechanisms that target the root metabolic defect rather than solely mitigating downstream consequences. At the same time, pharmacological innovations have expanded treatment options for patients who do not achieve optimal biochemical control with dietary measures alone, and nutritional supplement formulations continue to improve tolerability and adherence.
These shifts are reinforced by technology-enabled care models, including digital adherence tools, telehealth-supported dietary management, and specialty pharmacy coordination, which collectively reduce barriers to consistent long-term management. Payer engagement has evolved in parallel, with value-based contracting discussions increasingly focused on clinical outcomes, patient-reported measures, and long-term cost offsets associated with improved neurocognitive trajectories. Consequently, stakeholders must align development strategies, commercial models, and care delivery designs to capture the clinical promise of innovation while addressing operational complexity and equitable access.
Practical analysis of how the United States tariff changes anticipated in 2025 could alter supply chain resilience, access pathways, and procurement strategies for PKU-related therapies
Anticipated tariff adjustments implemented in the United States in 2025 introduce a new layer of complexity for supply chains that support specialty therapies, medical foods, and laboratory reagents essential to phenylketonuria care. Many therapy components, including specialized proteins, reagents for diagnostic testing, and niche nutritional ingredients, rely on a global manufacturing footprint. Changes to import costs can therefore influence procurement strategies, push sourcing diversification, and catalyze nearshoring conversations among manufacturers seeking to preserve price stability and supply continuity.
Clinically, any reconfiguration of supply lines has the potential to create disparities in access if cost pressures translate into constrained distribution of high-cost therapies or interruptions in availability of specialized dietary products. Payers and health systems are likely to emphasize formulary resilience and contract language that guards against supply volatility. Strategically, biopharmaceutical developers and specialty manufacturers should accelerate supply chain mapping, including alternative sourcing, domestic manufacturing options, and inventory-buffer strategies, while engaging with policymakers to highlight the clinical ramifications of tariff-driven disruptions. In parallel, clinicians and patient advocacy groups will need proactive communication plans to manage expectations and support continuity of care during transitional periods.
Granular segmentation insights that connect treatment modalities, administration routes, age-based clinical pathways, and distribution channels to real-world access and adherence dynamics
A segmentation-focused lens clarifies how therapeutic uptake and care delivery differ across treatment modalities, routes of administration, age cohorts, and distribution pathways. Treatments that target the underlying defect, including gene therapy and enzyme substitution approaches, attract distinct development and commercialization pathways compared with nutritional supplements and pharmacological treatments, the latter category of which includes therapies such as pegvaliase and sapropterin dihydrochloride that address metabolic control through enzymatic replacement or cofactor modulation. Route of administration further differentiates patient and provider preferences, with oral therapies typically favored for ease of use and parenteral administrations requiring specialized clinic-based coordination or supportive home infusion models.
Age is a central determinant of clinical strategy and adherence design; pediatric populations face developmental and nutritional considerations that shape device selection, dosing strategies, and caregiver education, whereas adults contend with long-term adherence, comorbidity management, and transitions from pediatric to adult specialty care. Distribution channels likewise influence access and patient experience: hospital pharmacies and specialty clinics offer integrated clinical oversight for complex parenteral therapies and enzymatic treatments, while retail and online pharmacy models provide convenient refills for oral agents and nutraceuticals. Understanding these intersecting segments enables targeted clinical pathways, payer engagement strategies, and commercial models that reflect the operational realities of each patient cohort and product archetype.
Regional analysis highlighting how distinct regulatory environments, care infrastructures, and distribution capabilities shape phenylketonuria treatment pathways across major global territories
Regional dynamics exert a profound influence on regulatory pathways, reimbursement architectures, clinical practice norms, and supply chain logistics across the global phenylketonuria landscape. In the Americas, established newborn screening programs, concentrated specialty care centers, and an evolving payer environment create opportunities and constraints that shape therapy adoption patterns and patient management infrastructures. Stakeholders in this region must navigate a complex interplay of private and public payer requirements alongside robust patient advocacy networks that influence guideline development and access programs.
The Europe, Middle East & Africa region presents diverse regulatory frameworks and heterogenous health system capacities; centralized regulatory mechanisms coexist with country-level reimbursement heterogeneity, creating fragmentation that developers must address through phased approvals, localized evidence generation, and tailored access strategies. In the Asia-Pacific region, increasing investment in genetic and rare disease capabilities, expanding newborn screening coverage, and growing local manufacturing capacity are altering the supply and demand equation. Each regional bloc requires bespoke engagement plans that account for regulatory timelines, local clinical practice patterns, procurement norms, and the maturity of specialty distribution infrastructures.
Insightful synthesis of competitive strategies, partnership models, and operational enablers that define company positioning and program execution in the PKU treatment ecosystem
The competitive and collaborative landscape features a blend of established specialty pharmaceutical organizations, nimble biotechnology companies, diagnostic firms, and manufacturers of medical foods and nutritional supplements. Strategic activity increasingly centers on partnerships that combine clinical development expertise with manufacturing scale, as well as collaborations that integrate diagnostics with therapeutic delivery to enable precision-tailored interventions. Across the continuum, stakeholders prioritize evidence generation that demonstrates durable biochemical control, neurocognitive benefit, and meaningful patient-reported outcomes to support clinical adoption and payer engagement.
Operationally, companies investing in vertically integrated capabilities-spanning clinical manufacturing, cold-chain logistics for parenteral products, and specialized distribution-are better positioned to manage complexity and ensure continuity of supply. In parallel, commercial leaders emphasize robust patient support programs, specialty pharmacy relationships, and digital tools to optimize adherence and outcomes. Mergers, licensing deals, and strategic alliances continue to shape the innovation pipeline and route-to-market strategies, with an emphasis on technologies that reduce treatment burden and expand the pool of patients who can sustain long-term metabolic control.
Actionable and pragmatic recommendations for companies to strengthen evidence generation, supply resilience, distribution strategies, and patient-centric commercial models in PKU care
Industry leaders should adopt a multipronged approach that aligns clinical development priorities with pragmatic commercial and operational design. First, invest in evidence-generation frameworks that link biochemical endpoints to functional and cognitive outcomes, thereby strengthening value propositions for payers and clinicians. Next, prioritize supply chain resilience by diversifying sourcing, validating alternate suppliers for critical inputs, and investing in manufacturing redundancies or regional production capacities to mitigate tariff and geopolitical risks.
Additionally, companies should tailor product and service delivery to segmented patient needs, optimizing formulations and administration modalities for pediatric versus adult populations and coordinating distribution strategies that leverage both specialty clinic integration and patient-centric retail or online fulfillment. Engage proactively with payers through outcomes-based contracting pilots and real-world evidence programs that demonstrate clinical and economic impact. Finally, cultivate robust patient support ecosystems that combine clinical education, digital adherence tools, and coordinated specialty pharmacy services to reduce attrition and improve long-term outcomes. Executing these priorities will require cross-functional alignment across R&D, regulatory, commercial, and supply chain teams to translate strategic intent into operational reality.
Robust and transparent research methodology combining literature synthesis, expert interviews, and scenario-based supply chain mapping to validate strategic conclusions
The research underpinning this analysis integrates a disciplined blend of primary and secondary methodologies designed to ensure validity, reproducibility, and relevance for decision-makers. Secondary evidence was synthesized from peer-reviewed clinical literature, regulatory documents, treatment guidelines, and public policy materials to construct a baseline understanding of disease biology, therapeutic mechanisms, and care standards. Primary input was obtained through structured interviews with clinical experts, specialty pharmacists, supply chain leaders, and patient advocates to capture real-world operational challenges, access barriers, and emergent best practices.
These qualitative insights were triangulated across multiple sources to validate thematic conclusions and to identify actionable implications for stakeholders. Supply chain mapping exercises and scenario analyses informed the assessment of tariff-related impacts and operational mitigations. Throughout the study, rigorous editorial and methodological controls were applied to ensure that conclusions reflect consensus where present and transparently highlight areas of uncertainty where evidence remains emergent. The result is a pragmatic, stakeholder-oriented evidence base intended to inform clinical program design, commercial strategy, and operational planning.
Concluding synthesis of clinical promise, operational complexity, and stakeholder responsibilities to translate therapeutic advances into improved long-term outcomes for people with PKU
The evolving therapeutic and care-delivery environment for phenylketonuria presents both significant promise and practical complexity. Scientific advances are expanding the range of interventions that can alter disease trajectory, yet each innovation brings distinct operational demands related to manufacturing, distribution, clinical administration, and payer engagement. Concurrently, policy shifts and macroeconomic factors, including trade and tariff dynamics, introduce supply-side uncertainties that can influence access and cost management strategies.
To translate therapeutic potential into consistent clinical benefit, stakeholders must coordinate across clinical, commercial, and policy domains. Clinicians and researchers should continue to prioritize outcomes that matter to patients and payers, while manufacturers and distributors should build resilient, patient-centered delivery systems. Payers and policymakers play a critical role by creating frameworks that reward durable health outcomes and support equitable access. Together, these aligned efforts can ensure that advances in treatment meaningfully improve long-term cognitive, functional, and quality-of-life outcomes for people living with phenylketonuria.
Please Note: PDF & Excel + Online Access - 1 Year
Table of Contents
191 Pages
- 1. Preface
- 1.1. Objectives of the Study
- 1.2. Market Segmentation & Coverage
- 1.3. Years Considered for the Study
- 1.4. Currency
- 1.5. Language
- 1.6. Stakeholders
- 2. Research Methodology
- 3. Executive Summary
- 4. Market Overview
- 5. Market Insights
- 5.1. Expansion of pegylated enzyme substitution therapies improving patient adherence and outcomes
- 5.2. Development of gene editing approaches targeting PAH gene mutations to provide one-time curative treatments
- 5.3. Advances in large neutral amino acid therapies designed to reduce phenylalanine brain uptake in adults
- 5.4. Emergence of digital health platforms enabling remote phenylalanine monitoring and dietary management
- 5.5. Strategic partnerships between specialty diet companies and biotech firms to innovate PKU medical nutrition
- 5.6. Regulatory approvals of mRNA-based therapies for phenylalanine hydroxylase restoration in PKU patients
- 6. Cumulative Impact of United States Tariffs 2025
- 7. Cumulative Impact of Artificial Intelligence 2025
- 8. Phenylketonuria Treatment Market, by Treatment Type
- 8.1. Gene Therapy & Enzyme Substitution Therapies
- 8.2. Nutritional Supplements
- 8.3. Pharmacological Treatment
- 8.3.1. Pegvaliase
- 8.3.2. Sapropterin Dihydrochloride
- 9. Phenylketonuria Treatment Market, by Route of Administration
- 9.1. Oral
- 9.2. Parenteral
- 10. Phenylketonuria Treatment Market, by Distribution Channel
- 10.1. Offline
- 10.2. Online
- 11. Phenylketonuria Treatment Market, by Region
- 11.1. Americas
- 11.1.1. North America
- 11.1.2. Latin America
- 11.2. Europe, Middle East & Africa
- 11.2.1. Europe
- 11.2.2. Middle East
- 11.2.3. Africa
- 11.3. Asia-Pacific
- 12. Phenylketonuria Treatment Market, by Group
- 12.1. ASEAN
- 12.2. GCC
- 12.3. European Union
- 12.4. BRICS
- 12.5. G7
- 12.6. NATO
- 13. Phenylketonuria Treatment Market, by Country
- 13.1. United States
- 13.2. Canada
- 13.3. Mexico
- 13.4. Brazil
- 13.5. United Kingdom
- 13.6. Germany
- 13.7. France
- 13.8. Russia
- 13.9. Italy
- 13.10. Spain
- 13.11. China
- 13.12. India
- 13.13. Japan
- 13.14. Australia
- 13.15. South Korea
- 14. Competitive Landscape
- 14.1. Market Share Analysis, 2024
- 14.2. FPNV Positioning Matrix, 2024
- 14.3. Competitive Analysis
- 14.3.1. Abbott Laboratories
- 14.3.2. American Gene Technologies
- 14.3.3. APR Applied Pharma Research s.a.
- 14.3.4. BioMarin Pharmaceuticals Inc
- 14.3.5. Codexis, Inc.
- 14.3.6. Daiichi Sankyo Co. Ltd.
- 14.3.7. Galen Limited
- 14.3.8. Homology Medicines, Inc.
- 14.3.9. Nestlé S.A.
- 14.3.10. PIAM Farmaceutici S.P.A.
- 14.3.11. Pluvia AS
- 14.3.12. PTC Therapeutics, Inc.
- 14.3.13. Relief Therapeutics Holding SA
- 14.3.14. Sanofi group
- 14.3.15. Synlogic
- 14.3.16. Travere Therapeutics, Inc.
- 14.3.17. Ultragenyx Pharmaceutical Inc.
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