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Global Phenylketonuria Treatment Market Report and Forecast 2023-2031

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    Report

  • 160 Pages
  • May 2023
  • Region: Global
  • Expert Market Research
  • ID: 5805708
The global Phenylketonuria treatment market value was USD 0.88 billion in 2022, driven by the advancements in gene therapy and other innovative treatment approaches across the globe. The market size is anticipated to grow at a CAGR of 9.3% during the forecast period of 2023-2031 to achieve a value of USD 1.96 billion by 2031.

Introduction

Phenylketonuria (PKU) is a rare genetic disorder characterized by the body's inability to break down an amino acid called phenylalanine. If left untreated, phenylalanine can accumulate in the body and lead to intellectual disabilities and other serious health problems. The treatment of PKU primarily involves dietary management to restrict phenylalanine intake and ensure adequate nutrition.

The introduction of treatment for PKU has significantly improved the outcomes and quality of life for individuals with this condition. The cornerstone of PKU treatment is a special diet low in phenylalanine, often referred to as a phenylalanine-restricted diet. This diet involves the careful monitoring of phenylalanine intake from food and the use of specially formulated medical foods and low-protein foods to provide necessary nutrients while limiting phenylalanine. Additionally, individuals with PKU may require dietary supplements to ensure adequate intake of vitamins, minerals, and other essential nutrients.

In recent years, there have been key trends in the treatment of PKU. One notable trend is the development and use of medical foods and formulas specifically designed for individuals with PKU. These products are formulated to provide precise amounts of essential nutrients while maintaining low levels of phenylalanine. Advances in food science and technology have enabled the creation of better-tasting and more palatable products, improving patient compliance with the phenylalanine-restricted diet.

Key Trends in the Phenylketonuria Treatment Market

Some key trends in the market for Phenylketonuria (PKU) treatment include:
  • Development of novel therapies: Researchers are actively exploring new treatment approaches for PKU, including gene therapy, enzyme replacement therapies, and small molecule drugs. These innovative therapies aim to address the underlying metabolic defect in PKU and offer potential alternatives to the strict dietary management currently used
  • Improved formulations of medical foods: Medical foods play a crucial role in the management of PKU by providing essential nutrients while limiting phenylalanine intake. There is a growing trend towards the development of improved formulations of medical foods, including better taste, texture, and variety. These advancements aim to improve patient compliance and make the phenylalanine-restricted diet more manageable and enjoyable
  • Personalized treatment approaches: PKU is a heterogeneous disorder, and individual patients may respond differently to treatment. There is increasing emphasis on personalized treatment approaches that take into account the specific needs and characteristics of each patient. This includes tailoring dietary plans, adjusting phenylalanine intake based on individual tolerance, and considering factors such as age, metabolic control, and comorbidities

Phenylketonuria Treatment Market Segmentations

Market Breakup by Type

  • Protozoa
  • Helminths
  • Ectoparasite

Market Breakup by Population

  • Children
  • Adults

Market Breakup by Drug Class

  • Anthelmintics
  • Antiprotozoals
  • Others

Market by Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • online Pharmacies
  • Others

Market Breakup by Region

North America

  • United States of America
  • Canada

Europe

  • United Kingdom
  • Germany
  • France
  • Italy
  • Others

Asia Pacific

  • China
  • Japan
  • India
  • ASEAN
  • Australia
  • Others

Latin America

  • Brazil
  • Argentina
  • Mexico
  • Others

Middle East and Africa

  • Saudi Arabia
  • United Arab Emirates
  • Nigeria
  • South Africa
  • Others

Phenylketonuria Treatment Market Scenario

The market for Phenylketonuria (PKU) treatment is driven by the need to provide effective management options for individuals living with this genetic disorder. PKU is characterized by the inability to properly metabolize phenylalanine, leading to the accumulation of this amino acid in the body. Without proper management, high levels of phenylalanine can result in cognitive impairments and other complications.

The market for PKU treatment encompasses various segments, including medical foods, medications, and other therapies. Medical foods, such as low-protein formulas and special low-phenylalanine foods, form the cornerstone of PKU management. These products are designed to provide essential nutrients while strictly limiting phenylalanine intake. The market for medical foods is driven by the increasing demand for improved formulations that offer better taste, texture, and variety to enhance patient compliance and satisfaction.

Furthermore, advancements in gene therapy and other innovative treatment approaches are shaping the PKU treatment market. Gene therapy aims to address the underlying genetic defect responsible for PKU and potentially provide a long-term solution. Ongoing clinical trials and research in this area highlight the potential for future breakthroughs in PKU treatment.

Overall, the PKU treatment market is characterized by ongoing advancements in medical foods, medications, and innovative therapies. The focus is on improving patient outcomes, enhancing quality of life, and exploring new treatment options that address the underlying metabolic defect in PKU. As research and development continue to advance, the market is expected to witness further growth and innovation in the coming years.

Phenylketonuria Treatment Market: Competitor Landscape

The key features of the market report include patent analysis, grants analysis, clinical trials analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players. The major companies in the market are as follows:
  • Boehringer Ingelheim International GmbH
  • Eisai Co., Ltd
  • Pfizer, Inc
  • Sanofi
  • Teva Pharmaceutical Industries Ltd
  • Aspen Holdings
  • Mitsubishi Tanabe Pharma Corporation
  • Abbot
  • GlaxoSmithKline plc
  • Bristol-Myers Squibb Company

Table of Contents

1 Preface2 Report Coverage - Key Segmentation and Scope
3 Report Description
3.1 Market Definition and Outlook
3.2 Properties and Applications
3.3 Market Analysis
3.4 Key Players
4 Key Assumptions
5 Executive Summary
5.1 Overview
5.2 Key Drivers
5.3 Key Developments
5.4 Competitive Structure
5.5 Key Industrial Trends
6 Snapshot
6.1 Global
6.2 Regional
7 Opportunities and Challenges in the Market
8 Global Phenylketonuria Treatment Market Analysis
8.1 Key Industry Highlights
8.2 Global Phenylketonuria Treatment Historical Market (2018-2022)
8.3 Global Phenylketonuria Treatment Market Forecast (2023-2028)
8.4 Global Phenylketonuria Treatment Market by Product Type
8.4.1 Dietary Supplement
8.4.1.1 Market Share
8.4.1.2 Historical Trend (2018-2022)
8.4.1.3 Forecast Trend (2023-2028)
8.4.2 Drugs
8.4.2.1 Market Share
8.4.2.2 Historical Trend (2018-2022)
8.4.2.3 Forecast Trend (2023-2028)
8.4.2.4 Breakup by Type
8.4.2.4.1 Kuvan
8.4.2.4.2 Palynziq
8.4.2.4.3 CNSA-001
8.4.2.4.4 SYNB1618
8.5 Global Phenylketonuria Treatment Market by Route of Administration
8.5.1 Oral
8.5.1.1 Market Share
8.5.1.2 Historical Trend (2018-2022)
8.5.1.3 Forecast Trend (2023-2028)
8.5.2 Parenteral
8.5.2.1 Market Share
8.5.2.2 Historical Trend (2018-2022)
8.5.2.3 Forecast Trend (2023-2028)
8.6 Global Phenylketonuria Treatment Market by Distribution Channel
8.6.1 Hospital Pharmacies
8.6.1.1 Market Share
8.6.1.2 Historical Trend (2018-2022)
8.6.1.3 Forecast Trend (2023-2028)
8.6.2 Online Pharmacies
8.6.2.1 Market Share
8.6.2.2 Historical Trend (2018-2022)
8.6.2.3 Forecast Trend (2023-2028)
8.6.3 Paediatric Clinics
8.6.3.1 Market Share
8.6.3.2 Historical Trend (2018-2022)
8.6.3.3 Forecast Trend (2023-2028)
8.6.4 Drug Stores
8.6.4.1 Market Share
8.6.4.2 Historical Trend (2018-2022)
8.6.4.3 Forecast Trend (2023-2028)
8.7 Global Phenylketonuria Treatment Market by Region
8.7.1 North America
8.7.1.1 Market Share
8.7.1.2 Historical Trend (2018-2022)
8.7.1.3 Forecast Trend (2023-2028)
8.7.2 Europe
8.7.2.1 Market Share
8.7.2.2 Historical Trend (2018-2022)
8.7.2.3 Forecast Trend (2023-2028)
8.7.3 Asia Pacific
8.7.3.1 Market Share
8.7.3.2 Historical Trend (2018-2022)
8.7.3.3 Forecast Trend (2023-2028)
8.7.4 Latin America
8.7.4.1 Market Share
8.7.4.2 Historical Trend (2018-2022)
8.7.4.3 Forecast Trend (2023-2028)
8.7.5 Middle East and Africa
8.7.5.1 Market Share
8.7.5.2 Historical Trend (2018-2022)
8.7.5.3 Forecast Trend (2023-2028)
9 North America Phenylketonuria Treatment Market Analysis
9.1 United States of America
9.1.1 Market Share
9.1.2 Historical Trend (2018-2022)
9.1.3 Forecast Trend (2023-2028)
9.2 Canada
9.2.1 Market Share
9.2.2 Historical Trend (2018-2022)
9.2.3 Forecast Trend (2023-2028)
10 Europe Phenylketonuria Treatment Market Analysis
10.1 United Kingdom
10.1.1 Market Share
10.1.2 Historical Trend (2018-2022)
10.1.3 Forecast Trend (2023-2028)
10.2 Germany
10.2.1 Market Share
10.2.2 Historical Trend (2018-2022)
10.2.3 Forecast Trend (2023-2028)
10.3 France
10.3.1 Market Share
10.3.2 Historical Trend (2018-2022)
10.3.3 Forecast Trend (2023-2028)
10.4 Italy
10.4.1 Market Share
10.4.2 Historical Trend (2018-2022)
10.4.3 Forecast Trend (2023-2028)
10.5 Others
11 Asia Pacific Phenylketonuria Treatment Market Analysis
11.1 China
11.1.1 Market Share
11.1.2 Historical Trend (2018-2022)
11.1.3 Forecast Trend (2023-2028)
11.2 Japan
11.2.1 Market Share
11.2.2 Historical Trend (2018-2022)
11.2.3 Forecast Trend (2023-2028)
11.3 India
11.3.1 Market Share
11.3.2 Historical Trend (2018-2022)
11.3.3 Forecast Trend (2023-2028)
11.4 ASEAN
11.4.1 Market Share
11.4.2 Historical Trend (2018-2022)
11.4.3 Forecast Trend (2023-2028)
11.5 Australia
11.5.1 Market Share
11.5.2 Historical Trend (2018-2022)
11.5.3 Forecast Trend (2023-2028)
11.6 Others
12 Latin America Phenylketonuria Treatment Market Analysis
12.1 Brazil
12.1.1 Market Share
12.1.2 Historical Trend (2018-2022)
12.1.3 Forecast Trend (2023-2028)
12.2 Argentina
12.2.1 Market Share
12.2.2 Historical Trend (2018-2022)
12.2.3 Forecast Trend (2023-2028)
12.3 Mexico
12.3.1 Market Share
12.3.2 Historical Trend (2018-2022)
12.3.3 Forecast Trend (2023-2028)
12.4 Others
13 Middle East and Africa Phenylketonuria Treatment Market Analysis
13.1 Saudi Arabia
13.1.1 Market Share
13.1.2 Historical Trend (2018-2022)
13.1.3 Forecast Trend (2023-2028)
13.2 United Arab Emirates
13.2.1 Market Share
13.2.2 Historical Trend (2018-2022)
13.2.3 Forecast Trend (2023-2028)
13.3 Nigeria
13.3.1 Market Share
13.3.2 Historical Trend (2018-2022)
13.3.3 Forecast Trend (2023-2028)
13.4 South Africa
13.4.1 Market Share
13.4.2 Historical Trend (2018-2022)
13.4.3 Forecast Trend (2023-2028)
13.5 Others
14 Market Dynamics
14.1 SWOT Analysis
14.1.1 Strengths
14.1.2 Weaknesses
14.1.3 Opportunities
14.1.4 Threats
14.2 Porter’s Five Forces Analysis
14.2.1 Supplier’s Power
14.2.2 Buyer’s Power
14.2.3 Threat of New Entrants
14.2.4 Degree of Rivalry
14.2.5 Threat of Substitutes
14.3 Key Indicators for Demand
14.4 Key Indicators for Price
15 Competitive Landscape
15.1 Market Structure
15.2 Company Profiles
15.2.1 BioMarin Pharmaceutical Inc.
15.2.1.1 Company Overview
15.2.1.2 Product Portfolio
15.2.1.3 Demographic Reach and Achievements
15.2.1.4 Certifications
15.2.2 Synlogic, Inc.
15.2.2.1 Company Overview
15.2.2.2 Product Portfolio
15.2.2.3 Demographic Reach and Achievements
15.2.2.4 Certifications
15.2.3 Retrophin, Inc.
15.2.3.1 Company Overview
15.2.3.2 Product Portfolio
15.2.3.3 Demographic Reach and Achievements
15.2.3.4 Certifications
15.2.4 Codexis, Inc.
15.2.4.1 Company Overview
15.2.4.2 Product Portfolio
15.2.4.3 Demographic Reach and Achievements
15.2.4.4 Certifications
15.2.5 SOM Innovation Biotech, S.A.
15.2.5.1 Company Overview
15.2.5.2 Product Portfolio
15.2.5.3 Demographic Reach and Achievements
15.2.5.4 Certifications
15.2.6 Homology Medicines, Inc.
15.2.6.1 Company Overview
15.2.6.2 Product Portfolio
15.2.6.3 Demographic Reach and Achievements
15.2.6.4 Certifications
15.2.7 Others
16 Key Trends and Developments in the Market

Companies Mentioned

  • BioMarin Pharmaceutical Inc.
  • Synlogic Inc.
  • Retrophin Inc.
  • Codexis Inc.
  • SOM Innovation Biotech
  • S.A.
  • Homology Medicines Inc.

Methodology

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Table Information