Phenylketonuria Treatment Market

Phenylketonuria Treatment Market by Drugs (Kuvan, Palynziq, CNSA-001, and SYNB1618), and Regions (Asia Pacific, North America, Latin America, Europe, and Middle East & Africa) - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast 2021 – 2028

  • Report ID: HC-1270
  • Author: Growth Market Reports
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  • No. Of Pages: 190
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The global phenylketonuria treatment market size is projected to register a significant CAGR during the forecast period, 2021–2028. The growth of the market is attributed to the growing prevalence of phenylketonuria around the world.
 

Phenylketonuria Treatment Market Key Takeaways


Phenylketonuria (PKU) is a birth defect that take place due to mutation of gene which are responsible for encoding phenylalanine hydroxylase. In PKU defect metabolism of amino acid decreases which is known as phenylalanine. In affected people, higher existence of phenylalanine is found in blood levels. Phenylketonuria has no certain cure but, it can be managed by use of additional therapy and diet controls. Uncontrolled PKU causes several intellectual disability, mental and behavioral disorders, and seizures in children. Based on data given by the Genetics Home Reference, in the U.S., around 1 in 12,000 cases of PKU occurs in infants. In Europe, some countries have high presence of PKU, such as around 1 in 4550 newborns in Ireland and 1 in every 4100 babies in Turkey, are having target disease.

The occurrence of pipeline drugs includes RTX-134, SYNB1618, and CNSA-001, which aid the market. Phase I study of CNSA-001 was completed and started Phase II clinical trial whereas SYNB1618 is presently in Phase 2 and received Food and Drug Administration (FDA) fast-track designation. Increasing commercialization and development of new drugs helps the market expansion. For instance, in 2018, in the U.S., Palynziq (pegvaliase), which is an enzyme therapy drug was launched by BioMarin Pharmaceuticals, Inc. for PKU treatment. Moreover, this drug was approved in the U.S. in 2018 and it was approved by the European Medicines Agency (EMA) in May 2019 for the Marketing Authorization Application (MAA).

Increasing M&A and collaboration between key player’s results in propelling the market. Synlogic, Inc. discovered and developed several novel drugs for Synthetic Biotic medicines. These medicines were designed with use of synthetic biology for reprogramming microbes genetically for the treatment of inflammatory and metabolic diseases. Retrophin, Inc., in January 2018 enters into joint agreement with Censa Pharmaceuticals for manufacturing CNSA-001. In this, Censa performs clinical trial programs and Retrophin acquire exclusive rights after drug development from Censa.

Market Trends, Drivers, Restraints, and Opportunities

  • Rising number of pipeline drugs, growing development and commercialization of novel drugs are projected to fuel the market growth.
  • Growing prevalence of phenylketonuria among infants, rising awareness among people are expected to boost the market growth over the forecast period.
  • Advancements in healthcare industry, growing per capita income in developing countries, and increasing research of genomics and bioinformatics are estimated to propel the market expansion in coming years.
  • Increasing acquisitions and collaborations between major market players and changing lifestyle are anticipated to drive the market expansion.
  • Rise in use of several gene and targeted therapy for phenylketonuria management is projected to boost the growth of the market.
  • High cost of treatments and stringent government regulations are expected to impede the market growth in the coming years.
  • Lack of awareness about phenylketonuria among physician and patients and unfavorable reimbursement policies are major factors hampering the market expansion over the forecast period.

Scope of the Report

The report on the global phenylketonuria treatment market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.

Attributes

Details

Report Title

Phenylketonuria Treatment Market - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast

Base Year

2020

Historic Data

2018–2019

Forecast Period

2021–2028

Segmentation

Drugs (Kuvan, Palynziq, CNSA-001, and SYNB1618)

Regional Scope

Asia Pacific, North America, Latin America, Europe, and Middle East & Africa

Report Coverage

Company Share, Market Analysis and Size, Competitive Landscape, Growth Factors, and Trends, and Revenue Forecast

Key Players Covered in the Report

BioMarin Pharmaceuticals, Inc., Censa Pharmaceutical, Inc., Retrophin, Inc., Synlogic, Inc., Rubius Therapeutics, Inc., and Homology Medicines, Inc.

 

Market Segment Insights

Kuvan drug segment is projected to hold a key market share

Based on drugs, the phenylketonuria treatment market is divided into Kuvan, Palynziq, CNSA-001, and SYNB1618. The Kuvan drug segment is expected to hold a key share of the market during the forecast period. Kuvan (sapropterin dihydrochloride) is manufactured by BioMarin Pharmaceutical, Inc. It is an oral drug and presently efficient for the treatment of phenylketonuria. Kuvan adds BH4 compound which stimulates phenylalanine hydroxylase enzyme and then breakdown the phenylalanine protein. In the U.S. Kuvan was approved in 2007 and in Europe it was in 2008. The patent of drug got expired in 2015 in the U.S. and in Europe it was in 2020. Though, BioMarin Pharmaceuticals gave license to Par Pharmaceuticals for launching generic version of Kuvan 100mg tablet and powder form.

However, BioMarin Pharmaceutical manufactured Palynziq (pegvaliase) which is a subcutaneous injectable enzyme therapy drug. This drug was approved in the U.S. in 2018 and it is approved by the European Medicines Agency (EMA) in May 2019 for the Marketing Authorization Application (MAA).
 

Phenylketonuria Treatment Market by Drugs

North America is expected to dominate the market

On the basis of regions, the phenylketonuria treatment market is categorized as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. The market of North America is anticipated to dominate the market during the forecast period. The regional market growth can be attributed to increasing government initiatives and favorable regulations for phenylketonuria treatment. Addition to this, continuous R&D and commercialization of novel drugs are projected to fuel the regional market growth.

On the other hand, the market of Europe is expected to exhibit a rapid growth rate in the coming years due to introduction of Palynziq drug and delay in generic version of Kuvan. Moreover, high expenditure on healthcare and well-established healthcare infrastructure are anticipated to drive the regional market growth.
 

Phenylketonuria Treatment Market by Regions

1. Executive Summary
2. Assumptions and Acronyms Used
3. Research Methodology
4. Phenylketonuria Treatment Market Overview
  4.1. Introduction
     4.1.1. Market Taxonomy
     4.1.2. Market Definition
  4.2. Macro-Economic Factors
     4.2.1. Industry Outlook
  4.3. Phenylketonuria Treatment Market Dynamics
     4.3.1. Market Drivers
     4.3.2. Market Restraints
     4.3.3. Opportunity
     4.3.4. Market Trends
  4.4. Phenylketonuria Treatment Market - Supply Chain
  4.5. Global Phenylketonuria Treatment Market Forecast
     4.5.1. Phenylketonuria Treatment Market Size (US$ Mn) and Y-o-Y Growth
     4.5.2. Phenylketonuria Treatment Market Size (000’ Units) and Y-o-Y Growth
     4.5.3. Phenylketonuria Treatment Market Absolute $ Opportunity
5. Global Phenylketonuria Treatment Market Analysis and Forecast by Region
  5.1. Market Trends
  5.2. Introduction
     5.2.1. Basis Point Share (BPS) Analysis by Region
     5.2.2. Y-o-Y Growth Projections by Region
  5.3. Phenylketonuria Treatment Market Size and Volume Forecast by Region
     5.3.1. North America
     5.3.2. Latin America
     5.3.3. Europe
     5.3.4. Asia Pacific
     5.3.5. Middle East and Africa (MEA)
  5.4. Absolute $ Opportunity Assessment by Region
  5.5. Market Attractiveness/Growth Potential Analysis by Region
  5.6. Global Phenylketonuria Treatment Demand Share Forecast, 2019-2026
6. North America Phenylketonuria Treatment Market Analysis and Forecast
  6.1. Introduction
     6.1.1. Basis Point Share (BPS) Analysis by Country
     6.1.2. Y-o-Y Growth Projections by Country
  6.2. North America Phenylketonuria Treatment Market Size and Volume Forecast by Country
     6.2.1. U.S.
     6.2.2. Canada
  6.3. Absolute $ Opportunity Assessment by Country
  6.4. Market Attractiveness/Growth Potential Analysis
     6.4.1. By Country
     6.4.2. By Product Type
     6.4.3. By Application
  6.5. North America Phenylketonuria Treatment Demand Share Forecast, 2019-2026
7. Latin America Phenylketonuria Treatment Market Analysis and Forecast
  7.1. Introduction
     7.1.1. Basis Point Share (BPS) Analysis by Country
     7.1.2. Y-o-Y Growth Projections by Country
     7.1.3. Latin America Average Pricing Analysis
  7.2. Latin America Phenylketonuria Treatment Market Size and Volume Forecast by Country
      7.2.1. Brazil
      7.2.2. Mexico
      7.2.3. Rest of Latin America
   7.3. Absolute $ Opportunity Assessment by Country
  7.4. Market Attractiveness/Growth Potential Analysis
     7.4.1. By Country
     7.4.2. By Product Type
     7.4.3. By Application
  7.5. Latin America Phenylketonuria Treatment Demand Share Forecast, 2019-2026
8. Europe Phenylketonuria Treatment Market Analysis and Forecast
  8.1. Introduction
     8.1.1. Basis Point Share (BPS) Analysis by Country
     8.1.2. Y-o-Y Growth Projections by Country
     8.1.3. Europe Average Pricing Analysis
  8.2. Europe Phenylketonuria Treatment Market Size and Volume Forecast by Country
     8.2.1. Germany
     8.2.2. France
     8.2.3. Italy
     8.2.4. U.K.
     8.2.5. Spain
     8.2.6. Russia
     8.2.7. Rest of Europe
  8.3. Absolute $ Opportunity Assessment by Country
  8.4. Market Attractiveness/Growth Potential Analysis
     8.4.1. By Country
     8.4.2. By Product Type
     8.4.3. By Application
  8.5. Europe Phenylketonuria Treatment Demand Share Forecast, 2019-2026
9. Asia Pacific Phenylketonuria Treatment Market Analysis and Forecast
  9.1. Introduction
     9.1.1. Basis Point Share (BPS) Analysis by Country
     9.1.2. Y-o-Y Growth Projections by Country
     9.1.3. Asia Pacific Average Pricing Analysis
  9.2. Asia Pacific Phenylketonuria Treatment Market Size and Volume Forecast by Country
     9.2.1. China
     9.2.2. Japan
     9.2.3. South Korea
     9.2.4. India
     9.2.5. Australia
     9.2.6. Rest of Asia Pacific (APAC)
  9.3. Absolute $ Opportunity Assessment by Country
  9.4. Market Attractiveness/Growth Potential Analysis
     9.4.1. By Country
     9.4.2. By Product Type
     9.4.3. By Application
  9.5. Asia Pacific Phenylketonuria Treatment Demand Share Forecast, 2019-2026
10. Middle East & Africa Phenylketonuria Treatment Market Analysis and Forecast
  10.1. Introduction
     10.1.1. Basis Point Share (BPS) Analysis by Country
     10.1.2. Y-o-Y Growth Projections by Country
     10.1.3. Middle East & Africa Average Pricing Analysis
  10.2. Middle East & Africa Phenylketonuria Treatment Market Size and Volume Forecast by Country
     10.2.1. Saudi Arabia
     10.2.2. South Africa
     10.2.3. UAE
     10.2.4. Rest of Middle East & Africa (MEA)
  10.3. Absolute $ Opportunity Assessment by Country
  10.4. Market Attractiveness/Growth Potential Analysis
     10.4.1. By Country
     10.4.2. By Product Type
     10.4.3. By Application
  10.5. Middle East & Africa Phenylketonuria Treatment Demand Share Forecast, 2019-2026
11. Competition Landscape
  11.1. Global Phenylketonuria Treatment Market: Market Share Analysis
  11.2. Phenylketonuria Treatment Distributors and Customers
  11.3. Phenylketonuria Treatment Market: Competitive Dashboard
  11.4. Company Profiles (Details: Overview, Financials, Developments, Strategy)
     11.4.1. BioMarin Pharmaceuticals, Inc.
     11.4.2. Censa Pharmaceutical, Inc.
     11.4.3. Retrophin, Inc.
     11.4.4. Synlogic, Inc.
     11.4.5. Rubius Therapeutics, Inc.
     11.4.6. Homology Medicines, Inc.

Segments Covered in the Report
The global phenylketonuria treatment market has been segmented on the basis of

Drugs

  • Kuvan
  • Palynziq
  • CNSA-001
  • SYNB1618

Regions

  • Asia Pacific
  • North America
  • Latin America
  • Europe
  • Middle East & Africa

Key Players

  • BioMarin Pharmaceuticals, Inc.
  • Censa Pharmaceutical, Inc.
  • Retrophin, Inc.
  • Synlogic, Inc.
  • Rubius Therapeutics, Inc.
  • Homology Medicines, Inc.

Key players competing in the phenylketonuria treatment market are BioMarin Pharmaceuticals, Inc., Censa Pharmaceutical, Inc., Retrophin, Inc., Synlogic, Inc., Rubius Therapeutics, Inc., and Homology Medicines, Inc.

Major market players are adopting several business strategies such as mergers, acquisitions, partnerships, collaborations, capacity expansion, and product launches to increase their market shares. Furthermore, they support and conduct programs for providing treatment and care to patients having rare disorders such as PKU. For instance, Retrophin, Inc., in May 2019, funded the Rare Caregiver Respite Program. This program was launched by the National Organization for Rare Disorders (NORD), for providing different services to the patients.
 

Phenylketonuria Treatment Market by Key Players

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