Global Phenylketonuria Market Analysis


  • Type: Rare Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 383

  • Format: PPT, PDF



Report Overview

The Global Phenylketonuria drugs market size stood at USD 0.54 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Phenylketonuria (PKU) refers to a genetic disorder and the disease is inherited from parents. The patient suffers from a metabolic defect, in which the body stops being able to break down phenylalanine amino acid that is comprised of protein. Phenylalanine starts getting stored in the blood stream and it damages the brain in consequence

PKU is caused due a defect in the gene which helps to produce phenylalanine hydroxylase. Absence of phenylalanine hydroxylase leads to a buildup of phenylalanine in the body. Timely diagnosis and treatment helps in terminating the symptoms of PKU and avoid brain damage

Market Drivers

The Phenylketonuria Treatment market is anticipated to grow in the forecast, owing to the factors such as increasing research and development of novel drugs, growing acquisitions and collaborations between key market players, approvals of new drugs and growing prevalence of PKU among the children

1. Global Phenylketonuria Drugs Market Overview…….…
A. Market Size
2. Market Growth Drivers……………………………………
A. Rise in Global Prevalence of Phenylketonuria
B. Growing R&D Investments and Reimbursement Policies
3. Major Types of Treatments…….………..……………..……….
A. Oral drugs
B. Parental drugs
C. Others
4. Phenylketonuria Drugs Market Segmentation….………..
A. Drug Class
I. Kuvan
II. Palynziq
III. Others
B. Geography
I. North America Market Analysis
II. Europe Market Analysis
III. Asia-Pacific Market Analysis
IV. Middle East and Africa Market Analysis
C. Distribution Channel
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
IV. Others
5. Phenylketonuria Drugs Market Share…………..…………….
A. Market Analysis, Insights and Forecast – By Revenue
6. Competitive Landscape……………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles…………….…………………
A. Biomarin Company Product & Services, Strategies & Financials
B. Censa Pharmaceutical Product & Services, Strategies & Financials
C. Retrophin Inc. Company, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape…………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth………………………………
A. New Trends and Development of Phenylketonuria Drugs Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Drug Class:

Based on drugs, Kuvan and Palynziq are the two approved drugs for the treatment of PKU. Kuvan (sapropterin dihydrochloride) is an oral drug manufactured by BioMarin Pharmaceutical, Inc. The drug was approved in 2007 in the U.S. and 2008 in Europe. The drug patent got expired in 2015 in the U.S. and in 2020 in Europe

However, BioMarin has entered into a settlement agreement with Par Pharmaceutical for the launch of a generic version of its Kuvan 100mg tablet and powder form. On the other hand, Palynziq (pegvaliase) is the subcutaneous injectable enzyme therapy drug manufactured by BioMarin Pharmaceutical. The drug was approved in 2018 in the U.S. and received the EMA approval for the Marketing Authorization Application (MAA) in May 2019

By Distribution Channel:

Based on the distribution channel, the market has been segregated into hospitals, retail, online and other pharmacies

By Region:

North America held the largest market share in 2018. Favorable regulations and rising government initiatives for the treatment of PKU are the key factors driving the market. In addition, continuous research & development and commercialization of novel drugs are expected to further boost the PKU treatment market growth in this region

Europe is expected to witness the fastest CAGR over the forecast period owing to the delay in entry of generic version of Kuvan, and introduction of palynziq drug in 2019. Presence of well-established healthcare infrastructure and high healthcare expenditure are also projected to impel the fastest market in the region

Major Players

Some of the key companies in the market are BioMarin Pharmaceuticals, Inc.; Censa Pharmaceutical Inc.; Retrophin Inc.; Synlogic Inc., Rubius Therapeutics, Inc.; Homology Medicines, Inc. These companies focus on strategies, such as partnerships, new drug launch, collaborations, and mergers & acquisitions, to gain competitive advantage

In addition, they also conduct and support programs to provide care and treatment to patients suffering from rare disorders, such as PKU. For instance, in May 2019, Retrophin, Inc. provided initial funds to the Rare Caregiver Respite Program. The program was launched by the National Organization for Rare Disorders (NORD), which aims to provide various services and financial assistance to the patients

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

Primary Interviews: We have developed a network of experts, freelancers and researchers across countries through which we engage with local experts to gather key data points and assumptions around each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

Secondary Data Collection: We have developed strong expertise and experience in secondary data collection methods for developing unique data sets and research material. We gather data from multiple reliable sources to maintain the high level of accuracy and consistency. The market data is analysed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, market share as well as segment-level analysis of the specific market. Our report includes precise, to the point information related to the overall market, competition, growth drivers, challenges, regulatory updates, and competition.

Data Sources: We have access to multiple highly reliable free and subscription data sources. We have many years of experience to understand which sources are more dependable for what and which to prefer for the reliable and latest information. The key sources of information include the following, but are not limited to:

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Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameter and micro indicators. The macro parameters includes changes in government policies, demand and supply of the market, government intervention programs and major market share. The micro indicators GDP growth, market size, market volume etc. We also understand nuances specific to each country like US, Canada, India, Germany, etc. and have worked across 60+ countries and hence no only understand global trends, but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

Data Triangulation & Validation:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases and the company level averages etc.

We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate market

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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

Presentation & Reporting: The market research report is presented in different forms such as charts by using a scientific approach for easy understanding. Historic, current and future analysis is provided for each market in terms of both value and volume. The size of the market is interpreted in the US Dollar value and the respective unit, based on the product, for volume consumptions.
The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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