Phenylketonuria (PKU) Market: Overview

Phenylketonuria refers to a genetic disorder and the said disease is inherited from one’s parents. In this very disease, 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 consequently, it damages the brain. This happens owing to mutations that occurs in the PAH gene, which leads to low levels of the enzyme known as phenylalanine hydroxylase. Mental health disorder, intellectual impairment, and hyperactivity are neurological symptoms of phenylketonuria. Order and skin rashes on the skin are also physiological symptoms of the phenylketonuria disease.  If both the parents are carrying PKU, there exists around 25% chance that their child would also be conceived with the said confusion; around 50% of chance that the kid would act as a transporter; and around 25% possibility that it would neither transport nor create illness. The basic approach for the treatment of PKU is to consume a specially made diet that cuts off the food that contains phenylalanine. Newborn babies with PKU can be very much fed with mother’s milk. They, for the most part, need to go through an uncommon recipe which is called Lofenalac. When the child has grown enough to consume solid food and the baby gets a chance to eat foods that is high in protein, it is needed to be avoided.         

Taking vendor landscape into consideration, the international market for Phenylketonuria (PKU) can be regarded as moderately fragmented and the market players has been focusing on strategically important moves like partnerships, mergers and acquisitions so as to strengthen their hold on the said market.

 Phenylketonuria (PKU) Market: Trends and Opportunities

On the basis of type, the international market for phenylketonuria has been categorized into benign PKU, classic PKU, and mild PKU. Furthermore, classic PKU is considered the most common kind of phenylketonuria that leads to severe damage of the brain. Based on the type of test, the said market has been segmented into screening test and diagnostic test. The segment of screening test has been expanding at a faster pace, as the screening test aids in the detection of the disease at the most treatable and its earliest possible stage, particularly for the newborn baby. Based on the type of treatment, the said market has been divided into drug therapy, gene therapy, dietary therapy, and many others. In addition to that, the segment of drug therapy has been yet again sub-divided into parenteral therapy and oral therapy. The category of drug therapy is expanding by accounting for a large share of the market, as various external drugs like sapropterin dihydrochloride are needed to fight the disease. The drug therapy further works efficiently by fulfilling the requirements and also maintaining the level of phenylalanine in the human blood. The world market for phenylketonuria in many of the matured economies is experiencing growth, due to high diagnostic rate of the disease in these countries, a large number of patients who suffer from phenylketonuria, and high awareness about the disease. However, issues that are related to the screening of newborn limit the phenylketonuria market.

Phenylketonuria (PKU) Market: Geography

Based on geography, the global phenylketonuria market has been segmented into North America, Europe, Middle East and Africa, Asia Pacific, and Latin America. The phenylketonuria treatment market in the region of North America is anticipated to account for a high market share over the period of forecast. Reasons that fuel the market are the growing number of newborns who suffer from phenylketonuria in the said region. Increased awareness amongst the people and better infrastructure of health care system in Europe is also expected to boost the said market in the region. The market in Asia Pacific is further estimated to expand at a substantial rate of growth over the period of forecast due to a large pool of patients and augmented research and development activities for the invention of drugs for phenylketonuria in the region. Besides, progressive economy and presence of newborn screening infrastructure and various screening programs flourish the phenylketonuria market in the region of Middle East & Africa.

Phenylketonuria (PKU) Market: Company Profiling

Some of the noteworthy market players that are operating in the international market for phenylketonuria are Codexis, Inc., American Gene Technologies International Inc., Erytech Pharma SA, BioMarin Pharmaceutical Inc., Dimension Therapeutics, Inc., SOM Innovation Biotech SL, Daiichi Sankyo Company, Limited, and Synthetic Biologics, Inc.

For the purpose of this study, the market has been segmented as follows:

  • Phenylketonuria (PKU) Market, by Types
    • Classic PKU
    • Mild PKU
    • Benign PKU
  • Phenylketonuria (PKU) Market, by Test
    • Diagnostic Test
    • Screening Test
  • Phenylketonuria (PKU) Market, by Treatment
    • Drug therapy
    • Dietary therapy
    • Gene therapy

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

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The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

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The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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