Phenylketonuria (PKU) Market is Expected to Showcase a Significant Growth During the Forecast Period (2023-2032), Predicts DelveInsight | Moderna, SOM Biotech, Agios, Generation Bio, BioMarin

Phenylketonuria (PKU) Market is Expected to Showcase a Significant Growth During the Forecast Period (2023-2032), Predicts DelveInsight | Moderna, SOM Biotech, Agios, Generation Bio, BioMarin
DelveInsight Business Research LLP
As per DelveInsight, the Phenylketonuria Market is anticipated to evolve immensely in the coming years owing to the rise in the number of cases of Phenylketonuria and the launch of new therapies in the market. The pipeline for Phenylketonuria consists of promising drugs that are anticipated to change the treatment landscape of Phenylketonuria. The current market anticipates the emergence of products namely, PTC923 (PTC Therapeutics) and SYNB1934 (Synlogic).

DelveInsight’s “Phenylketonuria Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Phenylketonuria Market Size and Share in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The market report covers emerging drugs, treatment practices, market share of individual Phenylketonuria therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Phenylketonuria treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of opportunities and assess the underlying potential of the market. 

Phenylketonuria: An Overview

Phenylketonuria (Phenylketonuria) is a rare genetic condition that causes an amino acid called phenylalanine to build up in the body; amino acids are the building blocks of protein. Phenylalanine is found in all proteins and some artificial sweeteners. A less severe form of Phenylketonuria is called variant Phenylketonuria or non-Phenylketonuria hyperphenylalaninemia. This occurs when the baby has too much phenylalanine in its body. Infants with this form of the disorder may have only mild symptoms.

The treatment approach depends on the severity of the person. The first line of treatment includes a low-protein diet. Infants need a special formula to control the amount of protein they eat. Medications and enzyme replacement therapy are also useful in helping the body to break down phenylalanine or to help the body tolerate it better.

Currently, there are only two approved therapies available in the market landscape. The first is sapropterin, a synthetic form of tetrahydrobiopterin (BH4), which is a cofactor in the catabolism of phenylalanine. It acts by increasing PAH activity in patients who have residual enzyme function. The second therapy is pegvaliase (PALYNZIQ), a phenylalanine ammonia-lyase (PAL) enzyme that temporarily restores the levels of deficient enzyme and reduces blood phenylalanine concentrations by converting phenylalanine to ammonia and trans-cinnamic acid. 

Phenylketonuria Market Key Facts

  • The Phenylketonuria Market size in the United States was approximately USD 410 million in 2022 and is projected to grow during the forecast period (2023–2032).

  • A few players like PTC Therapeutics (PTC923), Synlogic (SYNB1934), and others are evaluating their lead candidates for the treatment of Phenylketonuria (Phenylketonuria). These therapies are expected to change the Phenylketonuria (Phenylketonuria) treatment landscape.

  • According to the estimates, Japan recorded the least market share, i.e., around 1% of the total market size of phenylketonuria among the 7MM in 2022.

  • Among the EU4 (Germany, France, Italy, Spain) and the UK, Germany had the maximum revenue share in 2022, while Spain had the lowest market share.

  • In 2022, sapropterin hydrochloride captured the highest market size of Phenylketonuria (Phenylketonuria) in the 7MM.

  • In 2022, among the 7MM, the United States accounted for the highest number of diagnosed prevalent cases of Phenylketonuria.  

  • EU4 and the UK accounted for ~31,300 cases in 2022, which is expected to increase by 2032. 

  • As per the age-specific cases of Phenylketonuria, the ≤14 years age group accounted for ~50% of cases of Phenylketonuria in the United Kingdom.

Phenylketonuria Market

The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Phenylketonuria therapies in the market. It also provides a detailed assessment of the Phenylketonuria market drivers & barriers, unmet needs, and emerging technologies.

The report gives complete detail of the market trend for each marketed Phenylketonuria drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MOA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.

Phenylketonuria Epidemiology

The epidemiology section covers detailed insights into the historical, and current Phenylketonuria patient pool and forecasted epidemiology trends for every seven major countries (7MM)  from 2019 to 2032.

The Report Covers the Phenylketonuria Epidemiology Segmented as-

  • Total Diagnosed Prevalent Cases of Phenylketonuria in the 7MM [2019-2032]

  • Age-Specific Cases of Phenylketonuria in the 7MM [2019–2032]

  • Severity-Specific Cases of Phenylketonuria in the 7MM [2019–2032]

  • Mutation type-specific Cases of Phenylketonuria in the 7MM [2019–2032]

Phenylketonuria Drugs Uptake and Pipeline Development Activities

The drugs’ uptake section focuses on the rate of uptake of the potential drugs recently launched in the Phenylketonuria market or expected to be launched during the study period. The analysis covers the market share of Phenylketonuria drugs, patient uptake by therapies, and sales of each drug. The report’s drugs uptake section helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs. It allows the comparison of the drugs based on market share and size, which again will be helpful in investigating factors important in understanding the market dynamics and in making financial and regulatory decisions.

The report also covers the Phenylketonuria Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Explore More About Ongoing Pipeline Development Activities @

Phenylketonuria Therapeutics Assessment

Several major pharma and biotech companies are developing therapies for Phenylketonuria. Currently, PTC Therapeutics is leading the therapeutics market with its Phenylketonuria drug candidates in the most advanced stage of clinical development.

Some of the leading Phenylketonuria Companies in the Therapeutics Market, include:

  • BioMarin Pharmaceutical

  • Homology Medicine

  • Jnana Therapeutics

  • Nestlé Health Science

  • Moderna

  • SOM Biotech

  • Agios Pharmaceuticals

  • APR Applied Pharma Research

  • American Gene Technologies

  • Generation Bio

  • PTC Therapeutics

And Many Others

Emerging and Marketed Phenylketonuria Therapies Covered in the Report Include:

  • Sepiapterin: PTC Therapeutics

  • SYNB1618: Synlogic

  • HMI-102: Homology Medicine

  • KUVAN (Sapropterin Hydrochloride): Asubio-Pharma/BioMarin-Pharmaceutical

  • PALYNZIQ (pegvaliase-pqpz/rAvPAL-PEG/BMN 165): BioMarin Pharmaceutical

And Many More

Learn More About the Emerging Therapies & Key Companies in the Phenylketonuria Therapeutics Market @

Table of Content (TOC)

1. Key Insights

2. Executive Summary 

3. Phenylketonuria Competitive Intelligence Analysis

4. Phenylketonuria Market Overview at a Glance

5. Phenylketonuria Background and Overview

6. Phenylketonuria Patient Journey

7. Phenylketonuria Epidemiology and Patient Population

8. Phenylketonuria Treatment Algorithm, Current Treatment, and Medical Practices

9. Phenylketonuria Unmet Needs

10. Key Endpoints of Phenylketonuria Treatment

11. Phenylketonuria Marketed Products

12. Phenylketonuria Emerging Therapies

13. Phenylketonuria Seven Major Market Analysis

14. Attribute Analysis

15. Phenylketonuria Market Outlook (7 major markets)

16. Phenylketonuria Access and Reimbursement Overview

17. KOL Views on the Phenylketonuria Market.

18. Phenylketonuria Market Drivers

19. Phenylketonuria Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

*The table of contents is not exhaustive; the final content may vary. 

Download the Sample PDF to Get Detailed Insights About the Report’s Offerings @


About DelveInsight

DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.


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