Phenylketonuria Therapeutics Market Size in the 7MM was 700 USD Million in 2023, is expected to grow by 2034, estimated DelveInsight

DelveInsight's 'Phenylketonuria Market Insights, Epidemiology, and Market Forecast-2034' report deliver an in-depth understanding of the Phenylketonuria, historical and forecasted epidemiology as well as the PKU market trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Discover Key Insights into the Phenylketonuria Market with DelveInsight's In-Depth Report @ Phenylketonuria Market Size [https://www.delveinsight.com/sample-request/phenylketonuria-pku-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Key Takeaways from the Phenylketonuria Market Report

* In November 2024:- PTC Therapeutics- A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin. The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
* In November 2024:- Sanofi- This is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria (PKU) on a chronic, stable diet. The purpose of the study is to evaluate the safety and efficacy of SAR444836 in reducing phenylalanine (Phe) levels and in the elimination of a Phe restricted diet.
* In November 2024:- NGGT (Suzhou) Biotechnology Co., Ltd.- This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is a rAAV8 based vector carrying a functional copy of the human PAH gene.
* The majority of PKU cases are diagnosed in infants due to the high rate of newborn screening. The agespecific data revealed that most patients affected with PKU were less than or equal to 14 years, with nearly 60% cases in the 7MM in 2023.
* According to DelveInsight's estimates, missense mutation cases accounted for 60%, and nonsense mutations for 5% of the total diagnosed prevalent cases of PKU in 2023 in the 7MM.
* In EU4 and the UK, Germany had the highest number of diagnosed prevalent cases of PKU, followed by France. While Spain had the least number of cases in 2023.
* In 2023, classical PKU recorded the highest number of cases with approximately 11,000 cases based on severity-specific cases of PKU in the United States.
* The leading Phenylketonuria Companies such as BioMarin Pharmaceutical, Synlogic, PTC Therapeutics, Jnana Therapeutics, Homology Medicines, Inc, Nestle Health Science, Moderna, SOM Biotech, Agios Pharmaceuticals, APR Applied Pharma Research, American Gene Technologies, Generation Bio , and others.
* Promising Phenylketonuria Therapies such as BMN 307, SYNB1934v1, PTC923, JNT-517, HMI-102, HMI-103 , and others.

Stay ahead in the Phenylketonuria Therapeutics Market with DelveInsight's Strategic Report @ Phenylketonuria Market Outlook [https://www.delveinsight.com/sample-request/phenylketonuria-pku-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Phenylketonuria Epidemiology Segmentation in the 7MM

* Total Phenylketonuria Diagnosed Prevalent Cases
* Phenylketonuria Mutation Type-specific Cases
* Phenylketonuria Age-Specific Cases
* Phenylketonuria Severity-Specific Cases

Download the report to understand which factors are driving Phenylketonuria epidemiology trends @ Phenylketonuria Prevalence [https://www.delveinsight.com/sample-request/phenylketonuria-pku-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Phenylketonuria Marketed Therapies

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

KUVAN is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4) responsive PKU and is to be used in conjunction with a Phe-restricted diet. In August 2021, NICE issued final draft guidance which recommends sapropterin (also called KUVAN) as an option for treating PKU in pregnant women until they give birth and for treating the condition in people until they turn 22.

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

PALYNZIQ (pegvaliase-pqpz) injection is the first FDA-approved enzyme substitution therapy for adults with PKU who have uncontrolled blood Phe levels above 600 mol/L (10 mg/dL) on their current treatment. PALYNZIQ is a once-daily self-administered therapy that acts independently of the phenylalanine hydroxylase (PAH) enzyme, so it is an option for all eligible adult patients living with PKU. In October 2020, the US FDA approved the supplemental biologics license application to increase the maximum allowable dose of 60 mg with PALYNZIQ Injection for treating adults with PKU.

Phenylketonuria Emerging Therapies

* Sepiapterin (PTC923): PTC Therapeutics

PTC923 is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products. Sepiapterin reductase plays an enzymatic role in the biosynthesis of tetrahydrobiopterin, which is reported in limited studies to regulate the progression of several tumors. PTC has submitted an MAA to the EMA for sepiapterin for the treatment of PKU in March 2024. The company also expects to submit an NDA to the FDA for sepiapterin by the third quarter of 2024 and to complete regulatory submissions in Japan in 2024.

* SYNB1934: Synlogic

SYNB1934 is an orally administered, non-systemically absorbed drug candidate being studied as a potential biotherapeutic for phenylketonuria (PKU), an inherited metabolic disease marked by an inability to break down the amino acid phenylalanine (Phe), which can be neurotoxic. In July 2023, the US FDA has granted fast track designation to labafenogene marselecobac (previously known as SYNB1934) for the treatment of phenylketonuria (PKU). Currently, it is being investigated under Phase III clinical trial.

Get In-Depth Knowledge on Phenylketonuria Market Trends and Forecasts with DelveInsight @ Phenylketonuria Treatment Market [https://www.delveinsight.com/sample-request/phenylketonuria-pku-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Phenylketonuria Market Outlook

The goal of treatment for PKU is to keep plasma phenylalanine levels within 120360 mol/L (26 mg/dL). This is generally achieved through a carefully planned and monitored diet. In 2007, the US FDA approved KUVAN (sapropterin hydrochloride) to treat PKU. KUVAN is an oral pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates the activity of the residual PAH enzyme to metabolize phenylalanine into tyrosine. KUVAN is to be used in conjunction with a phenylalanine-restricted diet. KUVAN is manufactured by BioMarin Pharmaceutical; however, it does not work for everyone with PKU. It is most effective in children with mild cases of PKU.

Phenylketonuria Market Dynamics

The phenylketonuria market dynamics have been experiencing significant changes over recent years. One key driver of the phenylketonuria market is the increasing awareness and early diagnosis of the disorder. Advances in medical technology and genetic testing have enabled healthcare professionals to identify phenylketonuria cases more accurately and at an earlier stage, allowing for timely interventions and better disease management.

Unlock Strategic Insights with DelveInsight's Comprehensive Phenylketonuria Market Report @ Phenylketonuria Market Drivers and Barriers [https://www.delveinsight.com/sample-request/phenylketonuria-pku-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Scope of the Phenylketonuria Market Report

* Coverage- 7MM
* Study Period- 2020-2034
* Phenylketonuria Companies- BioMarin Pharmaceutical, Synlogic, PTC Therapeutics, Jnana Therapeutics, Homology Medicines, Inc, Nestle Health Science, Moderna, SOM Biotech, Agios Pharmaceuticals, APR Applied Pharma Research, American Gene Technologies, Generation Bio, and others.
* Phenylketonuria Therapies- BMN 307, SYNB1934v1, PTC923, JNT-517, HMI-102, HMI-103, and others.
* Phenylketonuria Market Dynamics: Phenylketonuria Market Drivers and Barriers
* Phenylketonuria Unmet Needs, KOL's views, Analyst's views, Phenylketonuria Market Access and Reimbursement

Table of Content

1. Key Insights

2. Report Introduction

3. Phenylketonuria Market Overview at a Glance

4. Executive Summary

5. Key Events

6. Disease Background and Overview

7. Methodology

8. Epidemiology and Patient Population

9. Patient Journey

10. Marketed Drugs

11. Emerging Drugs

12. Phenylketonuria (PKU): Seven Major Market Analysis

13. KOL Views

14. SWOT Analysis

15. Unmet needs

16. Market Access and Reimbursement

17. Appendix

18. DelveInsight Capabilities

19. Disclaimer

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=phenylketonuria-therapeutics-market-size-in-the-7mm-was-700-usd-million-in-2023-is-expected-to-grow-by-2034-estimated-delveinsight]
Phone: 9650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/case-study/respiratory-domain-conference-coverage



This release was published on openPR.