Phenylketonuria (PKU) Drug Pipeline Analysis Report 2025

Phenylketonuria (PKU) is a rare genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase, leading to the accumulation of phenylalanine in the blood. Globally, the prevalence of phenylketonuria is estimated to be about 1 in every 10,000 newborns. There is a significant unmet clinical need for more effective therapies, as current treatments, such as dietary restrictions and supplementation, are often inadequate. Innovations in gene therapies and enzyme replacement treatments show promise in addressing these limitations. Additionally, the rising focus on personalized medicine and advanced biotechnology is expected to drive pipeline growth and improve outcomes for individuals with phenylketonuria in the coming years.

Report Coverage

The Phenylketonuria (PKU) Drug Pipeline Insight Report by the publisher gives comprehensive insights into phenylketonuria (PKU) therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for phenylketonuria (PKU). The phenylketonuria (PKU) report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The phenylketonuria (PKU) pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with phenylketonuria (PKU) treatment guidelines to ensure optimal care practices.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to phenylketonuria (PKU).

Phenylketonuria (PKU) Drug Pipeline Outlook

Phenylketonuria (PKU) is a rare genetic disorder caused by mutations in the PAH gene, leading to a deficiency in the enzyme phenylalanine hydroxylase. This deficiency results in the accumulation of phenylalanine, which can cause brain damage if untreated. The drug pipeline for phenylketonuria is focused on innovative treatments like gene therapies and enzyme replacements to address the underlying metabolic dysfunction.

Phenylketonuria (PKU) is traditionally managed with a strict low-phenylalanine diet and supplementation of amino acids. Newer therapies, such as enzyme substitution therapies and gene editing, aim to restore normal enzyme function and offer more effective, less restrictive solutions. These novel treatments promise to improve the quality of life for patients.

Phenylketonuria (PKU) Epidemiology

A 2024 report suggests that the incidence of Phenylketonuria (PKU) varies globally. Worldwide, PKU affects approximately 1 in 23,930 newborns. In the United States., the incidence ranges from 1 in 13,500 to 25,000, with African American populations showing lower rates. In Europe, rates vary significantly, from 1 in 2,700 in Italy to under 1 in 100,000 in Finland. Asia shows the lowest rates, with China being an exception.

Phenylketonuria (PKU) - Drug Pipeline Therapeutic Assessment

This section of the report covers the analysis of phenylketonuria (PKU) drug candidates based on several segmentations, including:

By Phase

• Late-Stage Products (Phase 3 and Phase 4)
• Mid-Stage Products (Phase 2)
• Early-Stage Products (Phase I)
• Preclinical and Discovery Stage Products

By Drug Class

• Small Molecules
• Gene Therapies
• RNA-based Therapies

By Route of Administration

• Oral
• Parenteral
• Others

Phenylketonuria (PKU) - Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to analysis, phase I and phase II cover a major share of the total phenylketonuria (PKU) clinical trials.

Phenylketonuria (PKU) - Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the phenylketonuria (PKU) pipeline analysis include small molecules, gene therapies, and RNA-based therapies. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for phenylketonuria (PKU).

Phenylketonuria (PKU) Clinical Trials Therapeutic Assessment - Competitive Dynamics

The phenylketonuria (PKU) drug report insights include the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in phenylketonuria (PKU) clinical trials:

• Sanofi
• PTC Therapeutics
• Jnana Therapeutics
• NGGT (Suzhou) Biotechnology Co., Ltd.
• BioMarin Pharmaceutical
• Erytech Pharma SA.
• Synlogic Inc.

Phenylketonuria (PKU) Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for phenylketonuria (PKU). It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of phenylketonuria (PKU) drug candidates.

Drug: RDD to Palynziq

BioMarin Pharmaceutical is sponsoring a Phase 4 rapid drug desensitization (RDD) study for adults with phenylketonuria experiencing hypersensitivity reactions to Palynziq. The objective is to improve drug tolerability and treatment persistence. Expected to conclude by November 2025, the study involves 10 participants and includes up to 30 weeks of screening, RDD, and follow-up.

Drug: Sepiapterin

The Phase 3b open-label study, sponsored by PTC Therapeutics, aims to evaluate the long-term efficacy of sepiapterin in preserving neurocognitive function in children with PKU when treatment is initiated early in childhood. The study is expected to be completed by February 28, 2031, and will enroll approximately 56 participants.

Drug: JNT-517 Tablet

This Phase 2 clinical study, sponsored by Jnana Therapeutics, aims to evaluate the safety, tolerability, and pharmacokinetics of oral JNT-517 in adolescent participants with phenylketonuria. Approximately 10 participants will be involved, receiving either JNT-517 or a placebo. The study is expected to be completed by September 2025, with primary results anticipated by July 2025.

Drug: BMN 307

This AAV gene therapy study for phenylketonuria is sponsored by BioMarin Pharmaceutical. The objective of this Phase 1/2 study is to evaluate the safety, efficacy, and tolerability of BMN 307 in adult subjects with phenylalanine hydroxylase (PAH) deficiency. The study is expected to be completed by December 2027, with an estimated 100 participants involved.

Reasons To Buy This Report

The Phenylketonuria (PKU) Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for phenylketonuria (PKU). It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into phenylketonuria (PKU) collaboration, regulatory environments, and potential growth opportunities.

Key Questions Answered in the Phenylketonuria (PKU) - Pipeline Insight Report

• Which companies/institutions are leading the phenylketonuria drug development?
• What is the efficacy and safety profile of phenylketonuria pipeline drugs?
• Which company is leading the phenylketonuria pipeline development activities?
• What is the current phenylketonuria commercial assessment?
• What are the opportunities and challenges present in the phenylketonuria drug pipeline landscape?
• What is the efficacy and safety profile of phenylketonuria pipeline drugs?
• Which company is conducting major trials for phenylketonuria drugs?
• Which companies/institutions are involved in phenylketonuria collaborations aimed at providing enhanced therapeutic alternatives for patients?
• What are the geographies covered for clinical trials in phenylketonuria?

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