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.
The Phenylketonuria (PKU) Drug Pipeline Insight Report by Expert Market Research 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) 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.
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.
This section of the report covers the analysis of phenylketonuria (PKU) drug candidates based on several segmentations, including:
By Phase
By Drug Class
By Route of Administration
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 EMR analysis, phase I and phase II cover a major share of the total phenylketonuria (PKU) clinical trials.
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).
The EMR 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:
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.
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.
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.
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.
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.
*Please note that this is only a partial list; the complete list of drugs will be available in the full 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.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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Australia
63 Fiona Drive, Tamworth, NSW
+61-448-061-727
India
C130 Sector 2 Noida, Uttar Pradesh 201301
+91-723-689-1189
Philippines
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+63-287-899-028, +63-967-048-3306
United Kingdom
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+44-753-713-2163
Vietnam
193/26/4 St.no.6, Ward Binh Hung Hoa, Binh Tan District, Ho Chi Minh City
+84-865-399-124
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