Phenylketonuria Market to Exhibit Significant Growth, Assesses DelveInsight | Key Companies – BioMarin, Synlogic, PTC Therapeutics, Jnana Therapeutics, Homology Medicines

New York, USA, July 31, 2023 (GLOBE NEWSWIRE) — Phenylketonuria Market to Exhibit Significant Growth, Assesses DelveInsight | Key Companies – BioMarin, Synlogic, PTC Therapeutics, Jnana Therapeutics, Homology Medicines

The phenylketonuria market is expected to grow in 7MM in the coming years due to factors like an increase in the patient pool and the expected entry of emerging therapies by leading pharma and biotech companies. 

DelveInsight’s Phenylketonuria Market Insights report includes a comprehensive understanding of current treatment practices, phenylketonuria emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Key Takeaways from the Phenylketonuria Market Report

• As per DelveInsight’s analysis, the phenylketonuria market size in the 7MM was approximately USD 610 million in 2022.
• According to the assessment done by DelveInsight, the total diagnosed prevalent cases of phenylketonuria in the 7MM made up approximately 50.5K cases in 2022 and are projected to increase during the forecast period.
• Globally, leading phenylketonuria companies such as BioMarin Pharmaceutical, Synlogic, PTC Therapeutics, Jnana Therapeutics, Homology Medicines, Inc, Nestlé Health Science, Moderna, SOM Biotech, Agios Pharmaceuticals, APR Applied Pharma Research, American Gene Technologies, Generation Bio, and others are developing novel phenylketonuria drugs that can be available in the Phenylketonuria market in the coming years.
• Some of the key therapies for phenylketonuria treatment include BMN 307, SYNB1934v1, PTC923, JNT-517, HMI-102, HMI-103, and others. 

Discover which therapies are expected to grab the major phenylketonuria market share @ Phenylketonuria Market Report

Phenylketonuria Overview

Phenylketonuria (PKU) is a rare inherited metabolic disorder that affects the way the body processes the amino acid phenylalanine. Normally, an enzyme called phenylalanine hydroxylase converts phenylalanine into another amino acid called tyrosine. The main cause of phenylketonuria is a mutation in the PAH gene, which is responsible for producing phenylalanine hydroxylase. This genetic abnormality is usually passed down from both parents who carry the mutated gene, making phenylketonuria an autosomal recessive condition. 

Phenylketonuria symptoms usually become apparent in infancy or early childhood. Without treatment, the excessive build-up of phenylalanine can lead to intellectual disabilities, developmental delays, and other neurological problems. Other common phenylketonuria symptoms include seizures, skin rashes, and an unusual musty odor in the breath, skin, and urine, which is caused by the accumulation of phenylalanine by-products. Diagnosis of phenylketonuria is often performed through newborn screening programs, which are routine in many countries. A blood test is used to measure the levels of phenylalanine in a baby’s blood shortly after birth.

Phenylketonuria Epidemiology Segmentation

The phenylketonuria epidemiology section provides insights into the historical and current phenylketonuria patient pool and forecasted trends for the seven individual major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders.

The phenylketonuria market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:

• 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 Epidemiological Insights

Phenylketonuria Treatment Market 

The primary phenylketonuria treatment involves a strict and lifelong low-phenylalanine diet. Foods containing high levels of phenylalanine, such as meat, fish, dairy products, and certain grains, must be avoided. Instead, individuals with phenylketonuria consume a specialized formula that provides all the necessary nutrients without phenylalanine. This diet aims to maintain blood phenylalanine levels within a safe range, preventing the harmful effects of its buildup. Apart from dietary management, some phenylketonuria patients may also benefit from sapropterin dihydrochloride (Kuvan®), a medication that can help lower phenylalanine levels in some cases. However, its effectiveness varies among individuals, and not all phenylketonuria patients are responsive to this drug.

In 2018, PALYNZIQ (pegvaliase-pqpz) was approved by the FDA for adults with phenylketonuria. PALYNZIQ is an injectable enzyme therapy for patients with uncontrolled blood phenylalanine concentrations on current treatment. PALYNZIQ is manufactured by BioMarin Pharmaceutical.

Managing phenylketonuria requires constant monitoring by healthcare professionals, including regular blood tests to assess phenylalanine levels. The goal of phenylketonuria treatment is to allow affected individuals to lead normal, healthy lives while minimizing the risk of neurological complications. Compliance with the prescribed diet and regular medical supervision are crucial for successful phenylketonuria management and a better quality of life for those living with this rare genetic condition. Research continues to explore new therapies and potential gene-editing approaches to further improve phenylketonuria treatment outcomes in the future.

To know more about phenylketonuria treatment, visit @ Phenylketonuria Treatment Drugs 

Key Phenylketonuria Therapies and Companies

• BMN 307: BioMarin Pharmaceutical
• SYNB1934v1: Synlogic
• PTC923: PTC Therapeutics
• JNT-517: Jnana Therapeutics
• HMI-102: Homology Medicines, Inc
• HMI-103: Homology Medicines, Inc

Learn more about the FDA-approved drugs for phenylketonuria @ Drugs for Phenylketonuria Treatment 

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.

Moreover, research and development in the pharmaceutical industry have resulted in the introduction of new and innovative treatment options for phenylketonuria patients. Novel therapies, such as enzyme replacement therapies and gene therapies, have shown promising results in clinical trials, offering hope for improved outcomes and a better quality of life for phenylketonuria patients.

On the regulatory front, governments and health authorities are taking a keen interest in rare diseases like phenylketonuria, leading to more favorable policies, increased funding for research, and expedited approval processes for new phenylketonuria treatments. These efforts have contributed to a growing phenylketonuria market therapeutics and a rise in investment opportunities in the field.

However, challenges still persist in the phenylketonuria market, including limited patient awareness in certain regions, high treatment costs, and the need for ongoing monitoring and adherence to strict dietary restrictions. These factors can hinder the phenylketonuria market growth and access to proper care for phenylketonuria patients.

Scope of the Phenylketonuria Market Report

• Therapeutic Assessment: Phenylketonuria current marketed and emerging therapies
• Phenylketonuria Market Dynamics: Attribute Analysis of Emerging Phenylketonuria Drugs
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL’s views, Analyst’s views, Phenylketonuria Market Access and Reimbursement

Discover more about phenylketonuria drugs in development @ Phenylketonuria Clinical Trials

Table of Contents

Related Reports

Phenylketonuria Epidemiology Forecast

Phenylketonuria Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted phenylketonuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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Phenylketonuria Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key phenylketonuria companies, including 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, among others.

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