Phenylketonuria (PKU) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Phenylketonuria (PKU) Market Outlook

Thelansis’s “Phenylketonuria (PKU) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Phenylketonuria treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Phenylketonuria (PKU) Overview

Phenylketonuria (PKU) is a genetic disorder characterized by a deficiency in phenylalanine hydroxylase (PAH), resulting in impaired phenylalanine (Phe) metabolism. Most cases of PKU and hyperphenylalaninaemia (HPA) stem from mutations in the PAH gene located on chromosome 12q23.2. If left untreated, PKU leads to an abnormal phenotype, including seizures, diminished skin pigmentation, microcephaly, growth retardation, global developmental delay, and severe intellectual impairment. However, thanks to the implementation of newborn screening programs and early dietary interventions, children born with PKU can now anticipate relatively normal lives. PKU patients also have alternative treatment options, such as pharmacological treatment with BH4 (sapropterin) and enzyme substitution therapy involving Phe ammonia lyase (PAL).

• In the United States, the reported incidence of PKU varies between 1 to 2 cases in every 20,000 newborns.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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