UK Phenylketonuria (PKU) Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2584

  • Format: PPT, PDF

UK Phenylketonuria Therapeutics Market: Segmented by Therapeutics, Route of Administration, and Distribution Channels – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

SKU: 2584 Categories: ,

Report Overview

The UK Phenylketonuria Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Phenylketonuria (PKU) is a rare inherited condition that causes a build-up of Phenylalanine. Phenylalanine (Phe) is one of 20 amino acids that make up the building blocks of protein. Proteins are made up of building blocks called amino acids. In PKU, the body cannot break down the amino acid phenylalanine it builds up in the blood and tissues. This build-up prevents the brain from developing properly. Progressive intellectual disability results if PKU is not treated from early infancy. With treatment, children with PKU can grow and develop normally. People with PKU need to follow a diet that limits foods with phenylalanine. The diet should be followed carefully and be started as soon after birth as possible. Food mainly suggested are Milk and cheese, Eggs, Nuts, Soybeans, Beans, Chicken, beef, or pork, Fish, Peas, Beer.

With an early diagnosis and the correct treatment, most children with PKU are able to live healthy lives. The prevalence of PKU is around 1:10,000 babies born in the UK. PKU affects approximately 70 babies each year. Males and females are affected equally by PKU in the UK. Parents of a child with PKU have about one in four chances of having another child with the debilitating condition.

Market Growth Drivers

Frequent research for the development of novel drugs for the treatment of PKU is expected to drive the growth of the PKU treatment market during the forecast period. Furthermore, increasing acquisitions and collaborations between key players are also expected to create a lucrative environment for the growth of the PKU treatment market during the forecast period.

In recent years, small and large pharmaceutical and nutraceutical companies have become increasingly dependent on contract research organizations (CROs) and other clinical services to provide research services to their R&D operation. Also, phenylketonuria treatment-based products and rising demand for essential and nonessential amino acids in the form of protein ingredients as well as finished products are expected to drive market growth during the forecast period.

Market Restraints

However, rising treatment costs and a lack of awareness among patients and physicians about phenylketonuria are among the major factors acting as restraints and will continue to pose a challenge to the PKU treatment market during the forecast period.


1. UK Phenylketonuria Therapeutics Market Overview…………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Frequent Research for Development of Novel Therapies
B. Acquisitions and Collaborations Creates a Lucrative Market
C. Rise in Prevalence and Incidence of Phenylketonuria
D. Increasing Demand for Essential and Non-essential Amino Acids
Market Restraints
A. Rising Treatment Costs
B. Lack of Awareness
3. Major Types of Phenylketonuria………………………………………………………
A. Hyperphenylalaninemia
B. Classic Phenylketonuria
C. Mild Phenylketonuria
D. Moderate Phenylketonuria
4. Phenylketonuria Therapeutics Market Segmentation………………..
A. By Therapeutics
I. Dietary Supplements
II. Kuvan
III. Pegvaliase
IV. Other Therapies
B. By Route of Administration
I. Oral
II. Intravenous
III. Subcutaneous
IV. Others
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
IV. Others
5. Phenylketonuria Major Drugs Market Share………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. BioMarin Company overview, Product & Services, Strategies & Financials
B. Merck Company overview, Product & Services, Strategies & Financials
C. Dimension Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………
A. New Trends and Development of Phenylketonuria Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapeutics:

On the basis of therapeutics, the PKU therapeutics market is segmented into dietary supplement, approved medications like Kuvan (sapropterin dihydrochloride) of BioMarin Pharmaceutical Inc. and Pegvaliase (PEGylated recombinant phenylalanine ammonia-lyase) is an investigational enzyme substitution therapy from Merck for the treatment of patients with phenylketonuria (PKU) who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l). The dietary supplement segment, among other therapeutics, accounts for a significant share in the industry owing to its increased preference among health care professionals to treat patients with phenylketonuria and also projected to gain more market share during the forecast period.

By Route of Administration:

Based on the route of administration, the PKU therapeutic market can be segmented into

  • Oral
  • Intravenous
  • Subcutaneous
  • Other

The oral route remains the more preferred route of administration over parenteral due to its ease of administration and convenience. The oral route has a major share as the Dietary segment is the mainstay as the most common preferred drug, Kuvan is an oral therapy indicated for the treatment of hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) and also anticipated during the forecast period.

By Distribution Channel:

In terms of distribution channels, the PKU therapeutic market has been bifurcated into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies
  • Others

The hospital pharmacies segment accounted for the highest market share and is anticipated to witness growth during the forecast period due to most of the major products are prescription products, which are available in hospital pharmacies. Thus, the potential growth of hospital pharmacies can be anticipated during the forecast period.

Top Major Players

The UK PKU therapeutic market is a great market to enter and generate more revenues. The prominent players in the market have adopted various strategies to garner maximum market share. Major players operating in the PKU therapeutic market include BioMarin Pharmaceutical Inc, Merck Corporation, Dimension Therapeutics are among others, and are expected to gain high market share during the forecast period.

Research Methodology

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  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
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  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

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