Phenylketonuria | A Pipeline Analysis Report 2018 | Technavio

Technavio has published a new report on the drug development pipeline for phenylketonuria, including a detailed study of the pipeline molecules. (Graphic: Business Wire)

LONDON--()--Technavio has announced their latest pipeline analysis report on the phenylketonuria market. The report includes a detailed analysis of the pipeline molecules under investigation within the defined data collection period to treat phenylketonuria.

This report by Technavio presents a detailed analysis of the market, including regulatory framework, drug development strategies, recruitment strategies, and key companies that are expected to play an essential role in the growth of the market in the future.

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Phenylketonuria: Market overview

Phenylketonuria is an inherited disorder that increases the level of a substance called phenylalanine (Phe) in blood. Phe is a building block of proteins and is obtained through the diet. It is found in all proteins and in some artificial sweeteners. If phenylketonuria is left untreated, Phe can build up to harmful levels in the body, causing intellectual disabilities and other serious health problems. The main symptoms of phenylketonuria include eczema, tremors, and recurrent vomiting. A carefully maintained diet can prevent intellectual disabilities, as well as neurological, behavioral, and dermatological problems.

According to a senior market research analyst at Technavio, “The treatment must be started at a very young age. However, some late-treated children also have recovered from the disease. As per the National Institutes of Health, the prevalence of PKU varies by country ranging from between one in 10,000 and one in 20,000 births in the US and Europe. The prevalence of the phenylketonuria in Arab countries was reported to be highest in Turkey and the lowest prevalence was reported in the UAE. With the increasing incidences of the disease, the need for the drug development for phenylketonuria is expected to increase considerably in the forthcoming years.”

Phenylketonuria: Segmentation analysis

This pipeline analysis report segments the phenylketonuria market based on therapies employed (monotherapy), RoA (oral, and intravenous), therapeutic modality (gene therapy, cell therapy, biological, small molecule, recombinant enzyme, polymer, and protein), targets (Phe hydroxylase, phe ammonia lyase, and phe), MoA (Phe hydroxylase activator, phe ammonia lyase replacement, phe hydroxylase stabilizer, phe hydroxylase replacement and phe inhibitor), geographical segmentation (US, and Australia) and recruitment status (not yet recruiting, and recruiting). It provides an in-depth analysis of the prominent factors influencing the market, including drivers, opportunities, trends, and industry-specific challenges.

Based on the mechanism of action (MoA), around 27% of the molecules that are being investigated for the treatment of phenylketonuria are phe hydroxylase activator.

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Some of the key topics covered in the report include:

Scope of the Report

Regulatory Framework

Drug Development Landscape

  • Drugs under development
  • Indications coverage

Drug Development Strategies

  • Therapies employed
  • RoA
  • Therapeutic modality
  • Geographical coverage

Recruitment Strategies

  • Recruitment status
  • Gender
  • Age

Key Companies

  • Type of players
  • Company overview

Discontinued or Dormant Molecules

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Technavio Research
Jesse Maida
Media & Marketing Executive
US: +1 844 364 1100
UK: +44 203 893 3200

Release Summary

Technavio has published a new report on the drug development pipeline for phenylketonuria, including a detailed study of the pipeline molecules.


Technavio Research
Jesse Maida
Media & Marketing Executive
US: +1 844 364 1100
UK: +44 203 893 3200