Market Overview

Phenylketonuria | A Pipeline Analysis Report 2018 | Technavio


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

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

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

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.

This report is available at a USD 1,000 discount for a limited time
market snapshot before purchasing

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

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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