Phenylketonuria - A Pipeline Analysis Report

Overview of the drug development for phenylketonuria

Phenylketonuria is an inherited disorder that increases the levels of a substance called phenylalanine (Phe) in the 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. The treatment must be started at a very young age. However, some late-treated children also have recovered from the disease. As per NIH, 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 highest prevalence was reported in Turkey and the lowest prevalence was reported in the UAE. However, with increasing incidences for the disease, the need for the drug development for phenylketonuria is expected to increase considerably in the forthcoming years.

According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for phenylketonuria. Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of phenylketonuria. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are –

• Agios
• BioMarin
• Codexis
• EryDel
• Erytech Pharma

Therapeutic assessment of the drug development for phenylketonuria by route of administration

• Oral
• Intravenous

The oral route of administration (ROA) involves the application of the drug directly through the mouth cavity, which will have a more direct effect on the target cells.

Therapeutic assessment of the drug development for phenylketonuria by therapy

• Monotherapy

According to this pipeline analysis report, all the molecules that are currently in the drug development pipeline for phenylketonuria are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.

Key questions answered in the report include

• What are the drug molecules in the various development stages for phenylketonuria?
• What are the companies that are currently involved in the development of drug molecules for phenylketonuria?
• Insight into discontinued/inactive molecules with appropriate reasoning?
• What are the major regulatory authorities approving drugs in various regions?
• Detailed profiling of each active molecule

Technavio also offers customization on reports based on specific client requirement.