Prader-Willi Syndrome - A Pipeline Analysis Report

Overview of the drug development pipeline for Prader-Willi-syndrome

It has been observed that the Prader-Willi-syndrome (PWS) is a disorder caused by the loss of active genes in a specific region of chromosome 15 and is characterized by the lack of feeling of fullness in people with PWS. The common symptoms of Prader-Willi-syndrome are hypotonia (poor muscle tone), hyperphagia (chronic overeating), mild to moderate intellectual impairment and learning disabilities, behavioral problems, and unusual fair skin and light-colored hair.

According to tests carried out by Fluorescence in situ hybridization (FISH), the cause of PWS has been identified to be the deleted genes in the chromosomes of the patients with PWS. Another testing method for PWS genetic testing is DNA methylation testing for the diagnosis of Prader-Willi-syndrome. As per the data published by the EMA based on the Committee for Orphan Medical Records (COMP), it is estimated that around 0.4 people in 10,000 people are affected by the symptoms of Prader-Willi-syndrome in the EU. Monotherapy is considered to be the most efficient approach for the treatment of Prader-Willi-syndrome as it uses a single drug to treat the disorder.

As per our pipeline analysis report, all of the drug molecules in the pipeline are being developed for the treatment of PWS. 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.

Want a bigger picture? Try a FREE sample of this report now!

Pipeline analysis report on drug development for PWS: 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 prader-willi-syndrome. 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

• Biospherics
• Dover Life Sciences
• GeneScience Pharmaceuticals
• GLWL Research
• INSYS THERAPEUTICS

Therapeutic assessment of the drug development pipeline for PWS by route of administration

• Oral
• Subcutaneous
• Intranasal
• Parenteral
• Unspecified

The oral route of administration (ROA) involves the administration of the drug molecules by placing them in the mouth cavity and in the subcutaneous method, the drug is delivered into the subcutis, which will have a more direct effect on the target cells.

Therapeutic assessment of the drug development pipeline for PWS by therapy

• Monotherapy
• Combination therapy

According to this pipeline analysis report, all the molecules that are currently in the drug pipeline for PWS 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 PWS?
• What are the companies that are currently involved in the development of drug molecules for PWS?
• 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.