Dravet Syndrome - A Pipeline Analysis Report

Overview of the gene therapy pipeline for Dravet syndrome

Dravet syndrome is a rare genetic disorder of the brain. It is also known as the SCN1A-related seizure disorder. Though the cause is unknown, it is likely to occur because of mutations in genes. DS is mainly caused due to mutations in the SCN1A gene. Sodium channels present in the brain do not function properly because of the misfunction of the SCN1A gene. DS is a rare, lifelong form of epilepsy with prolonged febrile and non-febrile seizures that begin in the first year of life. Different types of seizures are observed in individuals with DS and the treatment for DS is quite complicated since it develops with age and no two patients respond to treatment the same way. According to the NIH, a study conducted in the US showed that there were 125,547 births in the study population, of which 730 infants had two or more seizure visits prior to the age of one year.

The rising incidences of Dravet syndrome will boost the need for drug development for the treatment of the disease. According to this pipeline analysis report, most of the drug molecules in the pipeline are currently 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 Dravet 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 –

• Biscayne Neurotherapeutics
• GW Pharmaceuticals
• Sage Therapeutics

Therapeutic assessment of the drug development for Dravet syndrome by route of administration

• Oral
• Intravenous/subcutaneous

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

Therapeutic assessment of the drug development for Dravet syndrome by therapy

• Monotherapy

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