FDA Grants Orphan Drug Designation to Insys’ Pharmaceutical CBD in Pediatric Schizophrenia

Specialty pharmaceutical company, Insys Therapeutics’ cannabidiol program has been granted its fifth orphan drug designation.

The company recently announced that its pharmaceutical cannabidiol (CBD) was granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for treatment of pediatric schizophrenia. Prior to pediatric schizophrenia, Insys’ pharmaceutical CBD was granted ODD for the treatment of glioma, glioblastoma multiforme (GBM), and two rare forms of pediatric epilepsy, Lennox-Gastaut Syndrome and Dravet Syndrome.

“Receiving orphan drug designation for CBD to treat pediatric schizophrenia is a lifeline for the thousands of children living with this disorder, and the parents who care for them,” said Michael L. Babich, President and Chief Executive Officer. “We are excited to explore the potential utility of our CBD candidate for this severe disorder alongside the other indications for which it has been granted ODD. We look forward to dosing our first human subjects with CBD in a Phase I clinical trial, which we expect to take place in early 2015.”

Pediatric schizophrenia is a severe brain disorder that causes a child to interpret reality abnormally and has a profound impact on a child’s behavior and development. The early age of onset of the disease causes special challenges for diagnosis, treatment, educational needs, and emotional and social development. Pediatric schizophrenia is associated with cognitive, behavioral and emotional issues, with symptoms including hallucinations, delusions and disordered thinking that impair a child’s ability to function. The disease has no cure, and current standard of care includes behavioral therapies and anti-psychotic medications.

ODD is granted to novel drugs or biologics designed to treat rare diseases or conditions which affect fewer than 200,000 patients in the US. The designation provides Insys with a seven-year period of US market exclusivity upon approval, as well as certain financial incentives aimed to help support its development.

The company is also evaluating its pharmaceutical CBD in several other indications including adult epilepsy, chemotherapy-induced peripheral neuropathy, and addiction in cocaine, amphetamines and opioids.

Source: Insys Therapeutics, Inc.

Last updated: 11/25/14; 10:10am EST