FDA Awards Orphan Status To Insys' Cannabidiol For GBM

Insys Therapeutics announced that the U.S. Food and Drug Administration (FDA) has awarded a second Orphan Drug Designation to its pharmaceutical product cannabidiol (CBD) for the treatment of glioblastoma multiforme (GBM).

Cannabidiol belongs to the group of at least 60 active cannabinoids present in cannabis. Insys’ CBD is more than 99.5 percent pure cannabidiol, synthesized to be identical to cannabis-extracted CBD. It is produced in a controlled environment. Insys stated that its CBD product seems to have anti-convulsive action in animal models.

Glioblastoma multiforme is the leading and most aggressive malignant primary brain tumor in humans. GBM is known for its high invasiveness and proliferation rate, as well as its strong resistance to standard chemotherapy and radiotherapy. Patients diagnosed with GBM have an average survival of 12 to 14 months in spite of existing treatments.

Michael Babich, president and CEO of Insys Therapeutics, said, “We are pleased to have received orphan drug designation for this aggressive and often incurable form of brain cancer. We look forward to advancing development of this product and offering a potential efficacious treatment for patients.”

Insys also announced that it has entered into an exclusive licensing agreement with California Pacific Medical Center on behalf of its Research Institute (CPMCRI) for the licensure of CPMCRI's patent rights for the usage of CBD in treating GBM. Dr. Sean McAllister at CPMCRI said, “Based on previous research conducted with CBD to treat brain tumors, we believe that there is supportive evidence for the use of CBD as an adjunct treatment in GBM and eagerly anticipate the results from our in-vivo models to further support clinical studies in humans.”

Aside from its orphan designation for glioblastoma multiforme, CBD has received orphan status for Dravet syndrome as well as Lennox-Gastaut Syndrome, both rare forms of epilepsy.

The company said it is assessing the potential use of CBD in other indications including chemotherapy-induced peripheral neuropathy, adult epilepsy, and substance addictions.