Insys Therapeutics, Inc. (INSY – Snapshot Report) announced that the FDA has granted orphan drug designation to its pharmaceutical cannabidiol (CBD) candidate for the treatment of pediatric schizophrenia.
We note that the FDA grants orphan drug designation to candidates that are being developed to treat rare diseases or conditions affecting less than 200,000 people in the U.S. This status makes the candidate eligible for seven years of marketing exclusivity in the U.S. following approval. This designation also makes Insys eligible for certain financial benefits for developing pharmaceutical CBD.
We note that Insys already has orphan drug status for its CBD program for four other indications – glioma, glioblastoma multiforme, Lennox-Gastaut Syndrome and Dravet Syndrome.
The company plans to submit investigational new drug applications for CBD for Dravet Syndrome and Lennox-Gastaut Syndrome by the end of 2014. It intends to start dosing patients in a phase I study on CBD in early 2015.
Meanwhile, Insys is evaluating the possibility of developing CBD for additional indications including adult epilepsy, chemotherapy-induced peripheral neuropathy and addiction in cocaine, amphetamines and opioids. Insys intends to seek orphan drug status for its CBD in these indications, provided they qualify.
Currently, the company is preparing for the resubmission of its new drug application (NDA) for dronabinol oral solution. Last month, Insys had received a Refusal to File Letter from the FDA for the candidate owing to the inadequacy and incompleteness of the pediatric study plan submitted in its new drug application (NDA) in Aug 2014. With no additional studies necessary, the company expects to re-submit the NDA shortly. Insys is looking to get the candidate approved for the treatment of anorexia associated with weight loss in AIDS patients and chemotherapy-induced nausea and vomiting suffered by cancer patients who respond inadequately to conventional antiemetic …read more