Neurocrine Biosciences has announced that FDA has granted breakthrough therapy designation for NBI-98854 (Vesicular Monoamine Transporter 2 inhibitor) drug in tardive dyskinesia. A breakthrough therapy designation is granted for a drug that is intended to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on clinically significant endpoints over available therapies. The breakthrough designation also allows intensive discussions with the FDA which are intended to expedite the development and review process of eligible drugs.
"We are pleased that after reviewing our dataset the FDA recognizes that NBI-98854 is potentially an important therapy for the treatment of tardive dyskinesia, and we look forward to working closely with the Division of Psychiatry to advance our development program," said Christopher F. O'Brien, Chief Medical Officer of Neurocrine Biosciences.
AdvertisementThe Breakthrough Therapy Designation was granted, in part, based on the results of the Phase IIb Kinect studies of NBI-98854 in approximately 220 patients with tardive dyskinesia. Data from the Phase IIb program were presented in June 2014 at the Annual Congress of Parkinson's Disease and Movement Disorders in Stockholm, Sweden.
VMAT2 is a protein concentrated in the human brain that is primarily responsible for re-packaging and transporting monoamines (dopamine, norepinephrine, serotonin, and histamine) in pre-synaptic neurons. NBI-98854, developed in the Neurocrine laboratories, is a novel, highly-selective VMAT2 inhibitor that modulates dopamine release during nerve communication, while at the same time having minimal impact on the other monoamines, thereby reducing the likelihood of "off-target" side effects. NBI-98854 is designed to provide low, sustained, plasma and brain concentrations of active drug to minimize side effects associated with excessive monoamine depletion.
Modulation of neuronal dopamine levels in diseases such as tardive dyskinesia, Tourette syndrome, Huntington's chorea, schizophrenia, and tardive dystonia, which are characterized, in part, by a hyperdopaminergic state, should provide symptomatic benefits for patients with these diseases.
The Company recently initiated a Phase III study of NBI-98854 in tardive dyskinesia, the Kinect 3 study. The Kinect 3 study, along with the previous efficacy studies of NBI-98854, is designed to complete the placebo-controlled clinical efficacy evaluation of NBI-98854 for tardive dyskinesia. The Company also intends to conduct a separate one-year open-label safety study of NBI-98854 to support the anticipated 2016 filing of a New Drug Application with the FDA in tardive dyskinesia.
In addition to the tardive dyskinesia clinical effort, the Company has recently initiated a clinical study assessing NBI-98854 in children and adolescents with Tourette syndrome.
About Tardive Dyskinesia
Tardive dyskinesia is characterized by involuntary, repetitive movements of the extremities: lip smacking, grimacing, tongue protrusion, facial movements or blinking, puckering and pursing of the lips, or involuntary movements of the limbs. These symptoms are rarely reversible and there are currently no approved treatments.