Neurocrine Biosciences acquired the exclusive rights to NBI-827104 from Idorsia Ltd. NBI-827104 is a potent, selective, orally active and brain penetrating T-type calcium channel blocker.
Epileptic Encephalopathy with Continuous Spike-and-Wave During Sleep (EE-CSWS) is a rare pediatric epilepsy. Typical onset occurs between ages 2 and 4 years old with seizures occurring infrequently. Diagnosis of EE-CSWS is based on a unique electroencephalographic (EEG) pattern for electrical status epilepticus in sleep (ESES), together with cognitive stagnation and regression. There is currently no approved treatment for the disorder.
Neurocrine Biosciences has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration for NBI-827104 in EE-CSWS.
Essential tremor is one of the most common movement disorders, with an estimated 10 million people living with essential tremor in the U.S. alone. It affects approximately 5% of adults aged 65 or older. Essential tremor begins gradually, often affecting the upper limbs (but can affect the head, voice, and legs), and commonly runs in families. It involves involuntary and rhythmic shaking of the limbs and other body parts during movement that can impact activities of daily living, including eating, drinking, writing, and dressing. Essential tremor may be mistaken for tremor in Parkinson disease, and can be aggravated by emotional stress, fatigue, or substances like caffeine. Beta-blockers or anti-seizure medications are often used to treat the disorder. However, many patients become resistant to these therapies over time. The only approved medication for essential tremor was approved in the 1970s.