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NBI-827104 is an investigational, potent, selective, and orally active small molecule that acts as a brain-penetrating T-type calcium channel blocker. It selectively inhibits the three T-type voltage-gated calcium channel subtypes (Cav3.1, Cav3.2, Cav3.3). The drug is being developed primarily for pediatric patients with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS), a rare childhood epilepsy syndrome associated with neurodevelopmental impairment and cognitive deficits[1][2][4][6][7]. NBI-827104 has also been evaluated in clinical trials for essential tremor in adults[6][7][8]. The drug was generally well tolerated in clinical studies but failed to meet its primary endpoint in a Phase 2 trial for EECSWS[4].
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