Potential Treatment for Spinocerebellar Ataxia, CAD-1883, Granted Orphan Drug Status

The Food and Drug Administration (FDA) has granted orphan drug designation to CAD-1883 (Cadence Pharmaceuticals, San Diego, CA), an investigational treatment for spinocerebellar ataxia (SCA).The drug is also under investigation for treatment of essential tremor and other movement disorders. 

In preclinical trials, CAD-1883 was shown to regulate neuronal firing, reduce tremor, and improve motor control. Phase 1 dose escalation studies are ongoing with tolerability seen at all doses and minimal transient adverse effects. A phase 2 clinical trial (NCT03688685) has begun in participants with essential tremor, and a phase 2 trial for SCA is expected to begin in the second half of 2019.

A novel first-in-class compound, CAD-1883 is a selective positive allosteric modulator of small-conductance, calcium-activated potassium channels (SK channels). 

“We are pleased to obtain orphan drug Designation for CAD-1883 in SCA, a progressively debilitating disease for which there are currently no approved treatments,” said Jodie Morrison, CEO, Cadence Therapeutics. “This deeply underserved patient population deserves new therapies, and we look forward to advancing CAD-1883, our therapy that holds great potential for addressing their unmet needs.”