FDA Grants Orphan Drug Status to Investigational Treatment for Spinocerebellar Ataxia

The Food and Drug Administration (FDA) has granted Orphan Drug designation to CAD-1883 (Cadent Therapeutics), an investigational treatment for spinocerebellar ataxia (SCA).

CAD-1883 is a selective positive allosteric modulator of SK channels (small-conductance, calcium-activated potassium ion channels). SCA is a very rare genetic disorder that is associated with loss of coordination, abnormal speech, involuntary eye movement, and cognitive dysfunction.

The Company believes that the treatment has the potential to regulate neuronal firing and reduce disabilities in patients with SCA. A phase 2 trial is currently assessing safety, tolerability and efficacy in 20 patients with a history of tremor that fulfills the diagnostic criteria of essential tremor according to Movement Disorder Society (MDS) Consensus Statement on the classification of tremors from the task force on tremor of the International Parkinson and Movement Disorder Society.

“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 of Cadent Therapeutics.

Orphan designation is granted to therapies of diseases that affect fewer than 200,000 in the US. The designation means that should the treatment be eventually approved, the Company can qualify for 7 years market exclusivity.

For more information visit Cadenttx.com.

This article originally appeared on MPR