The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAD-1883, an investigational treatment for spinocerebellar ataxia (SCA), a genetic disorder characterized by progressive loss of coordination, slurred speech, difficulty controlling eye movements and cognitive dysfunction. Designed as a selective positive allosteric modulator of small-conductance, calcium-activated potassium ion channels (SK PAM), CAD-1883 has the potential to regulate neuronal firing and reduce disabilities in patients with SCA.
“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, Chief Executive Officer of Cadent 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.”
The FDA Office of Orphan Products Development grants Orphan Drug Designation to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The designation allows Cadent to qualify for a number of incentives, including seven years of market exclusivity upon regulatory approval, if received; exemption from FDA application fees for spinocerebellar ataxia; and tax credits for qualified clinical trials.
CAD-1883 is a first-in-class selective positive allosteric modulator of SK channels (small-conductance, calcium-activated potassium ion channels) in development for the treatment of movement disorders including essential tremor (ET) and spinocerebellar ataxia (SCA).
Spinocerebellar ataxia (SCA) is estimated to affect 11,000 people in the U.S. and an additional 15,500 people in the EU5 and Japan. SCA is a genetically defined degenerative disease with four identified genotypes affecting more than 50% of patients with SCA and is characterized by a progressive loss of coordination, abnormal speech, involuntary eye moment and cognitive dysfunction. There are no approved treatments available for SCA.