OPKO announced that it has received Orphan Drug designation from the Food and Drug Administration (FDA) for its oligonucleotide-based AntagoNAT (CUR-1916) in the treatment of Dravet Syndrome. Currently, FDA approved treatments for this disorder are unavailable in the U.S.

AntagoNAT, anti-Natural Antisense Transcript, uses a mechanism of single strand oligonucleotide molecules designed to interfere with regulatory gene expression in order to enhance production of endogenous functional proteins. CUR-1916 works by upregulating a defective gene to increase the production of functional protein. 

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Dravet Syndrome or severe myoclonic epilepsy of infancy is caused by defects in the SCN1A genes required for the proper function of brain cells. Children with Dravet Syndrome begin to decline in their development at age 2, resulting in weakened mental and motor skills.

OPKO plans to initiate a clinical trial of CUR-1916 for treatment of Dravet Syndrome this year.

For more information visit OPKO.com.