OPKO Health, through its subsidiaries, Eirgen Pharma Limited, Ireland and OPKO Pharmaceuticals LLC, has received notification from the European Commission designating OPKO's oligonucleotide-based AntagoNAT (CUR-1916) an orphan medicinal product under Regulation (EC) No 141/2000 for the treatment of Dravet Syndrome. An orphan drug application is under review by the US FDA. There is currently no approved treatment for Dravet Syndrome in the U.S.

AntagoNAT, anti-Natural Antisense Transcripts, is an OPKO platform technology in which single strand oligonucleotide molecules are designed to interfere with regulatory gene expression in order to enhance production of endogenous functional proteins.  The AntagoNAT technology was part of CURNA Pharmaceuticals, acquired by OPKO in 2011, and then further developed in OPKO's Miami research laboratories under the direction of Dr. Jane Hsiao, Ph.D., OPKO's vice chairman and chief technical officer.

OPKO has studied over 250 genes and confirmed involvement of natural antisense transcripts (NAT) in their regulatory pathways. Of those, 89 genes were demonstrated to be subject to significant upregulation of mRNA in in vitro screening, and 7 AntagoNAT oligonucleotides have been validated in vivo to date. OPKO plans to initiate a clinical trial of CUR-1916 for treatment of Dravet Syndrome this year.

It is worth noting that several oligonucleotide compounds have been reported to be in late phase clinical development and one has been approved by the FDA in 2016. They work by down regulating transcription (antisense) or by correcting gene defects.

The European Medicines Agency (EMA) grants Orphan Designation to medicines intended to treat, prevent or diagnose life threatening and debilitating diseases, with a prevalence no greater than five in 10,000 in the EU, and for which no satisfactory method of treatment, prevention or diagnosis exists, and the proposed medicine offers significant medical benefit to those with the condition. Following Orphan Designation, sponsors can access a number of incentives including market exclusivity for a ten-year period following approval, protocol assistance, and potential fee reductions.

On November 24, 2016, OPKO requested Scientific Advice for the clinical development strategy of CUR-1916, and Committee for the Medicinal Products for Human Use (CHMP) has since met with OPKO and provided its advice for the CUR-1916 clinical development program. This is an important milestone for initiating a clinical trial of CUR-1916 designed to assess drug safety and significant medical benefits to patients with Dravet Syndrome.