US FDA grants orphan drug status to OPKO’s new oligonucleotide to treat genetic neurological disorder

Orphan drug designation provides certain marketing exclusivity, tax credits for research and a waiver of the New Drug Application user fee. 

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, part of CURNA Pharmaceuticals, acquired by OPKO in 2011, was further developed in OPKO's Miami research laboratories under the direction of 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 seven 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.

Oligonucleotides are synthetic chemical compounds consisting of mixtures of modified DNAs and RNAs. Only five oligonucleotide compounds are approved by FDA for various indications and others have been reported to be in late phase clinical development. The majority of the compounds work by down regulating, or depressing transcription (anti-sense) or by correcting gene defects. CUR-1916 works by upregulating a defective gene to increase the production of functional protein.

Us fda grants orphan drug status, opko’s new oligonucleotide, treat genetic, neurological disorder