OPKO Receives FDA Orphan Drug Status for its New Oligonucleotide to Treat Genetic Neurological Disorder
MIAMI, March 22, 2017 (GLOBE NEWSWIRE) -- OPKO Pharmaceuticals LLC, a subsidiary of OPKO Health, Inc. (OPK) announces that the Company has received orphan drug designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for OPKO’s oligonucleotide-based AntagoNAT (CUR-1916) for the treatment of Dravet Syndrome. Currently, there is no approved treatment for Dravet Syndrome in the U.S. On March 7, 2017, OPKO Health received orphan drug designation for CUR-1916 for the treatment of Dravet Syndrome from the European Commission.
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.
About FDA Orphan Drug Designation
Under the Orphan Drug Act (ODA) the FDA grants Orphan Drug status to drugs, vaccines, and diagnostic agents intended to treat a disease affecting less than 200,000 American citizens. Under the ODA, orphan drug sponsors qualify for seven-year FDA administered market Orphan Drug Exclusivity, tax credits of up to 50% of R&D costs, R&D grants, waived FDA fees, protocol assistance and may get clinical trial tax incentives.
What is Dravet Syndrome?
Dravet Syndrome, also called severe myoclonic epilepsy of infancy (SMEI), is a severe form of epilepsy that affects children and adults. It is caused by defects in the SCN1A genes (voltage gated sodium channel) required for the proper function of brain cells.
In Dravet Syndrome, seizures begin in the first year of life, and are most often associated with elevated body temperature (febrile convulsions). Later, other types of seizures occur, including status epilepticus (seizures lasting at least 5 minutes and requiring emergency medical care). From age 2, the child’s development begins to decline or reverse, and results in impaired mental and motor skills, leading to long term disability. Dravet Syndrome also qualifies as a Rare Pediatric Disease under Section 529 of Food, Drug, and Cosmetic Act (the FD&C Act).
Dravet Syndrome is debilitating and the death rate is reported to be 10-15%.
