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news.The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to the intravenous (IV) formulation of Marinus Pharmaceuticals' CNS-selective GABAA modulator, ganaxolone, for the treatment of status epilepticus.
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A Phase 1 clinical trial evaluating the safety, tolerability and pharmacokinetics of ganaxolone IV is expected to initiate in the first half of 2016.
Marinus Pharmaceuticals CEO Christopher Cashman said: "Status epilepticus is a life-threatening condition, associated with high mortality and if not treated immediately, can result in brain damage, cognitive impairment and death.
"We are pleased to receive the Orphan Drug Designation for ganaxolone IV in this difficult to treat seizure disorder with limited treatment options."
Orphan Drug Designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S.
The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.