New and Noteworthy Information—October 2017

Orphan Drug Designation Granted for EPX-300

The FDA has granted Orphan Drug Designation for EPX-300 for the treatment of patients with Dravet syndrome. EPX-300 is a repurposed antidepressant that acts via modulation of serotonin signaling pathways. Researchers discovered its potential as a treatment for patients with Dravet syndrome using a phenotype-based zebrafish drug screening platform. Using the zebrafish model for Dravet syndrome, investigators identified drug candidates from a screen of more than 3,000 drugs that suppress seizures and other symptoms associated with neurologic diseases. Epygenix Therapeutics, which is developing EPX-300, is headquartered in Paramus, New Jersey.

FDA Approves Expanded Indication for Aptiom

The FDA has approved a supplemental new drug application to expand the indication for Aptiom (eslicarbazepine acetate) to include treatment of partial-onset seizures in children age 4 to 17. The safety and efficacy of Aptiom as monotherapy and adjunctive therapy for the treatment of partial-onset seizures in adults was established in five multicenter, randomized, controlled clinical trials. Data from three clinical trials supported the safety and tolerability of Aptiom for the treatment of partial-onset seizures in pediatric patients. Pharmacokinetic analyses of adult and pediatric data supported its use in the pediatric population. Aptiom is a once-daily, immediate release drug that can be taken whole or crushed, with or without food. Sunovion Pharmaceuticals, which markets Aptiom, is headquartered in Marlborough, Massachusetts.

—Kimberly Williams