According to a news release in Yahoo! Finance, biopharmaceutical company Zogenix, Inc. recently submitted a Supplemental New Drug Application (sNDA) for its drug candidate FINTEPLA (fenfluramine). The therapy is designed to treat patients with Lennox-Gastaut syndrome (LGS). A New Drug Application (NDA) asks the FDA to approve a drug for nationwide sales and marketing; the sNDA builds upon that application, or an already approved application, by submitting additional data.
FINTEPLA
Currently, FINTEPLA is approved in both the United States and the European Union for patients ages 2+ with Dravet syndrome, another form of rare pediatric epilepsy. According to the European Medicines Agency (EMA):
The active substance in Fintepla, fenfluramine, causes the release of serotonin, a substance used by nerve cells to communicate with neighboring cells, in the brain. Fenfluramine may also protect against seizures by acting on the sigma-1 receptor found on the surface of nerve cells.
This particular sNDA seeks to expand the use of FINTEPLA for patients with Lennox-Gastaut syndrome. As some patients’ conditions are not well-managed using the current standards-of-care, FINTEPLA offers the chance to significantly improve patient outcomes. To support the sNDA, Zogenix submitted data from the Phase 3 Study 1601 clinical trial. Altogether, 263 patients, ranging in age from 2 to 35, enrolled. Findings from the study include:
- FINTEPLA is relatively safe and well-tolerated. While some adverse reactions did occur, these mirror those seen previously in Dravet syndrome studies.
- 0.7mg/kg FINTEPLA daily helped reduce the frequency of drop seizures.
Lennox-Gastaut Syndrome (LGS)
Lennox-Gastaut syndrome (LGS) is a rare, severe, and often difficult-to-treat epilepsy which begins in childhood. An estimated 5% of childhood epilepsy diagnoses are attributed to LGS. The condition is characterized by frequent and varied seizures, including atonic seizures, tonic seizures, and absence seizures. Typically, these seizures begin between ages 2-6. LGS may be idiopathic (without a known cause). However, lack of oxygen at birth, low birth weight or premature birth, severe brain infections, or brain development issues have all been linked to LGS. Outside of the seizures, patients may experience frequent falls or injuries, muscle stiffening or weakness, behavioral problems and psychosis, and cognitive and developmental delays.
