The U.S. Food and Drug Administration (FDA) has approved fenfluramine (Fintepla) oral solution for the treatment of seizures associated with a rare childhood-onset epileptic disease called Dravet syndrome in patients above 2 years.
Headquartered in Emeryville, California, Zogenix, a global pharmaceutical company, received the FDA approval of fenfluramine oral solution, which will be available by the end of July.
President and CEO of Zogenix Stephen Farr said, “The approval of Fintepla by the FDA is a significant milestone we are proud to celebrate with the patients and families living with Dravet syndrome.”
“We began this global development program nearly six years ago after researchers in Belgium recognized the potential of fenfluramine, a drug with distinct pharmacology from all other anticonvulsant agents, to treat intractable seizures in Dravet syndrome,” he added. “Our heartfelt gratitude goes to the patients, families, and everyone who supported the rigorous development program that led to FINTEPLA’s approval.”
Dravet syndrome is a rare childhood-onset epileptic condition associated with frequent and severe treatment-resistant seizures, requiring hospitalizations. It is a medical emergency that can lead to significant developmental and motor impairments. In some cases, the condition could lead to sudden unexpected death.
Dr. Joseph Sullivan, Director of the Pediatric Epilepsy Center of Excellence at the UCSF Benioff Children’s Hospitals, said, “There remains a huge unmet need for the many Dravet syndrome patients who continue to experience frequent severe seizures even while taking one or more of the currently available anti-seizure medications.”
“Given the profound reductions in convulsive seizure frequency seen in the Fintepla clinical trials, combined with the ongoing, robust safety monitoring that will be part of its use, I feel Fintepla will offer an extremely important treatment option for Dravet syndrome patients,” added Dr. Sullivan, who is also the Principal Investigator for Fintepla in Dravet syndrome.
The FDA approved Fintepla for seizures associated with Dravet syndrome based on the findings of Phase III clinical trials, which were published in The Lancet and JAMA Neurology.
The findings suggested that the drug significantly reduced the monthly frequency of seizures in patients whose seizures were not adequately controlled with one or more antiepileptic drugs. Also, most participants responded well to the drug within three to four weeks.
The most common side effects of Fintepla were loss of appetite, sleepiness, sedation, lethargy, diarrhea, constipation, abnormal ECG, fatigue, malaise, asthenia, gait disturbances, elevated blood pressure, increased salivation, and vomiting.
Executive Director of the Dravet Syndrome Foundation Mary Anne Meskis said, “Having a new FDA-approved treatment option is so important because it improves our ability to optimize each patient’s treatment.”
“Moreover, because families living with Dravet syndrome never know when the next seizure is going to occur, whether they will end up in the ER, or what the consequences might be following the seizure, having a strong support program like Zogenix Central to reduce the strain on families is very welcome,” she added. “This will allow family members to remain focused on providing the best care of their loved one with Dravet.”