FDA Greenlights UCB Drug for Severe Form of Epilepsy
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UCB's oral drug for seizures related to Lennox-Gastaut syndrome is now approved in the United States for patients ages two years and older.
The U.S. Food and Drug Administration gave its approval and granted pediatric exclusivity for Fintepla (fenfluramine) C-IV as an oral solution for LGS, a severe type of childhood-onset developmental and epileptic encephalopathy (DEE) with high morbidity rates. The illness is characterized by drug-refractory seizures that lead to the serious decline of neurodevelopmental, cognitive and motor functions. Disease progression can further result in problems with communication, sleep, behavior and mobility, and even cause manifest psychiatric symptoms. People with LGS also live in fear of sudden unexpected death in epilepsy (SUDEP).
"The approval of fenfluramine for Lennox-Gastaut syndrome highlights our continued commitment to bringing differentiated medicines to patients who may not be well controlled on current therapies, and their caregivers. We are proud to add fenfluramine as a treatment for Dravet syndrome, and now Lennox-Gastaut syndrome, to our portfolio of epilepsy medicines to help reduce the impact and burden of seizures, including severe epilepsy syndromes that have high pediatric morbidity and mortality rates," noted Mike Davis, head of the global epilepsy arm at UCB, in a statement.
Fenfluramine for GLS is already approved in the U.S. for seizures linked with Dravet syndrome for the same age demographic. The drug has demonstrated a strong ability to treat the most challenging types of seizures, including drop seizures, which lead to sudden loss of muscle tone. Fenfluramine works differently from typical anti-seizure drugs and can be used without any disruptions to current medications.
Some of the common adverse reactions observed include somnolence, decreased appetite, diarrhea, fatigue, sedation, ataxia, balance and gait issues, increased blood pressure, upper respiratory tract infection and status epilepticus.
"LGS is one of the most challenging epileptic encephalopathies to treat, and the vast majority of patients are not well controlled, despite a regimen of multiple antiepileptic drugs. As a complementary therapy, fenfluramine offers a different mechanism of action and demonstrated ability to significantly reduce the number of seizures associated with a drop, a critical measure for managing this severe form of epilepsy," commented Kelly Knupp, M.D., MSCS, FAES, the associate professor at the Children's Hospital Colorado, in a statement.
The FDA's decision was based on positive outcomes from UCB's Phase III study on 263 patients who failed to achieve desired results from previous and current treatments. In the trial, which covered participants ages two to 35 years old, fenfluramine demonstrated significant efficacy and safety figures. It also supported long-term cardiovascular safety data in LGS and DS patients taking the drug for up to three years.
Patients needing access to the drug can do so through a comprehensive support program called Zogenix Central, which was created to provide support and product assistance to patients, their caregivers and medical teams. More details about this prescription drug are available at the official Fintepla website.
Fintepla is available in the U.S. through a restricted distribution program called the Fintepla REMS program (Risk Evaluation and Mitigation Strategy), and prescribers must be certified by first enrolling. It is available in the European Union are a controlled access program as well. UCB acquired Zogenix and fenfluramine on March 7, 2022.