The US Meals and Drug Administration (FDA) has accepted fenfluramine (Fintepla, Zogenix) oral resolution, a Schedule IV managed substance, for the therapy of seizures related to Dravet syndrome in kids age 2 years and older.
Dravet syndrome is a uncommon childhood-onset epilepsy characterised by frequent, drug-resistant convulsive seizures which will contribute to mental incapacity and impairments in motor management, behaviour, and cognition, in addition to an elevated danger of sudden surprising demise in epilepsy (SUDEP).
Dravet syndrome takes a “great toll on each sufferers and their households. Fintepla affords a further efficient therapy possibility for the therapy of seizures related to Dravet syndrome,” Billy Dunn, MD, director, Workplace of Neuroscience within the FDA’s Heart for Drug Analysis and Analysis, stated in a information launch.
The FDA accepted fenfluramine for Dravet syndrome primarily based on the outcomes of two randomized, double-blind, placebo-controlled part three trials involving kids aged 2 to 18 years with Dravet syndrome.
In each research, kids handled with fenfluramine skilled considerably larger reductions within the frequency of convulsive seizures than their friends who acquired placebo. These reductions occurred inside three to four weeks, and remained usually constant over the 14- to 15-week therapy intervals, the FDA stated.
“There stays an enormous unmet want for the numerous Dravet syndrome sufferers who proceed to expertise frequent extreme seizures even whereas taking a number of of the at the moment obtainable antiseizure drugs,” Joseph Sullivan, MD, who labored on the fenfluramine for Dravet syndrome research, stated in an organization information launch.
Given the “profound reductions” in convulsive seizure frequency seen within the medical trials, mixed with the “ongoing, sturdy security monitoring,” fenfluramine affords “a particularly vital therapy possibility for Dravet syndrome sufferers,” stated Sullivan, director of the Pediatric Epilepsy Heart of Excellence on the College of California San Francisco (UCSF) Benioff Youngsters’s Hospital.
Fenfluramine is an anorectic agent that was used to deal with weight problems till it was faraway from the market in 1997 over studies of elevated danger of valvular coronary heart illness when prescribed in greater doses and most frequently when prescribed with phentermine. The mixture of the 2 medicine was generally known as fen-phen.
Within the medical trials of Dravet syndrome, the commonest adversarial reactions had been decreased urge for food; somnolence, sedation, lethargy; diarrhea; constipation; irregular echocardiogram; fatigue, malaise, asthenia; ataxia, stability dysfunction, gait disturbance; elevated blood stress; drooling, salivary hypersecretion; pyrexia; higher respiratory tract an infection; vomiting; decreased weight; fall; and standing epilepticus.
The Fintepla label has a boxed warning stating that the drug is related to valvular coronary heart illness (VHD) and pulmonary arterial hypertension (PAH). As a result of these dangers, sufferers should bear echocardiography earlier than therapy, each 6 months throughout therapy, and as soon as three to six months after therapy is stopped.
If indicators of VHD, PAH, or different cardiac abnormalities are current, clinicians ought to weigh the advantages and dangers of continuous therapy with Fintepla, the FDA stated.
Fintepla is accessible solely by means of a danger analysis and mitigation technique (REMS) program, which requires physicians who prescribe the drug and pharmacies that dispense it to be licensed within the Fintepla REMS and that sufferers be enrolled in this system.
As a part of the REMS necessities, prescribers and sufferers should adhere to the required cardiac monitoring to obtain the drug.
Fintepla might be obtainable to licensed prescribers in the US in July. Zogenix is launching Zogenix Central, a complete assist service that can present ongoing product help to sufferers, caregivers, and their medical groups. Additional info is accessible on-line.
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