RARE Daily

FDA Approves New Therapy for Dravet Syndrome

June 26, 2020

Rare Daily Staff

The U.S. Food and Drug Administration has approved Zogenix’ Fintepla for the treatment of seizures associated with Dravet syndrome in patients age two and older.

Dravet syndrome is a rare genetic form of epilepsy caused by a mutation in the SCN1A gene. The severity of cognitive impairment is related to the frequency and duration of seizures. It occurs in about 1 in 300,000 people and currently there is no cure.

“Dravet syndrome is a debilitating disease that takes a tremendous toll on both patients and their families,” said Billy Dunn, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “Fintepla offers an additional effective treatment option for the treatment of seizures associated with Dravet syndrome. The FDA will continue to work with companies on drug development for Dravet syndrome and other types of epilepsy.”

Approval of Fintepla for the treatment of seizures associated with Dravet syndrome was demonstrated in two late-stage clinical studies in 202 subjects between ages two and 18. The studies measured the change from baseline in the frequency of convulsive seizures. In both studies, subjects treated with Fintepla had significantly greater reductions in the frequency of convulsive seizures during the trials than subjects who received placebo (inactive treatment). These reductions were seen within three to four weeks, and remained generally consistent over the 14 to 15 week treatment periods.

The approval comes after the company worked with the FDA to overcome issues that led to the agency’s refusal to accept it new drug application more than one year ago. Fintepla will compete with two drugs already approved to treat seizures associated with Dravet syndrome: GW’s Epidiolex, which is marketed by Greenwich Biosciences in the United States, and Diacomit, produced by Biocodex.

Zogenix reported that Fintepla reduced seizures by 62.7 percent in the first trial, and by 72.4 percent in the second trial, compared to the 40 percent reduction seen by GW Pharma for Epidiolex in the late-stage study that led to its approval in 2018. Zogenix plans to charge on average $96,000 for Fintepla, about three times the cost of Eidiolex.

Fintepla, however, carries a boxed warning stating the drug is associated with valvular heart disease (VHD) and pulmonary arterial hypertension (PAH). Because of these risks, patients must have cardiac monitoring using echocardiograms performed before treatment, every six months during treatment, and once three to six months after treatment is discontinued. Because of the risks of VHD and PAH, Fintepla is available only through a restricted drug distribution program, under a risk evaluation and mitigation strategy.

“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,” said Stephen Farr, president and CEO of Zogenix.

The FDA granted this application Priority Review. Fintepla received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.




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