Zogenix Wins Approval for Dravet Syndrome Treatment Fintepla

Shares of Zogenix were up more than 16% in premarket trading following the regulatory approval of the epilepsy drug Fintepla for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy, in patients ages two and above.

The U.S. Food and Drug Administration approved an oral solution, CIV of Fintepla (fenfluramine) for this indication. Emeryville, Calif.-based Zogenix said the medication will be launched through a restricted distribution program and will be available through the company’s specialty pharmacy partner by the end of July. 

Stephen J. Farr, president and chief executive officer of Zogenix, called the approval of Fintepla a significant milestone for the company, particularly after the FDA rejected the company’s New Drug Application last year.

In April 2019 the FDA issued a Refusal to File letter for the drug. The regulatory agency said the NDA was not “sufficiently complete to permit a substantive review.” The rejection raised two concerns. The first regarded non-clinical studies that were not submitted to allow assessment of the chronic administration of fenfluramine, the active ingredient in Fintepla. The second reason cited was that the application contained an “incorrect version of a clinical dataset, which prevented the completion of the review process that is necessary to support the filing of the NDA.”

All that changed through with the new regulatory win for the epilepsy drug, which some analysts have predicted could hit blockbuster status.

“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. Our heartfelt gratitude goes to the patients, families, and everyone who supported the rigorous development program that led to Fintepla’s approval.” 

Dravet syndrome, also called Severe Myoclonic Epilepsy of Infancy, is typically caused by loss-of-function mutations in the SCN1A gene. The genetic disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays and an increased risk of early mortality due to sudden unexpected death in epilepsy. In 2018, drugmaker GW Pharmaceuticals won approval in 2018 for Epidiolex, a cannabis-based epilepsy drug approved for treatment of Dravet and Lennox-Gastaut syndrome.

Approval of Fintepla was based on data from two randomized, double-blinded, placebo-controlled Phase III clinical trials that showed the medication significantly reduced the monthly convulsive seizure frequency compared to placebo in study patients whose seizures were not adequately controlled on one or more antiepileptic drugs. In addition, most study patients responded to treatment with Fintepla within three to four weeks and effects remained consistent over the treatment period, Zogenix said.

Joseph Sullivan, director of the Pediatric Epilepsy Center of Excellence at the UCSF Benioff Children’s Hospitals and the Principal Investigator for Fintepla in Dravet syndrome, said there remains an unmet need for 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,” Sullivan said in a statement.