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FDA Approval of FINTEPLA for Treatment of Seizures Associated with Dravet Syndrome in Persons 2 Years of Age or Older

Zogenix Press Release About Fintepla FDA Approval

Dr. Jacqueline French Discusses the FDA Approval of Fintepla

 

Studies

Quotes

  • “We know that therapies work differently for each patient, but fenfluramine has been absolutely life-changing for our family,” said Abby H. “Our 10-year-old daughter, Norah, who suffers from Dravet syndrome, enrolled in the Zogenix trial in mid-2017. Her seizures just stopped cold. Three years later, she continues to enjoy near seizure freedom, along with improvements in mood, behavior, energy level, and overall well-being. Our family is now able to do so many things that simply were not possible before (including a trip to Disney World in February 2019) and we are so grateful for the opportunity to watch Norah and her brothers enjoying all that life has to offer.” 
     
  • “As the Principal Investigator for the Fintepla study, I am extremely excited about the robust efficacy that patients experienced, with 2/3 achieving a greater than 50% reduction in seizure frequency and 1/3 achieving a 75% reduction in seizure frequency,” said Dr. Joseph Sullivan. “These reductions in seizure frequency are rarely seen in epilepsy drug trials. In my opinion, this sets a new bar for what we can achieve in a highly refractory epilepsy syndrome such as Dravet. Furthermore, I am encouraged by the tolerability and safety profile and am confident the proposed monitoring program that will be part of its clinical use will allow this to be an extremely important treatment option for these patients and their families.” 

The History of Development of Fintepla

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What is FAiRE?

The FAiRE program of clinical trials is exploring whether an investigational drug (ZX008) can improve seizure control in children and young adults with Dravet syndrome. FAiRE stands for “Fenfluramine Assessment in Rare Epilepsy.”

The goal of the ZX008-Study 2/1504 trials is to show that ZX008 is a safe and effective treatment for children and adolescents with Dravet syndrome when added on to their other seizure medications. The trials will look at a number of outcomes including whether people taking the study drug have:

  • A decrease in the number of convulsive (also called tonic-clonic) or non-convulsive (for example absence or focal) seizures compared to before the study medication was started
  • More days without convulsive seizures
  • Less frequent use of rescue medication
  • Less frequent hospitalizations specifically to treat seizures

Eligibility criteria include1,2

  • Age 2-18 years (inclusive)
  • Documented medical history that supports a clinical diagnosis of Dravet syndrome
  • First seizure happened in the person’s first year of life
  • Uncontrolled seizures despite current medication

How can I participate?

  • After an eligibility screening, potential participants or their caregivers record baseline seizure activity in a seizure diary for six weeks.
  • After this baseline period:
    • Potential participants in study 1501 who still meet all eligibility criteria are randomly put into one of three groups to recieve study medication for 14 weeks. In 2 of the groups, diferent doses of the study medicine will be given. People in the 3rd group will receive a placebo (a pill that does not have any expected benefit).
    • Potential participants in study 1504 who still meet all eligibility criteria are randomly put into one of two groups to recieve study medication for 14 weeks. One group will receive the study medicine; while the second group will receive a placebo (a pill that does not have any expected benefit).
    • After completing the 14-week trial period in either study, all participants will be eligible to receive the active study medication (no one receives placebo) for up to one year in an optional extension study.
  • Although some participants will recieve placebo during the 14-week trial period, ALL participants will continue taking their normal background anti-seizure medication(s) through the entire study.
  • People will be asked to use a diary to record seizure activity and medication details for 16 weeks.
  • Throughout the study, everyone will have their health checked regularly, either at their local study clinic or over the phone. These checks will vary from visit to visit, but may include electrocardiograms (ECG) (test of heart rhythms), echocardiograms (an ultrasound of the heart), physical examination (including blood pressure and heart rate), and nervous system assessments.

Are there risks?

  • Fenfluramine has been used in the past to lower appetite in obese adults, but was discontinued when some people had heart-related side effects. The participants in this trial will receive a much lower dose than what was related to the heart-related side effects. However, during this trial, regularly scheduled heart-health check ups are done to help identify any potential issues early.
  • With all trial drugs and devices, there is the chance the treatment may not help or that participants' condition could worsen.
  • The dosing plans and safety checks that are designed to protect participants are detailed in a protocol. The protocol was designed by researchers and doctors, and was reviewed by an Institutional Review Board (IRB) and the U.S. Food and Drug Administration (FDA). This protocol also includes regular safety reviews of each subject by a group of independent experts to see how the study medication is affecting each individual.

For more information on Dravet syndrome and FAiRE, or to enroll, visit https://www.faireds.com/en-us/recruitment/can-i-take-part.

1Study 2 does not allow patients taking stiripentol to participate.
2Study 1504 requires patients to be taking stiripentol as part of their current treatment regimen.