Study Finds Positive Impact of Fintepla on Dravet Patients and Caregivers
April 19, 2021
Rare Daily Staff
Zogenix reported positive results from an investigator-initiated study designed to assess caregivers’ perspectives on the long-term seizure- and non-seizure-related benefits of Fintepla on patients with the rare epilepsy Dravet syndrome.
Data from the study is being presented during the virtual American Academy of Neurology (AAN) Annual Meeting, April 17 to 22, 2021.
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.
The U.S. Food and Drug Administration in June 2020 approved Fintepla for the treatment of seizures associated with Dravet syndrome in patients age 2 and older. The approval came with a black box warning that the drug’s use is associated with valvular heart disease and pulmonary arterial hypertension.
“The impact of Dravet syndrome on the lives of the diagnosed child and the family members who care and worry about their loved one can be severe and unrelenting,” said Mark Jensen, vice chair for research in Rehabilitation Medicine at UW Medicine, professor of Rehabilitation Medicine at the University of Washington, and lead author for the study. “Our study shows that treatment with Fintepla not only reduces seizure activity, but also results in substantial benefits for many other aspects of the lives of those impacted by Dravet syndrome.”
He said the majority of caregivers reported that after treatment began, they felt less overwhelmed, less anxiety, and less stress. They noted improvements in sleep in both the child and themselves, and even noticed improvements in the siblings of the child with Dravet syndrome.
For the study, caregivers of patients who received Fintepla through either the phase 3 clinical trial program or the U.S. early access program were recruited to participate in one-on-one semi-structured interviews to discuss the benefits of the drug for the child with Dravet syndrome, as well as the parents and the family as a whole.
Of the participants interviewed, 34 percent were caregivers of adult patients with Dravet syndrome. Mean age of patients with Dravet syndrome was 15 years (range, 2-33 years) and they had been on the Fintepla treatment regimen for an average of 21 months (range, 5-59 months).
The most commonly reported non-seizure-related improvements were in the areas of cognition (76 percent), alertness (68 percent), education (65 percent), problem solving (56 percent), speech (48 percent), sleep quality (47 percent), motor function (46 percent), and mood (41 percent).
Caregiver parents themselves reported feeling less overwhelmed (71 percent), less stress (66 percent), and less anxiety and depression (68 percent). More than two-thirds reported that the quality of their sleep improved (71 percent) and they had more time to do things they enjoy (56 percent). In addition, 44 percent of parents reported that the relationship with their spouse or partner improved and 62 percent of employed parents missed less work.
Reported family benefits included less family stress (76 percent), improved relationship between the Dravet syndrome child and their siblings (56 percent), improved behavior and/or mood of siblings (58 percent), and easier time for their family to do things with others (64 percent).
“This qualitative study provides insight directly from caregivers about the real world benefits they observe in their children and family as a result of their child being treated with Fintepla,” said Bradley Galer, executive vice president and chief medical officer at Zogenix and one of the study authors. “These findings further support the data reported in our phase 3 studies related to seizure and non-seizure outcomes.”
Photo: Bradley Galer, executive vice president and chief medical officer at Zogenix
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