Clinical Data The data presented highlighted the updated results from the new patient cohort, which now includes 9 Dravet syndrome patients. All of these patients began add-on treatment with low-dose fenfluramine (5 mg to 20 mg per day) at various starting points between 2010 and the end of January 2016. Median treatment duration was 1.5 years (range 0.3 to 5.1 years). During the 90-day run-in period prior to initiating low-dose fenfluramine treatment, the median frequency of major motor seizures (defined as tonic, clonic, tonic-clonic, atonic, and myoclonic seizures lasting >30 seconds) was 15.0 per month (range 0.4 to 39.7). Over the entire observation period, the median frequency of major motor seizures was reduced to 1.5 per month, and the median decrease was 75% (range 28-100%). Six of the 9 patients had at least a 70% reduction in major motor seizures.
In addition, parents/caregivers were asked to rate both their child’s and their own sleep quality and quality of life using 0-10 scales where 0 = extremely bad and 10 = very good. At the most recent visit, mean sleep quality reported for patients and parents was 8.1/10 and 7.9/10, respectively, while mean Quality of Life scores were 7.4/10 for both groups.In this new cohort of patients, treatment with low-dose fenfluramine continued to be generally well-tolerated, and did not result in any echocardiographic or clinical signs of cardiac valve abnormalities, pulmonary hypertension or any other cardiovascular abnormalities. The most common treatment-emergent adverse events were mild-to-moderate somnolence (n=6), diminished appetite (n=4), mood changes (n=2), and non-convulsive status epilepticus (n=2). There were no fenfluramine discontinuations due to adverse events or lack of effect.