Table 1.
A comparison of side effects experienced by participants from controlled case studies (Nabbout et al., 2020; Lagae et al., 2019) and an open label extension (Sullivan et al., 2020) of Fintepla. The most common adverse events observed in the three studies included decreased appetite, pyrexia, diarrhea, nasopharyngitis, and fatigue. Inputs labeled with (∗) represent very common effects (>1/10). Inputs labeled with (∗∗) represent common effects (≥1/100 to <1/10) in accordance with the FDA and EMA guidelines. Of note, the studies did not have adverse effects that met the criteria of rare (≥1/10,000 to <1/1,000) or very rare (<1/10,000) adverse effects. The column with a (†) contains no data points because the open-label study was a placebo-uncontrolled study and all participants received fenfluramine. Additionally, the data from the three studies could not be pooled due to the recruitment of patients from Lagae et al., and the Nabbout et al. study.
| Side Effects | % Adverse Events [rank] in those Receiving Fenfluramine (Lagae et al., 2019) (N = 79) | % Adverse Events [rank] in those Receiving Placebo (Lagae et al., 2019) (N = 40) | % Adverse Events [rank] in those Receiving Fenfluramine (Nabbout et al., 2020) (N = 43) | % Adverse Events [rank] in those Receiving Placebo (Nabbout et al., 2020) (N = 44) | % Adverse Events [rank] in those Receiving Fenfluramine (Sullivan et al., 2020) (N = 232)) | % Adverse Events [rank] in those Receiving Placebo (Sullivan et al., 2020) (N = 0)† |
|---|---|---|---|---|---|---|
| Patients with at least 1 adverse Event | 94.9 | 65.0 | 97.7 | 95.4 | 89.7 | N/A |
| Decreased Appetite | 29.1∗ | 5.0∗∗ | 44.2∗ | 11.4∗ | 15.9∗ | N/A |
| Diarrhea | 24.1∗ | 7.5∗∗ | 23.3∗ | 6.8∗∗ | 10.8∗ | N/A |
| Fall | 5.1∗∗ | 5.0∗∗ | 0.0 | 0.0 | 0.0 | N/A |
| Fatigue | 10.1∗ | 2.5∗∗ | 25.6∗ | 4.5∗∗ | 0.0 | N/A |
| Lethargy | 13.9∗ | 5.0∗∗ | 14.0∗ | 4.5∗∗ | 0.0 | N/A |
| Nasopharyngitis | 13.9∗ | 5.0∗∗ | 16.3∗ | 34.1∗ | 19.4∗ | N/A |
| Pyrexia | 11.4∗ | 7.5∗∗ | 25.6∗ | 9.1∗∗ | 21.6∗ | N/A |
| Seizure | 8.9∗∗ | 12.5∗ | 4.7∗∗ | 15.9∗ | 11.2∗ | N/A |
| Somnolence | 12.7∗ | 7.5∗∗ | 0.0 | 0.0 | 0.0 | N/A |
| Upper Respiratory Tract Infection | 10.1∗ | 12.5∗ | 0.0 | 0.0 | 10.3∗ | N/A |
| Vomiting | 8.9∗∗ | 10.0∗ | 0.0 | 0.0 | 0.0 | N/A |
| Weight Decrease | 8.9∗∗ | 0.0 | 0.0 | 0.0 | 0.0 | N/A |
| Blood Glucose Decrease | 0.0 | 0.0 | 14.0∗ | 4.5∗∗ | 0.0 | N/A |
| Bronchitis | 0.0 | 0.0 | 11.6∗ | 4.5∗∗ | 0.0 | N/A |
| Abnormal Heart Valves | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | N/A |