Publication date: Jul 06, 2025
Between baseline and the titration plus maintenance phase, fenfluramine demonstrated statistically significant changes relative to placebo on the primary end point of percent change in countable motor seizure frequency. In the latest data update, results showed a 0. 8-point increase in NSAA scores among participants from CANYON, the major phase 2 trial of sevasemten, after 18 months of treatment. Based on these data, the company plans to submit an application for fenfluramine to become a potential treatment option for patients living with CDD. Building on the mechanism of action of nusinersen (Spinraza; Biogen), salanersen is designed to enhance potency and allow for once-yearly dosing, offering potential improvements in convenience and efficacy. Based on these findings, Biogen plans to test the therapy in phase 3 studies, the design of which is being discussed with the FDA. MESA, an open-label extension, featured 99% of eligible participants from the previously completed ARCH, CANYON, GRAND CANYON, or DUNE trials. WATCH TIME: 4 minutesWelcome to this special edition of Neurology News Network.
| Concepts | Keywords |
|---|---|
| Expert | Biogen |
| Fda | End |
| Fox | Fda |
| Therapy | Fenfluramine |
| Yearly | Label |
| Mesa | |
| Muscular | |
| Open | |
| Phase | |
| Salanersen | |
| Sevasemten | |
| Therapy | |
| Treatment | |
| Trial | |
| Trials |
Semantics
| Type | Source | Name |
|---|---|---|
| drug | DRUGBANK | Fenfluramine |
| disease | MESH | spinal muscular atrophy |
| drug | DRUGBANK | Nusinersen |
| disease | MESH | Becker muscular dystrophy |
| drug | DRUGBANK | Methionine |
| disease | MESH | CDKL5 deficiency disorder |
| disease | MESH | motor seizure |
| disease | MESH | Dravet syndrome |
| disease | MESH | Lennox-Gastaut syndrome |
| drug | DRUGBANK | Coenzyme M |