Zorevunersen in Children and Adolescents with Dravet Syndrome - PubMed
3 hours ago
- #Dravet Syndrome
- #Epilepsy Treatment
- #Antisense Oligonucleotide
- Zorevunersen is an antisense oligonucleotide designed to up-regulate NaV1.1 sodium channels for treating Dravet syndrome.
- Dravet syndrome is a severe developmental and epileptic encephalopathy primarily caused by SCN1A haploinsufficiency.
- The study involved patients aged 2 to 18 years in phase 1-2a open-label studies (MONARCH and ADMIRAL) and extension studies (SWALLOWTAIL and LONGWING).
- Patients received single or multiple ascending doses of zorevunersen (10 to 70 mg), followed by maintenance doses in extension studies.
- Most adverse events were mild or moderate, with post-lumbar puncture syndrome and elevated cerebrospinal fluid protein being common.
- Serious adverse events included two deaths from sudden unexpected death in epilepsy and one from malnutrition.
- Patients receiving 70 mg doses showed a median reduction in convulsive-seizure frequency ranging from -58.82% to -90.91%.
- Improvements in clinical status, quality of life, and adaptive behavior were observed over up to 36 months of treatment.
- The safety profile and clinical improvements support further development of zorevunersen as a potential disease-modifying treatment.