Dravet Syndrome: Therapy Targeting Underlying Cause Gets Orphan Drug Status
The Food and Drug Administration has granted Orphan Drug designation to STK-001 (Stoke Therapeutics) for the treatment of Dravet syndrome.
The Food and Drug Administration has granted Orphan Drug designation to STK-001 (Stoke Therapeutics) for the treatment of Dravet syndrome.
Zogenix announced the submission of a New Drug Application to the FDA for Fintepla oral solution for the treatment of seizures associated with Dravet syndrome.
Diacomit was evaluated in 2 multicenter, placebo-controlled, double-blind, randomized studies.
Because of the small size of the study sample, the findings may be limited to this patient population and may be hard to generalize across all pediatric patients with Dravet syndrome.
The FDA has accepted for filing with Priority Review the NDA for cannabidiol as seizure treatment.
ZX008 has received Orphan Drug designation and Fast Track designation by the FDA for the treatment of Dravet syndrome.
Adverse reactions in the CBD group included decreased appetite, diarrhea, fatigue, lethargy, nasopharyngitis, pneumonia, pyrexia, rash, and somnolence.
In people with Dravet syndrome, language impairments increase with age, suggesting that early intervention with targeted oral motor therapies may be helpful.
Justin Gover, CEO of GW Pharmaceuticals, the parent company of Greenwich Biosciences, discusses the status of the company’s cannabidiol product Epidiolex on the heels of positive results from several placebo-controlled trials in epileptic syndromes.
Results of the phase 3, placebo-controlled trial support previous findings from an expanded-access program.