TABLE 1.

Summary of Pivotal Phase 3 Trials in Patients with LGS or DS

	GWEP1414 (LGS)	GWEP1423 (LGS)	GWEP1332B (DS)	GWEP1424 (DS)
Description	Adjunct to existing ASDs in patients with LGS who had inadequately controlled drop seizures		Adjunct to existing ASDs in patients with DS who had inadequately controlled convulsive seizures
Patient population	2‐55 years with a clinical diagnosis of LGS, ≥ 2 drop seizures each week during the 28‐day baseline period despite taking ≥ 1 ASD at a stable dose for ≥ 4 weeks		2‐18 years with a clinical diagnosis of DS, ≥ 4 convulsive seizures during the 28‐day baseline period despite taking ≥ 1 ASD at a stable dose for ≥ 4 weeks
Regions	US, UK, France, Spain	US, The Netherlands, Poland	US, UK, France, Poland	US, Spain, Poland, Australia, Israel, The Netherlands
Patients planned/randomized	150/225	100/171	100/120	186/199
Treatment group: number of patients treated	Placebo: 76 CBD 10 mg/kg/day: 73 CBD 20 mg/kg/day: 76	Placebo: 85 CBD 20 mg/kg/day: 86	Placebo: 59 CBD 20 mg/kg/day: 61	Placebo: 65 CBD 10 mg/kg/day: 66 CBD 20 mg/kg/day: 67
Treatment plan	Baseline Period (Days −28 to −1)	Baseline Period (Days −28 to −1)	Baseline Period (Days −28 to −1)	Baseline Period (Days −28 to −1)
	Double‐blind treatment period (Weeks 1‐14): Titration Period (Weeks 1‐2): CBD 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 7 days to 10 mg/kg/day, or 11 days to 20 mg/kg/day Placebo volumes equivalent to 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 7 or 11 days	Double‐blind treatment period (Weeks 1‐14): Titration Period (Weeks 1‐2): CBD 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 11 days Placebo volumes equivalent to 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 11 days	Double‐blind treatment period (Weeks 1‐14): Titration Period (Weeks 1‐2): CBD 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 11 days Placebo volumes equivalent to 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 11 days	Double‐blind treatment period (Weeks 1‐14): Titration Period (Weeks 1‐2): CBD 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 7 days to 10 mg/kg/day, or 11 days to 20 mg/kg/day Placebo volumes equivalent to 2.5 mg/kg/day increasing 2.5‐5.0 mg/kg QOD over 7 or 11 days
	Maintenance Period (Weeks 3‐14): CBD 10 or 20 mg/kg/day Placebo volumes equivalent to 10 or 20 mg/kg/day	Maintenance Period (Weeks 3‐14): CBD 20 mg/kg/day Placebo volumes equivalent to 20 mg/kg/day	Maintenance Period (Weeks 3‐14): CBD 20 mg/kg/day Placebo volumes equivalent to 20 mg/kg/day	Maintenance Period (Weeks 3‐14): CBD 10 or 20 mg/kg/day Placebo volumes equivalent to 10 or 20 mg/kg/day
	Optional OLE trial or taper (10% per day) and follow‐up		Optional OLE trial or taper (10% per day) and follow‐up
Efficacy endpoints	Primary: Percentage change from baseline in drop seizure frequency during the treatment period		Primary: Percentage change from baseline in convulsive seizure frequency during the treatment period
	Key secondary: Proportion of patients with a ≥ 50% reduction from baseline in drop seizure frequency during the treatment period		Key secondary: Proportion of patients with a ≥ 50% reduction from baseline in convulsive seizure frequency during the treatment period
Efficacy: reduction in seizure frequency	Placebo: 17.2% CBD 10 mg/kg/day: 37.2%, P = .0016 CBD 20 mg/kg/day: 41.9%, P = .0047	Placebo: 21.8% CBD 20 mg/kg/day: 43.9%, P = .0135	Placebo: 13.3% CBD 20 mg/kg/day: 38.9%, P = .0123	Placebo: 26.9% CBD 10 mg/kg/day: 48.7%, P = .0095 CBD 20 mg/kg/day: 45.7%, P = .0299
Efficacy: 50% responder rates	Placebo: 14.5% CBD 10 mg/kg/day: 35.6%, P = .0030 CBD 20 mg/kg/day: 39.5%, P = .0006	Placebo: 23.5% CBD 20 mg/kg/day: 44.2%, P = .0043	Placebo: 27.1% CBD 20 mg/kg/day: 42.6%, P = .0784	Placebo: 26.2% CBD 10 mg/kg/day: 43.9%, P = .0332 CBD 20 mg/kg/day: 49.3%, P = .0069

The seizures are drop seizures in LGS (atonic, tonic, or tonic‐clonic) and convulsive seizures in DS (tonic‐clonic, tonic, clonic, or atonic).

Abbreviations: ASD, antiseizure drug; CBD, cannabidiol; DS, Dravet syndrome; LGS, Lennox‐Gastaut syndrome; OLE, open‐label extension; QOD, every other day.