Significant seizure reductions in patients with TSC

EPIDIOLEX®
(cannabidiol)
significantly reduced the frequency of TSC-associated seizures by half

EPIDIOLEX®
(cannabidiol)
significantly reduced the frequency of TSC-associated seizures by half

EPIDIOLEX Tuberous sclerosis complex (TSC) – Percentage Reduction in Monthly Seizure Frequency vs Placebo

*The median percent change from baseline for patients with TSC was 43% for patients receiving EPIDIOLEX 25 mg/kg/day, compared with 20% for patients receiving placebo (P<0.01).

Results from the 16-week treatment period. Primary endpoint seizures included all countable TSC-associated seizures, including partial-onset seizures and generalized seizures (tonic-clonic, tonic, clonic, or atonic).

Patients at baseline1:

  • Had previously tried a median of 4 prior AEDs
  • Were currently uncontrolled with a median of 3 current AEDs

89% of patients were taking ≥2 AEDs at baseline and still experiencing 57 TSC-associated seizures per 28 days2

Venn Diagram Icon

The most commonly used concomitant AEDs were:
45% valproate
33% vigabatrin
29% levetiracetam
27% clobazam

See baseline demographics



Results from the 16-week treatment period. Primary endpoint seizures included all countable TSC-associated seizures, including partial-onset seizures and generalized seizures (tonic-clonic, tonic, clonic, or atonic).

*The median percent change from baseline for patients with TSC was 43% for patients receiving EPIDIOLEX 25 mg/kg/day, compared with 20% for patients receiving placebo (P<0.01).

Reductions in TSC-associated seizure frequency were reported as early as Day 6 in a post-hoc analysis of the TSC clinical trial3

The recommended maintenance daily dosage for EPIDIOLEX is 25 mg/kg/day for patients with TSC.

Patients with moderate to severe hepatic impairment require a dose adjustment.

See recommended
dosing information
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EPIDIOLEX reduced TSC-associated partial-onset seizure score by 46% in a prespecified exploratory analysis2

EPIDIOLEX Tuberous sclerosis complex (TSC) – Percentage Reduction in Frequency vs Placebo

Composite score is a weighted average of the 3 types of partial-onset seizures included in the primary endpoint1,2,4:

  • Simple partial seizures
  • Complex partial seizures
  • Secondary generalized tonic-clonic seizures

Results from the 16-week treatment period. Results of the composite seizure score were not controlled for multiplicity; therefore, P-values are not reported.1,2

More patients with TSC achieved ≥50% and ≥75% reductions in TSC-associated seizures with EPIDIOLEX than with placebo

EPIDIOLEX Tuberous sclerosis complex (TSC) – Percentage responders achieved reductions in seizures vs Placebo

Results from the 16-week treatment period.

More patients achieved seizure freedom with EPIDIOLEX than with placebo

Freedom from TSC-associated seizures in the 12-week maintenance period

0%

Placebo

6%

EPIDIOLEX
25 mg/kg/day

PRESPECIFIED EXPLORATORY ENDPOINT: CHANGE IN MEAN NUMBER OF TSC-ASSOCIATED SEIZURE-FREE DAYS/28 DAYS2†

Baseline
(per 28 days)
Maintenance period
(per 28 days)
EPIDIOLEX 25 mg/kg/day, n=71 7.4 days 14.7 days 97% increase
Placebo, n=76 7 days 10.9 days 55% increase
Placebo,
n=76
EPIDIOLEX
25 mg/kg/day,
n=71
Baseline
(per 28 days)
7
days
7.4
days
Maintenance
period
(per 28 days)
10.9
days

55%
increase
14.7
days

97%
increase

Analysis was not controlled for multiplicity and is considered exploratory in nature.

EPIDIOLEX maintained reductions in TSC-associated seizures in an ongoing open-label extension trial5

EPIDIOLEX Tuberous sclerosis complex (TSC) – Percentage Responders Who Maintained Reductions in Seizures in an Ongoing Open-Label Extension Trial

Decreasing N-values reflect rolling entry into the open-label extension

  • The retention rate at 48 weeks was 79%2
  • 28% (n=11) of withdrawals were due to adverse reactions5
  • LOCF sensitivity analyses showed no impact of withdrawn patients on change in seizure frequency5
99% (n=199) of patients with TSC who completed the controlled trial elected to continue into the open-label extension5

Adverse events5

  • The long-term safety profile of EPIDIOLEX in this open-label extension trial was generally similar to that observed in the 16-week trial for TSC
  • There was 1 death reported during the study, which was deemed unrelated to treatment by the investigator
  • In the open-label extension trial, titration to doses over 25 mg/kg/day was permitted. At higher doses, an increase in adverse reactions is possible
See recommended dosing information
LGS data Dravet syndrome data

EPIDIOLEX is contraindicated in patients with a history of hypersensitivity to cannabidiol or any ingredients in the product

SEE WARNINGS AND PRECAUTIONS