Epilepsy digest
American Academy of Neurology - 2021

Epilepsy digest
American Academy of Neurology - 2021

GW Research presentations @ AAN: Epidiolex

Epidiolex Cannabidiol (GWP42003-P, CBD) – GW Research Ltd

Interim and post-hoc analysis of Phase 3 study of add-on Epidiolex in tuberous sclerosis complex (TSC)-associated seizures presented @ AAN 2021

  • Objective: To evaluate the safety and efficacy of add-on CBD for treatment of TSC-associated seizures in the interim analysis of an OLE trial and the post hoc analysis to compare the response to add-on CBD in patients with TSC and treatment-resistant epilepsy with and without IS history
  • Trial details: Phase 3 in TSC-associated seizures; N= 224; 25 mg/kg/day GWP42003-P, 100 mg/ml GWP42003-P oral solution taken twice daily; Primary outcomes: Percent change from baseline in the number of TSC-associated seizures ; PCD: 22 Jan’ 19; Completed; Location: Global
  • Results:
    • Interim analysis, N= 199; Median (range) age: 10.7 (1.1–56.8) years
    • N=138 with IS history; Median (range) age: 12.2 years (1.1–56.8) and 10.5 years (1.6–55.8) for patients with and without IS history

Parameters

Value

Source

Median percentage reductions in TSC-associated seizures

53% -75%

Link

Seizure reductions

54%–80%

Improvement on Subject/Caregiver Global Impression of Change

85% and 89%

Percentage reduction in seizures: for patients with infantile spasms (IS) history / without IS history:

Link

CBD 25 mg/kg/day

45% / 54%

CBD 50 mg/kg/day

43% / 55%

Placebo

23% / 32%

  • Conclusion: Add-on CBD treatment was well tolerated and produced sustained reductions in TSC-associated seizures. CBD produced consistent reductions in TSC-associated seizures in patients with and without IS history

Add-on CBD treatment produced sustained reductions in TSC-associated seizures and produced consistent reductions in TSC-associated seizures in patients with and without IS history

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CI Scientists Remarks:

  • About Epidiolex (Cannabidiol): It acts on multiple systems and interacts with many neurotransmitters and neuromodulator systems implicated in a range of diseases
    • CBD may exert a cumulative anti-convulsant effect, modulating a number of endogenous systems including, but not limited to neuronal inhibition (synaptic and extrasynaptic GABA channels), modulation of intracellular calcium (TRPV, VDAC, GPR55), and possible anti-inflammatory effects (adenosine).
  • Regulatory status:
    • Epidiolex is approved in the US to treat seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome and TSC
  • Regulatory approvals across the regions:

Molecule

Region

Year of Approval

Indication

Cannabidiol Epidiolex

US

Jun 2018

Seizures associated with Lennox-Gastaut syndrome and Dravet syndrome

EU

Sep 2019

Seizures associated with Lennox-Gastaut syndrome and Dravet syndrome

US

Aug 2020

Seizures associated with tuberous sclerosis complex (TSC)

EU

Apr 2021

Seizures associated with TSC

  • Future plans:
    • In Feb 2021, Jazz Pharmaceuticals has entered into a definitive agreement to acquire GW Pharmaceuticals
    • GW Pharma is looking at TSC label expansion and continued penetration in Dravet and LGS
    • Plans to launch in Germany and UK and determine pricing and approval for reimbursement in France, Spain, Italy, and other European countries
    • Obtain approval of the Tuberous Sclerosis Complex indication in Europe
    • Commence pivotal trial in fourth target orphan epilepsy indication

– Monalisa Baral, CI Scientists

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