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Epidyolex
Sponsor
Device/Product name
Epidyolex
Active Ingredient
Cannabidiol
Date of decision
Published
Submission type
New chemical entity
ATC codes
N03AX24
Decision
Approved
What was the decision based on
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Epidyolex was considered favourable for the therapeutic use approved.
What steps were involved in the decision process
Registration timeline
The following table summarises the key steps and dates for this application.
| Description | Date |
|---|---|
| Designation: Orphan | 22 November 2019 |
| Designation: Priority | 18 December 2019 |
| Submission dossier accepted and first round evaluation commenced | 28 February 2020 |
| Evaluation completed | 6 July 2020 |
| Delegate's overall benefit-risk assessment and request for Advisory Committee advice | 7 July 2020 |
| Sponsor's pre-Advisory Committee response | 21 July 2020 |
| Advisory Committee meeting | 6 and 7 August 2020 |
| Registration decision (Outcome) | 18 September 2020 |
| Completion of administrative activities and registration on ARTG | 21 September 2020 |
| Number of working days from submission dossier acceptance to registration decision* | 140 |
*Statutory timeframe for standard applications is 255 working days
Date of entry onto ARTG
Original publication date
Black triangle scheme
Yes. This product will remain in the scheme for 5 years, starting on the date the product is first supplied in Australia
Dose forms
Oral liquid, solution
Strength
100 mg/mL
Other ingredients
Sesame oil, ethanol absolute, sucralose, strawberry flavour 501094A (ARTG Proprietary Ingredient No. 139687)
Containers
Bottle
Pack sizes
100 mL
Routes of administration
Oral
Dosage
Epidyolex should be initiated and supervised by a neurologist.
The recommended starting dose of Epidyolex is 2.5 mg/kg taken twice daily (5 mg/kg/day) for one week. After one week, the dose should be increased to a maintenance dose of 5 mg/kg twice daily (10 mg/kg/day). Based on individual clinical response and tolerability, each dose can be further increased in weekly increments of 2.5 mg/kg administered twice daily (5 mg/kg/day) up to a maximum recommended dose of 10 mg/kg twice daily (20 mg/kg/day).
For further information refer to the Product Information.
Pregnancy category
B2Drugs which have been taken by only a limited number of pregnant women and women of childbearing age, without an increase in the frequency of malformation or other direct or indirect harmful effects on the human fetus having been observed.Studies in animals are inadequate or may be lacking, but available data show no evidence of an increased occurrence of fetal damage.The use of any medicine during pregnancy requires careful consideration of both risks and benefits by the treating health professional. This must not be used as the sole basis of decision making in the use of medicines during pregnancy. The TGA does not provide advice on the use of medicines in pregnancy for specific cases. More information is available from obstetric drug information services in your State or Territory.
What was approved
Epidyolex (cannabidiol) was approved for the following therapeutic use:
Epidyolex is indicated for use as adjunctive therapy of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) for patients 2 years of age and older.
What is this medicine and how does it work
The precise mechanisms by which cannabidiol exerts its anticonvulsant effects in humans are unknown. Cannabidiol does not exert its anticonvulsant effect through interaction with cannabinoid receptors. Cannabidiol reduces neuronal hyper-excitability through modulation of intracellular calcium via G protein-coupled receptor 55 (GPR55) and transient receptor potential vanilloid 1 (TRPV-1) channels, as well as modulation of adenosine-mediated signalling through inhibition of adenosine cellular uptake via the equilibrative nucleoside transporter 1 (ENT-1).
What post-market commitments will the sponsor undertake
- Epidyolex (cannabidiol) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicine Information (CMI) for Epidyolex must include the black triangle symbol and mandatory accompanying text for five years, which starts from the date that the sponsor notifies the TGA of supply of the product.
The Epidyolex European Union (EU)-Risk Management Plan (RMP) (version 1.0, dated 2 July 2019, data lock point 1 May 2017), with Australian Specific Annex (version 1.0, dated 23 March 2020), included with submission PM-2020-00157-1-1, and any subsequent revisions, as agreed with the TGA will be implemented in Australia.
An obligatory component of risk management plans is routine pharmacovigilance. Routine pharmacovigilance includes the submission of periodic safety update reports (PSURs).
Reports are to be provided in line with the current published list of EU reference dates and frequency of submission of PSURs until the period covered by such reports is not less than three years from the date of the approval letter.
The reports are to at least meet the requirements for PSURs as described in the European Medicines Agency's Guideline on good pharmacovigilance practices (GVP) Module VII-periodic safety update report (Rev 1), Part VII.B Structures and processes. Note that submission of a PSUR does not constitute an application to vary the registration.