Trikafta
Registration timeline
The following table summarises the key steps and dates for this application.
| Description | Date |
|---|---|
| Designation (Orphan) | 11 December 2019 |
| Submission dossier accepted and first round evaluation commenced | 31 March 2020 |
| First round evaluation completed | 31 August 2020 |
| Sponsor provides responses on questions raised in first round evaluation | 30 September 2020 |
| Second round evaluation completed | 12 November 2020 |
| Delegate's overall benefit-risk assessment and request for Advisory Committee advice | 23 December 2020 |
| Sponsor's pre-Advisory Committee response | 18 January 2021 |
| Advisory Committee meeting | 4 and 5 February 2021 |
| Registration decision (Outcome) | 17 March 2021 |
| Completion of administrative activities and registration on ARTG | 24 March 2021 |
| Number of working days from submission dossier acceptance to registration decision* | 218 |
*Statutory timeframe for standard applications is 255 working days
Elexacaftor/tezacaftor/ivacaftor tablets: hypromellose, hypromellose acetate succinate, sodium lauryl sulfate, croscarmellose sodium, microcrystalline cellulose, magnesium stearate, and Opadry complete film coating system 20A130036 orange
Ivacaftor tablets: silicon dioxide, croscarmellose sodium, hypromellose acetate succinate, lactose monohydrate, magnesium stearate, microcrystalline cellulose, sodium lauryl sulfate, carnauba wax. Opadry II complete film coating system 85F90614 blue, and Opacode monogramming ink S-1-17823 black
Adults, adolescents, and children aged 12 years and older
The recommended dose is two tablets (each containing elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg) taken in the morning and one tablet (containing ivacaftor 150 mg) taken in the evening, approximately 12 hours apart.
For further information refer to the Product Information.
Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was approved for the following therapeutic use:
Trikafta is indicated for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
- Trikafta (elexacaftor/tezacaftor/ivacaftor) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicines Information (CMI) for Trikafta must include the black triangle symbol and mandatory accompanying text for five years, which starts from the date that the sponsor notifies the Therapeutic Goods Administration (TGA) of supply of the product.
- The Trikafta European Union (EU)-risk management plan (RMP) (version 1.1, dated 21 August 2020; data lock point dated 20 July 2020), with Australian specific Annex (version 2.0, dated 18 September 2020), included with submission PM-2020-00642-1, 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.