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Device/Product name
Active Ingredient
Gilteritinib (as fumarate)
Date of decision
Submission type
New chemical entity
ATC codes
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 Xospata 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, evaluated through Priority Review.

Description Date
Positive Designation (Orphan) 4 April 2019
Submission dossier accepted and first round evaluation commenced 23 August 2019
Evaluation completed 16 March 2020
Delegate's overall benefit-risk assessment 19 March 2020
Sponsor's pre-Advisory Committee response Not applicable
Advisory Committee meeting Not applicable
Registration decision (Outcome) 26 March 2020
Completion of administrative activities and registration on ARTG 2 April 2020
Number of working days from submission dossier acceptance to registration decision* 144

*Target timeframe for priority applications is 150 working days from acceptance for evaluation to the decision.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
Film coated tablet
40 mg
Other ingredients

Tablet core: Mannitol, Hyprolose, Magnesium Stearate

Coating: Hypromellose, Purified talc, Macrogol 8000, Titanium dioxide, Iron oxide yellow

Blister pack
Pack sizes
84 film coated tablets (21 tablets x 4 blisters)
Routes of administration

Treatment with Xospata should be initiated and supervised by a physician experienced in the use of anti-cancer therapies.

Before taking Xospata, relapsed or refractory AML patients must have confirmation of FMS-like tyrosine kinase 3 (FLT3) mutation (internal tandem duplication (ITD) or tyrosine kinase domain (TKD)).


The recommended starting dose of Xospata is 120 mg (three 40 mg tablets) once daily.

Blood chemistries, including creatine phosphokinase, should be assessed prior to initiation of treatment, on Day 15 and monthly for the duration of treatment.

An electrocardiogram (ECG) should be performed before initiation of Xospata treatment, on Day 8 and 15 and prior to the start of the next three subsequent months of treatment. In addition, an ECG should be performed following the same schedule in case of dose increase (see Product Information Section 4.4 Special warnings and precautions for use and section 4.8 Adverse effects (Undesirable effects)).

Treatment should continue until the patient is no longer clinically benefiting from Xospata or until unacceptable toxicity occurs. Response may be delayed; therefore, continuation of treatment at the prescribed dose for up to 6 months should be considered to allow time for a clinical response.

In the absence of a response (patient did not achieve a composite complete remission (CRc)) after 4 weeks of treatment, the dose can be increased to 200 mg (five 40 mg tablets) once daily, if tolerated or clinically warranted.

For further information refer to the Product Information.

Pregnancy category
DDrugs which have caused, are suspected to have caused or may be expected to cause, an increased incidence of human fetal malformations or irreversible damage. These drugs may also have adverse pharmacological effects. Accompanying texts should be consulted for further details.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

Xospata (gilteritinib (as fumarate)) was approved for the following therapeutic use:

Xospata is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation.
What is this medicine and how does it work
Gilteritinib fumarate is a FMS-like tyrosine kinase 3 (FLT3) and AXL (as well as other kinases) inhibitor.Gilteritinib inhibits FLT3 receptor signalling and proliferation in cells expressing FLT3 including FLT3-internal tandem duplication (ITD), FLT3-D835Y, and FLT3-ITD-D835Y, and it induced apoptosis in leukaemic cells expressing FLT3-ITD.
What post-market commitments will the sponsor undertake
  • Xospata (gliteritinib) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicines Information (CMI) for Xospata 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 Xospata European Union-Risk Management Plan (EU-RMP) (version 1.0, dated 4 October 2019, data lock point 17 September 2018), with Australian specific Annex (version 0.6, dated Feb 2020), included with submission PM-2019-03406-1-6, to be revised to the satisfaction of 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.

  • In relation to health care professional education material for differentiation syndrome, the sponsor is instructed to: (1) update the ASA with information on how it will be implemented in Australia (including target audience, method of dissemination, timeframe for implementation and how it will be evaluated); (2) provide a copy of the materials including Black Triangle symbol and wording for review and approval prior to product supply; and (3) implement an evaluation of the effectiveness of the material through assessing healthcare professional awareness and clinical knowledge of differentiation syndrome (for example using a cross-sectional study among healthcare professionals).

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