Xospata (Gilteritinib)

What it is and what it treats

Gilteritinib, marketed as Xospata, is a targeted tyrosine kinase inhibitor developed by Astellas Pharma. It is authorised for adults with relapsed or refractory acute myeloid leukaemia (AML) whose leukaemic cells carry a FLT3 mutation — either an internal tandem duplication (FLT3-ITD) or a tyrosine kinase domain mutation (FLT3-TKD) — detected using an approved companion diagnostic test.

FLT3 mutations occur in roughly one in three adults with AML and are associated with a higher risk of relapse and poorer prognosis with conventional chemotherapy alone. Gilteritinib selectively inhibits the FLT3 receptor, including both ITD and TKD mutant forms, and also inhibits AXL kinase, a pathway implicated in resistance to other FLT3 inhibitors. In the pivotal randomised trial supporting its approval, gilteritinib monotherapy was associated with longer overall survival than salvage chemotherapy in this relapsed or refractory population.

Because efficacy is confined to FLT3-mutated disease, Xospata is not used in AML without a documented FLT3 mutation. As mutation status can change between diagnosis and relapse, retesting at the time of relapse is described in guidance to confirm eligibility before treatment is considered by the treating haematologist.

Presentations and dosage forms

• 40 mg film-coated tablets
• Supplied in blister packaging for oral administration
• Typically taken as multiple tablets once daily to reach the prescribed total dose

How it is administered

The approved label describes a starting dose of 120 mg (three 40 mg tablets) taken orally once daily, with or without food, continued until disease progression or unacceptable toxicity, as determined by the treating haematologist. Tablets should be swallowed whole and not crushed, broken or chewed.

Dose modifications for specific adverse reactions, including differentiation syndrome, QT-interval prolongation or posterior reversible encephalopathy syndrome, are described in the prescribing information and are managed by the treating clinical team. Liver function and creatine phosphokinase are typically monitored during treatment, as is a baseline and periodic ECG given the associated QT-prolongation risk.

Differentiation syndrome is a labelled, potentially fatal complication of FLT3 inhibitor therapy, arising when treated leukaemic cells rapidly differentiate. It can present with fever, dyspnoea, pulmonary infiltrates, hypotension, rapid weight gain or peripheral oedema, sometimes within the first days of treatment and occasionally later in the course. The label describes prompt initiation of corticosteroids and haemodynamic monitoring if differentiation syndrome is suspected, with temporary interruption of gilteritinib in severe cases — decisions made by the treating physician based on the clinical picture.

Storage and handling

• Store below 30 °C in the original packaging
• Keep the blister strip in the outer carton to protect from light
• Keep out of the reach of children
• Do not use beyond the expiry date printed on the pack

Side effects

The prescribing information for Xospata, based on trial data in relapsed or refractory FLT3-mutated AML, describes a range of common reactions alongside several labelled serious risks that require active monitoring.

Commonly reported

Serious effects requiring medical attention

Patients should seek urgent medical attention for breathlessness, rapid weight gain, fever or swelling, and should report suspected adverse reactions through their national pharmacovigilance scheme, such as the MHRA Yellow Card Scheme or FDA MedWatch.

Availability and supply route

Xospata is registered in a number of countries, but authorised pack sizes and continuous commercial availability differ by market. Because it is used in a genetically defined subset of relapsed or refractory AML, treating centres sometimes need to arrange supply outside their immediate domestic distribution network to match patient need with confirmed availability.

Where local registration or stock is temporarily unavailable, hospitals and specialist pharmacies may use named-patient import mechanisms, permitted under most national regulatory frameworks, to obtain an internationally authorised medicine for a specific, identified patient under direct physician responsibility. This requires a valid prescription, confirmed FLT3-mutation testing and appropriate import documentation, and is separate from any form of retail sale.

Pharmacy Remedy is a licensed pharmaceutical wholesaler based in Turkey that supplies hospitals, haematology and oncology clinics, and licensed pharmacies against valid prescriptions and institutional documentation. It operates within licensed business-to-business pharmaceutical supply chains and does not sell Xospata, or any prescription oncology medicine, directly to members of the public.

Frequently asked questions

Primary sources

• FDA — Xospata prescribing information
• EMA — Xospata EPAR

Further reading and regulatory routes

• How to verify a medicine is genuine
• Why medicines cost less in Türkiye
• WHO falsified medicine alerts
• Named patient supply
• UAE import guide
• For healthcare professionals
• All medicine monographs

Related medicines

• Tasigna (nilotinib)
• Calquence (acalabrutinib)
• Erivedge (vismodegib)