RYDAPT

What it is

Rydapt contains 25 mg of midostaurin per soft gelatin capsule. Midostaurin is a protein kinase inhibitor used as an antineoplastic agent. It inhibits multiple receptor tyrosine kinases, including FLT3 and KIT kinase, and inhibits FLT3 receptor signaling to induce cell cycle arrest and apoptosis in leukemic cells.

Approved indications —

In combination with standard anthracycline and cytarabine induction and cytarabine consolidation chemotherapy, followed in patients in complete response by single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation-positive — For the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasms (SM-AHN), or mast cell leukaemia (MCL).

Dosing overview

For AML, the recommended dose of Rydapt is 50 mg twice daily, dosed on days 8–21 of induction and consolidation chemotherapy cycles and then twice daily as single agent maintenance therapy until relapse for up to 12 cycles of 28 days each. For advanced SM, the recommended starting dose is 100 mg twice daily.

Key safety warnings

Neutropenia has occurred in patients receiving Rydapt as monotherapy and in combination with chemotherapy, with severe neutropenia (absolute neutrophil count less than 0.5 × 10⁹/L) generally reversible by withholding Rydapt until recovery or discontinuation. White blood cells should be monitored regularly, especially at treatment initiation. In advanced SM studies, cardiac dysfunction such as congestive heart failure, some of which were fatal, and transient decreases in left ventricular ejection fraction have occurred. In patients at risk, Rydapt should be used with caution and patients should be closely monitored at baseline and during treatment. Interstitial lung disease and pneumonitis, some cases fatal, have occurred in patients treated with Rydapt monotherapy or in combination with chemotherapy. Patients should be monitored for pulmonary symptoms indicative of interstitial lung disease or pneumonitis and Rydapt should be discontinued in patients who experience pulmonary symptoms indicative of interstitial lung disease or pneumonitis without an infectious aetiology which are ≥Grade 3. Rydapt is subject to additional monitoring in Australia to allow quick identification of new safety information.

Contraindications

Rydapt is contraindicated in patients with hypersensitivity to midostaurin or to any of the excipients. Concomitant administration of potent CYP3A4 inducers (such as rifampicin, St. John's Wort, carbamazepine, enzalutamide, or phenytoin) is contraindicated.

PBS listing

Rydapt 25 mg capsules are listed on the PBS with authority required restriction and an ex-manufacturer price of A$9211.37.

Regulatory history

Rydapt (midostaurin 25 mg soft capsule) was first listed on the ARTG on 17 May 2018. In July 2018, the PBAC recommended Rydapt as a new listing for FLT3 mutation-positive AML under the Highly Specialised Drugs Program with authority required restriction, acknowledging high unmet clinical need and improved outcomes, with acceptably cost-effective pricing and a risk sharing agreement. In March 2025, the PBAC recommended Rydapt as a new listing for adult patients with advanced systemic mastocytosis.