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Quizartinib

Extended indication Monotherapy for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) who
Therapeutic value Possible added value
Total cost 975,000.00
Registration phase Registration application pending

Product

Active substance Quizartinib
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Leukemia
Extended indication Monotherapy for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) who are FLT3-ITD mutation-positive.
Manufacturer Daiichi Sankyo
Mechanism of action Tyrosine kinase inhibitor
Route of administration Oral
Therapeutical formulation Film-coated tablet
Budgetting framework Intermural (MSZ)
Additional remarks Quizartinib potently inhibits FLT3, a kinase that is mutated in approximately one-third of acute myeloid leukemia (AML) cases. Quizartinib specifically inhibits FLT3-ITD mutations. Patients with FLT3 mutations are less responsive to traditional therapies: For patients with FLT3-ITD mutations in the relapsed or refractory AML indication, the most aggressive form of the disease with the worst prognosis, no targeted therapy is currently available.

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Submission date October 2018
Expected Registration June 2019
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Midostaurin, salvage chemotherapy (e.g. FLAG-IDA, MEC or LoDAC).
Therapeutic value Possible added value
Substantiation Improved OS (primary endpoint) and EFS (secundary endpoint) over salvage chemotherapy. Geen directe vergelijking met midostaurine, maar indicatie is anders dan midostaurine (R/R setting).
Duration of treatment Median 4 month / months
Frequency of administration 1 times a day
References NCT02039726; Cortes et al. EHA Learning Center. Cortes J. Jun 16, 2018; 218882; Cortes et al. Lancet Oncol. 2018 Jul;19(7):889-903; Cortes et al. Blood. 2018 Aug 9;132(6):598-607
Additional remarks Median drug exposure: 4 cycles, at a 28-day cycle length (until lack of clinical benefit, toxicity, or receipt of transplant). Dose: 53.0 mg quizartinib (equivalent to 60 mg quizartinib dihydrochloride) with 26.5 mg quizartinib (equivalent to 30 mg quizartinib dihydrochloride) lead-in; 26.5 mg quizartinib (equivalent to 30 mg quizartinib dihydrochloride) with 17.7 mg quizartinib (equivalent to 20 mg quizartinib dihydrochloride) lead-in for patients treated with a strong CYP3A inhibitor

Expected patient volume per year

Patient volume

10 - 20

Market share is generally not included unless otherwise stated.
References Fabrikant; NKR; Levis. Hematology Am Soc Hematol Educ Program. 2013; 2013: 220–226; kanker.nl. expert opinie
Additional remarks 674 diagnoses AML in 2015. Ongeveer 30% heeft een FLT3 mutatie (202). Bij ongeveer de helft van het aantal patiënten komt AML terug (101). De fabrikant verwacht een marktaandeel van 50% (51). In de praktijk zal het waarschijnlijk om beperkte hoeveelheid patiënten gaan die gebridged worden naar allo HCT (met name ook omdat quizartinib pancytopenie geeft moet er gebridged worden naar allo HCT), inschatting expert opinie: 10-20 patiënten.

Expected cost per patient per year

Cost 60,000.00 - 70,000.00
References EPAR midostaurine; medicijnkosten.nl
Additional remarks Aangenomen wordt dat quizartinib vergelijkbaar is aan midostaurine. Gemiddeld kregen patiënten 12.763 mg midostaurine tijdens de klinische studies, dit komt neer op 511 tabletten van 25 mg. Totale kosten voor 511 tabletten midostaurine bedragen €64.552,09.

Potential total cost per year

Total cost

975,000.00

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Monotherapy for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who are FLT3-ITD mutation-positive
References Fabrikant; QuANTUM First (ongoing PhIII clinical trial)
Additional remarks Expected registration date Q4 2021

Other information

There is currently no futher information available.