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Astellas Pharma Acute Myeloid Leukemia Drug Gets Positive CHMP Opinion

NEW YORK (GenomeWeb) – Tokyo-based biopharmaceutical firm Astellas Pharma said on Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending its gilteritinib (Xospata) as a monotherapy for the treatment of adult patients who have relapsed or refractory (resistant to treatment) acute myeloid leukemia with a FLT3 mutation (FLT3mut+).

According to the firm, the CHMP decision was based on results of the Phase III ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML. The study enrolled 371 patients with relapsed or refractory AML and FLT3mut+ present in bone marrow or whole blood. Subjects were randomized in a 2:1 ratio to receive gilteritinib or salvage chemotherapy.

Patients who were treated with the oral once-daily therapy had significantly longer overall survival (median of 9.3 months) than those who received salvage chemotherapy (median of 5.6 months), according to the firm.

"The data are encouraging, showing a significant improvement in overall survival, and one-year survival rates doubled when comparing gilteritinib to the current standard of care," said Giovanni Martinelli, a study investigator from the Institute of Hematology, S.Orsola-Malpighi University Hospital, in Bologna, Italy said in a statement released by Astellas. "For relapsed or refractory FLT3mut+ AML patients the current prognosis is poor, with median [overall survival] of less than six months following treatment with salvage chemotherapy. If approved by the EC, gilteritinib has the potential to change the treatment landscape."

The CHMP opinion will be reviewed by the European Commission, which approves medicines for the 28 European Union member countries as well as Iceland, Norway, and Liechtenstein. The drug last year received an Orphan Designation from the European Commission and received accelerated assessment from the EMA.

Gilteritinib already has marketing approval in the US and Japan for the treatment of adult patients who have relapsed or refractory FLT3mut+ AML.

The drug was discovered through a research collaboration with Kotobuki Pharmaceutical Co., and Astellas holds exclusive global rights to develop, manufacture, and commercialize it.