New Option Soon for Some European Patients With Leukemia

Zosia Chustecka

September 20, 2019

A new option for some patients with leukemia looks likely to be available soon in Europe.

At its September meeting, the European Medicine Agency's human medicines committee (CHMP) recommended granting a marketing authorization for gilteritinib (Xospata, Astellas) for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with an FLT3 mutation.

A European approval usually follows 6 weeks later.

AML affects approximately 1 in 10,000 people in the European Union, the committee noted. The FTL3 mutation occurs in about one third of AML patients, and makes the disease highly aggressive. 

Gilteritinib has Orphan Drug status, and was reviewed under EMA's accelerated assessment procedure, reserved for medicines of major public health interest.

The product is already available for the same indication in the United States, where it was approved in November 2018.

"Impressive Activity"

Experts have commented on the "impressive" activity that the drug has shown in the treatment of FLT3-mutated relapsed/refractory AML.

Single-agent gilteritinib achieved significantly longer overall survival (OS) and response rates (ORR) than standard chemotherapy in this patient population in the phase 3 ADMIRAL study, which was presented earlier this year at the American Association for Cancer Research (AACR) 2019 annual meeting.

Median OS for patients who received gilteritinib was 9.3 months, compared with 5.6 months for patients who received salvage chemotherapy (hazard ratio, 0.64; 95% confidence interval, 0.49 - 0.83; P =.0004). Rates of 1-year survival were 37% for patients who received gilteritinib compared with 17% for patients who received salvage chemotherapy.

"For these patients with FLT3-mutated AML, gilteritinib represents a new standard of care," lead author Alexander E. Perl, MD, associate professor of hematology/oncology at the Perelman School of Medicine, University of Pennsylvania, Philadelphia, told Medscape Medical News at the time.

Standard chemotherapy, which historically was the only option, provides low remission rates of a short duration and a median survival of 2 to 4 months, Perl explained.

The superior survival seen with gilteritinib, along with its modest side effects and once daily oral dosing, means that it can be given in an outpatient setting, Perl pointed out. "This represents a paradigm shift for salvage therapy in AML," he said.

"The single-agent activity of gilteritinib in patients with relapsed or refractory AML is very impressive," Michael Caligiuri, MD, medical oncologist at City of Hope cancer center in Duarte, California, told Medscape Medical News

He pointed out that this is a real advantage for patients and warrants moving the drug into front-line therapy. He also noted there may be an opportunity for curing some patients with hematopoietic stem cell transplantation following treatment with gilteritinib.

Generic Drugs for Blood Cancers

At the same meeting, the CHMP also recommended granting marketing authorizations for three generic medicines that are used in the treatment of hematologic malignancies:


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