FDA Approves New Oral Drug for AML

Nick Mulcahy

November 21, 2018

The US Food and Drug Administration (FDA) approved glasdegib (Daurismo, Pfizer) tablets for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients aged 75 years or older or those who have comorbidities that disallow use of intensive chemotherapy, the usual standard of care.

Glasdegib, which belongs to a class of drugs known as oral smoothened inhibitors, is for use in combination with low-dose cytarabine (LDAC), a chemotherapy medication.

"Many adults with AML are unable to have intensive chemotherapy because of its toxicities," said Richard Pazdur, MD, of the FDA's Center for Drug Evaluation and Research in a press statement. "Today’s approval gives healthcare providers another tool to use in the treatment of AML patients with various, unique needs."

Almost half of the adults diagnosed with AML are not treated with standard, intensive chemotherapy because of comorbidities and chemotherapy-related toxicities, according to the FDA.

The efficacy and safety of glasdegib were evaluated in a clinical trial of 111 patients with newly diagnosed AML. Patients were randomly assigned to receive either glasdegib plus LDAC or LDAC alone. For those who received glasdebig plus LDAC, median overall survival was 8.3 months; for the patients who received LDAC alone, it was 4.3 months. The difference in overall survival was 4 months and was statistically significant.

Common side effects with glasdegib, reports the FDA, include anemia, fatigue, hemorrhage, febrile neutropenia, muscle pain, nausea, edema, thrombocytopenia, dyspnea, decreased appetite, dysgeusia, mucositis, constipation, and rash.

Healthcare providers should monitor patients for changes in the electrical activity of the heart (ie, QT prolongation).

The FDA granted the glasdegib application a priority review designation. The drug also received an orphan drug designation.

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