Glasdegib given positive opinion by the EMA CHMP for use in patients with newly diagnosed AML

On April 30, 2020, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion recommending approval of glasdegib, in combination with low-dose cytarabine (LDAC), for the treatment of newly diagnosed de novo or secondary acute myeloid leukaemia (AML) in patients ineligible for standard chemotherapy.

Glasdegib is an oral smoothened inhibitor decreasing glioma-associated oncogene transcription factor activity. It is already approved by the United States Food and Drug Administration (FDA), in combination with LDAC, for the treatment of patients with newly diagnosed AML ineligible for chemotherapy.

Glasdegib is well tolerated, with the most frequent severe adverse event being fatigue, and improves overall survival when used in combination with LDAC. In the ‘How I treat’ series in Blood, which covers the use of new drugs in different case studies, Courtney DiNardo and Andrew Wei reported that, even though the overall response rate for glasdegib plus LDAC was modest, the combination had better survival compared with LDAC alone in a randomized trial (read more here).