Daurismo is claimed to be the first and only FDA-approved Hedgehog pathway inhibitor for AML.
Daurismo, which is taken in combination with low-dose cytarabine (LDAC), has not been assessed in patients with severe renal impairment or moderate-to-severe hepatic impairment.
Pfizer Oncology global president Andy Schmeltz said: “As our second medicine approved in the last 14 months for patients with acute myeloid leukemia, Daurismo reinforces our commitment to delivering new medicines to patients living with some of the most difficult-to-treat cancers, especially those for which there are limited treatment options available.
“We are proud to now offer these patients for whom intensive chemotherapy is not an option a new oral medicine, taken in combination with low-dose chemotherapy, that may improve their chances of survival.”
The randomized phase 2 study, which formed the basis for the US regulator’s approval, included patients with cardiac disease or mild to moderate kidney disease, who are often excluded from clinical trials.
The Bright AML includes two randomized and placebo-controlled phase 3 trials assessing the addition of Daurismo to intensive or non-intensive chemotherapy in patients with newly diagnosed AML.
The patients with AML will be randomized to receive Daurismo plus cytarabine and daunorubicin or placebo plus cytarabine and daunorubicin in the first study.
The patients with AML for whom intensive chemotherapy is not an option will be randomized to receive Daurismo plus azacitidine or placebo plus azacitidine in the second study.
A separate phase 1b Bright 1012 study has also been designed to assess Daurismo in combination with azacitidine in patients with previously untreated high-risk myelodysplastic syndromes (MDS) or AML.
The 115 patients with newly diagnosed AML were randomized 2:1 to secure Daurismo plus LDAC or LDAC alone in the pivotal, randomized and international phase 2 Bright 1003 trial.
More than half had secondary AML or AML, of the 77 patients treated with Daurismo plus LDAC.
Pfizer Oncology global product development chief development officer Mace Rothenberg said: “Daurismo, a Hedgehog pathway inhibitor, was discovered in Pfizer laboratories and exemplifies our continued commitment to developing medicines that have the potential to advance cancer therapeutics.”