Novartis Wins Breakthrough Status for AML Candidate

The FDA has awarded Novartis with breakthrough therapy designation for PKC412 — also known as midostaurin — an experimental treatment for adults with newly diagnosed acute myeloid leukemia who are FLT3 mutation-positive.

Patients who received midostaurin in conjunction with chemotherapy experienced a substantial increase in overall survival, compared with those on chemotherapy alone.

In a Phase 3 study, patients on the study drug experienced median overall survival of 74.7 months, versus 25.6 months in the placebo group. Worldwide regulatory submissions for midostaurin are set to begin during the first half of the year. There are no approved targeted AML treatments at this time, according to the company.