Agios has hit another big goal in its decade-long track record. The FDA today signaled the accelerated approval of ivosidenib (AG-120) for advanced acute myeloid leukemia.
This marks the second drug approval for Agios, a biotech company founded by David Schenkein, which he’s fond of saying got started with a “blank sheet of paper.”
That sheet is getting quite crowded now.
The new drug will be marketed as Tibsovo.
Billed as “the first targeted treatment for R/R AML patients with an IDH1 mutation,” Agios submitted it back in February with nothing but early-stage data that had been on display at ASH.
The OK comes close to a year after the biotech’s first approval for Idhifa, an IDH2 drug for another slice of the AML market being handled by its partners at Celgene. And just like Idhifa, the FDA hurried this new approval along with a priority review.
Together, the two drugs cover 20% of the AML market.
This new drug was tested in an open label study, with no control arm. Investigator found:
•CR+CRh rate of 32.8% (57 of 174 patients) (95% CI: 25.8, 40.3).
•The CR rate was 24.7% (43 of 174 patients) (95% CI 18.5, 31.8) and the CRh rate was 8% (14 of 174 patients) (95% CI 4.5, 13.1).
•Median duration of CR+CRh was 8.2 months (95% CI: range 5.6, 12 months).
•For patients who achieved a CR or CRh, the median time to best response of CR or CRh was 2.0 months (range, 0.9 to 5.6 months).
•Among the 110 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 41 (37.3%) became independent of RBC and platelet transfusions during any 56-day post-baseline period.
Congrats to Agios (AGIO) for approval of its second drug for AML (TIBSOVO). Fastest ever approval of distinct drugs for two independent mechanisms. First company to have two drugs with internally discovered pathways in a decade from start. ARCH named it and seeded with Flagship.
— Robert Nelsen (@rtnarch) July 20, 2018
“The FDA approval of Tibsovo – our first wholly owned drug and the second approved medicine from our research platform in less than a year – is an incredibly exciting milestone for our company and, importantly, for the approximately 6-10% of AML patients with an IDH1 mutation who have been waiting for new treatment options that work radically different than conventional chemotherapy,” said Schenkein.