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Agios Submits sNDA for Tibsovo in IDH1-Mutant Bile Duct Cancer

NEW YORK – Agios submitted a supplemental new drug application on Monday to the US Food and Drug Administration for ivosidenib (Tibsovo) to treat IDH1-mutated bile duct cancer.

The submission is supported by data from the Phase III ClarIDHy study, in which the primary endpoint was progression-free survival. Data from the study presented in 2019 showed that patients with IDH1-mutated tumors on ivosidenib had a median progression-free survival of 2.7 months compared to 1.4 months on placebo. 

Agios also requested priority review for the drug, which if granted by the FDA, would put its application on a six-month review clock.

The latest data from that trial, presented in January, showed previously treated, IDH1-mutated bile duct cancer patients taking ivosidenib had a median overall survival of 10.3 months compared to 7.5 months for patients on placebo; this endpoint failed to reach statistical significance. Patients in the placebo arm could crossover to the ivosidenib arm, and when adjusted for crossover, the median overall survival was 5.1 months in the placebo arm, which did yield a statistically significant difference compared to the ivosidenib arm. 

"Cholangiocarcinoma is a rare, aggressive cancer with limited effective therapies, and patients are in desperate need of new treatment options — particularly those who experience disease progression after chemotherapy," Chris Bowden, chief medical officer at Agios, said in a statement. "We are proud of the work we have done on behalf of these patients and look forward to working closely with the FDA during the review of the first oral therapy targeting an IDH1 mutation for patients with previously treated IDH1-mutated cholangiocarcinoma."

Ivosidenib is already approved in the US to treat patients with IDH1-mutated acute myeloid leukemia. However, Agios recently withdrew its marketing application from the European Medicines Agency for ivosidenib in the same indication after negative feedback from the Committee for Medicinal Products for Human Use.