NDA Submitted to FDA for Ivosidenib for Previously Treated IDH1-Mutated Cholangiocarcinoma
A supplemental new drug application has been submitted to the FDA for ivosidenib tablets (Tibsovo) as a potential therapeutic option for patients with previously treated, IDH1-mutated cholangiocarcinoma.
A supplemental new drug application (sNDA) has been submitted to the FDA for ivosidenib tablets (Tibsovo) as a potential therapeutic option for patients with previously treated, IDH1-mutated cholangiocarcinoma.1 The application is based on findings from the phase 3 ClarIDHy trial (NCT02989857), which has shown that ivosidenib has resulted in a 63% reduction in the risk of disease progression or death compared with placebo in previously treated patients with IDH1-mutated, advanced cholangiocarcinoma.2
Specifically, the median progression-free survival (PFS) was 2.7 months with ivosidenib vs 1.4 months with placebo (HR, 0.37; 95% CI, 0.25-0.54; 1-sided P <.0001). The PFS rate with ivosidenib at 6 months was 32%, while the rate was 22% at 12 months. No participants who received placebo were free of progression at either of those time points. The disease control rates at 6 months and 12 months were 53% and 28%, respectively.
Data from the final overall survival (OS) analysis of the trial also showed that ivosidenib resulted in a 21% reduction in the risk of death vs placebo in this population.3 The median OS with ivosidenib was 10.3 months vs 7.5 months with placebo (HR, 0.79; 95% CI, 0.56-1.12; 1-sided P = .093). At 6 months, the OS rate with ivosidenib was 69% vs 57% with placebo. Notably, the rates were not adjusted for crossover. At 12 months, the OS rates in the investigative and control arms were 43% and 36%, respectively.
“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, MD, chief medical officer at Agios, stated in a press release. “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.”