FDA Approves Ivosidenib for Advanced or Metastatic Chloangiocarcinoma
On August 25, 2021, the FDA approved ivosidenib with a recommended dosage of 500 mg orally once daily for patients who received treatment for locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 (1DH1) mutation.
Additionally, the FDA approved the Oncomine Dx Target Test as a diagnostic device for 1DH1 mutation detections in cholangiocarcinoma.
Researchers enrolled 185 patients to the randomized, multicenter, ClarIDHy clinical trial with eligibility criteria met for disease progression, including no more than two prior regimens and at least one gemcitabine- or 5-flurouracil-containing regimen.
All patients were randomized to either receive ivosidenib 500 mg orally once daily or matched with placebo until disease progression or unacceptably toxicity.
The primary endpoint was progression-free survival (PFS), which was evaluated through an independent review committee according to RECIST 1.1
Ivosidenib demonstrated statistically significant improvement in PFS (HR 0.37; 95% CI: 0.25, 0.54; p<0.0001). Overall survival (OS) rates did not show statistical benefit (0.79; 95% CI: 0.56, 1.12; p=0.093). Notably, 70% of patients randomized to placebo crossed over to the ivosidenib arm after radiographic disease progression.
Various adverse events (AEs) occurred (≥15%) with incidents of fatigue, nausea, abdominal pain, diarrhea, cough, decreased appetite, ascites, vomiting, anemia, and rashes.
Results from the trial granted FDA approval with a recommended ivosidenib dosage of 500 mg orally once daily with or without food until disease progression or unacceptable toxicity for patients with cholangiocarcinoma. – Alexa Stoia
