In May, the FDA granted priority review to the IDH1 inhibitor ivosidenib. Then, on August 25, 2021, the FDA approved the drug for use in adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation, as detected by an FDA-approved test.
After recently granting ivosidenib priority review status, the FDA wasted no time in approving the IDH1 inhibitor for use in cholangiocarcinoma just weeks later.
This drug, which is sold under the brand name Tibsovo, is already used to treat IDH1-mutant acute myeloid leukemia. However, although IDH1 gene mutations are found in some advanced cholangiocarcinoma tumors, there have been no approved systemic therapies for IDH1-mutated cholangiocarcinoma until now.
A priority review usually means the FDA will review a drug within 6 months, instead of 10 months with a standard review. But considering the success of ivosidenib in clinical trials, it’s no surprise this drug was approved in half the time. And with liver cancer and cholangiocarcinoma predicted to be the third leading cause of cancer death by 2040, this news brings hope twice as fast to those who need it most.
Ivosidenib was investigated in a randomized, multicenter, double-blind, placebo-controlled Phase 3 clinical trial – Study AG120-C-005. The 185 participants were adults with locally advanced or metastatic IDH1-mutant cholangiocarcinoma, whose disease must have progressed following at least one, but not more than two, prior treatments, including at least one gemcitabine or 5-flurouracil based chemotherapy regimen.
The primary efficacy endpoint of Study AG120-C-005 was progression-free survival. The patients were randomized to receive either ivosidenib 500 mg orally once daily or matched placebo until disease progression or unacceptable toxicity. Fortunately, the trial demonstrated a statistically significant improvement in progression-free survival for patients randomized to ivosidenib, leading to its approval from the FDA.
The Rare Initiative’s Dr. Lipika Goyal has been working alongside Servier Pharmaceuticals to develop ivosidenib. Bill Cance, Chief Medical and Scientific Officer at the American Cancer Society, took to Twitter to praise the game-changing oncologist for her role in bringing another treatment option to those with the most limited options and the least hope.
