Rare Daily Staff

The U.S. Food and Drug Administration approved expanded use of Servier Pharmaceuticals’ Tibsovo to include the treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test.

Cholangiocarcinoma is a rare, aggressive cancer of the bile ducts within and outside of the liver. An estimated 8,000 people in the United States are diagnosed with cholangiocarcinoma each year. However, the actual number of these cases is likely to be higher, as cholangiocarcinoma can be hard to diagnose, and may be misclassified as other types of cancer.

Tibsovo is the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma.

The supplemental New Drug Application for Tibsovo received Priority Review, which accelerated the review timeline and is typically given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists.

“Servier has been focused on exploring the significant potential of inhibiting mutant IDH enzymes as a novel approach to treating cancers with high unmet needs, including cholangiocarcinoma,” said David Lee, CEO of Servier Pharmaceuticals.

The FDA approval of this indication is supported by data from the ClarIDHy study, the first and only randomized phase 3 trial for previously treated IDH1-mutated cholangiocarcinoma. Results from the ClarIDHy study demonstrated a statistically significant improvement in the primary endpoint of progression-free survival (PFS) by an independent review committee. The median PFS was 2.7 months for Tibsovo compared to 1.4 months for placebo. Some 32 percent remained free of progression or death at 6 months and 22 percent at 12 months compared to none on the placebo arm. 

The study also showed the key secondary endpoint of overall survival (OS) favoring patients randomized to Tibsovo compared to those randomized to placebo. Nevertheless, statistical significance was not reached on this secondary endpoint.

The safety profile observed in the study was consistent with previously published data. The most common adverse reactions (≥15 percent) in patients with cholangiocarcinoma were fatigue, nausea, abdominal pain, diarrhea, cough, decreased appetite, ascites, vomiting, anemia, and rash.

“Patients living with IDH1-mutated cholangiocarcinoma, especially those whose disease progresses following chemotherapy, are in urgent need of new treatment options,” said Rachna Shroff, associate professor of medicine at the University of Arizona, and chief of GI medical oncology at the University of Arizona Cancer Center. “In addition to an acceptable safety profile, Tibsovo demonstrated an impressive, significant benefit in progression-free survival, underscoring its importance as a new option for patients battling this aggressive cancer.”

Photo: David Lee, CEO of Servier Pharmaceuticals

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