Lirafugratinib for Cholangiocarcinoma (FGFR2 fusion)
The FDA is expected to decide on Lirafugratinib for Cholangiocarcinoma (FGFR2 fusion) by September 27, 2026, under a NDA from Elevar Therapeutics.
What is being decided
Elevar Therapeutics has a NDA under FDA review for Lirafugratinib in Cholangiocarcinoma (FGFR2 fusion). Selective FGFR2 inhibitor targeting FGFR2 fusion-driven tumors.
Who this decision matters to
Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine. It can occur inside the liver (intrahepatic), at the junction where bile ducts exit the liver (perihilar), or in the bile ducts outside the liver (distal). It is often diagnosed at an advanced stage because symptoms appear late.
Prevalence: About 8,000 new cases per year in the U.S.; rising incidence worldwide. See our full Cholangiocarcinoma page for current treatments, recruiting trials, and community resources.
What each outcome would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. CRLs are often about manufacturing or data presentation rather than efficacy, and resubmission is common. Either way, this page updates with the outcome and what it means.
Where Cholangiocarcinoma treatment stands today
Comprehensive genomic profiling is strongly recommended for all cholangiocarcinoma patients. About 15-20% of intrahepatic CCA has FGFR2 fusions (treatable with pemigatinib, futibatinib, or erdafitinib), and about 10-15% has IDH1 mutations (treatable with ivosidenib). Clinical trials are actively evaluating new targeted therapies, immunotherapy combinations, and novel approaches. The Cholangiocarcinoma Foundation is an excellent resource for finding trials and connecting with specialists. Many academic cancer centers have dedicated hepatobiliary programs with expertise in CCA. Getting a second opinion at a specialized center early in your diagnosis can significantly impact treatment options.
Meanwhile, 287 Cholangiocarcinoma trials are recruiting
Whatever the FDA decides here, research on Cholangiocarcinoma does not stop. A few currently enrolling studies, US sites first:
Other drugs Trial Friend tracks for Cholangiocarcinoma
Get notified when new cholangiocarcinoma trials open or existing trials change status, add sites, or update eligibility.
Frequently asked questions
When will the FDA decide on Lirafugratinib?
The FDA's target decision date (PDUFA date) for Lirafugratinib in Cholangiocarcinoma (FGFR2 fusion) is September 27, 2026, disclosed by Elevar Therapeutics. The agency can act before this date and occasionally runs past it.
What is Lirafugratinib being reviewed for?
Elevar Therapeutics submitted a NDA for Lirafugratinib in Cholangiocarcinoma (FGFR2 fusion). Selective FGFR2 inhibitor targeting FGFR2 fusion-driven tumors.
What happens after the Lirafugratinib decision?
If approved, availability is not immediate: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. If the FDA issues a complete response letter, the application was not approved in its current form; CRLs are often about manufacturing or data presentation rather than efficacy, and sponsors frequently resubmit. This page updates with the outcome either way.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from Elevar announcement. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified weekly.