Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. announced today that the U.S. Food and Drug Administration (FDA) has approved Lytgobi tablets for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.
This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
“Lytgobi is an effective, well-tolerated therapy for patients with intrahepatic CCA that can be taken orally,” said Tim Whitten, President and CEO of Taiho Oncology, Inc. “This approval is an important milestone for patients and may provide hope for improved outcomes. As someone whose family has been impacted by cholangiocarcinoma, I’m acutely aware of the impact this disease can have on the patient and their loved ones.”
As a whole, cholangiocarcinoma is an aggressive cancer of the bile ducts and is diagnosed in approximately 8,000 individuals each year in the U.S.1 This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) forms of the disease. Approximately 20% of patients diagnosed with CCA have the intrahepatic form of the disease.2,3 Within this 20%, approximately 10-16% of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation.4,5,6,7,8 Lytgobi covalently binds to FGFR2 and inhibits the signaling pathway.9 The other approved FGFR inhibitors are reversible ATP-competitive inhibitors.10,11,12
“Lytgobi is a key example of the potential of precision medicine in iCCA and represents another advance in the treatment of this rare and challenging disease,” said medical oncologist Lipika Goyal, MD, MPhil, of the Massachusetts General Hospital Cancer Center and lead investigator of the pivotal study that led to the approval of Lytgobi. “I am encouraged that treatment options continue to expand and evolve for this disease through the dedicated efforts of many over several years.”
The approval of Lytgobi is based on the results of the primary analysis of the FOENIX*-CCA2 trial, a global Phase 2 open-label trial evaluating 103 patients with unresectable, locally advanced or metastatic iCCA harboring FGFR2 gene rearrangements including fusions. In this trial, patients received Lytgobi orally once daily at a dose of 20mg until disease progression or unacceptable toxicity.
The trial met its primary endpoint with an objective response rate of 42% as measured by independent central review. The median duration of response (DOR) was 9.7 months, with 72% of responses lasting at least six months. The most common (≥20%) adverse reactions were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, dry skin, arthralgia, dysgeusia, abdominal pain, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting.
Lytgobi was discovered by Taiho Oncology’s parent company, Taiho Pharmaceutical, which continues to co-develop this product for other potential tumor types. “The Taiho group is working as one to optimize this agent for the patients who are waiting,” said Teruhiro Utsugi, Senior Managing Director at Taiho Pharmaceutical.
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