EC Grants Conditional Approval for Taiho’s Lytgobi to Treat Adults with Rare Bile Duct Cancer

Rare Daily Staff

The European Commission granted conditional marketing authorization for Taiho Pharmaceutical’s Lytgobi for the treatment of cholangiocarcinoma.

The approval is for Lytgobi monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

Cholangiocarcinoma (CCA) is an aggressive cancer of the bile ducts of the liver. While rare – in Europe, approximately 6,000-8,000 people are diagnosed with CCA1– this disease is associated with poor outcomes and is growing in prevalence worldwide, underscoring the need for new treatment options.

“Today is an important day for current and future patients with CCA as well as the healthcare providers who treat them,” said Peter Foertig, vice president, Medical Affairs, Taiho Oncology Europe, Taiho’s European subsidiary. “Lytgobi is an oral molecularly targeted medication that may provide clinically meaningful outcomes for patients undergoing treatment for CCA.”

The European Commission’s conditional marketing authorization for Lytgobi is based on the data from the FOENIX-CCA2 trial, a global open label trial evaluating 103 patients with unresectable, locally advanced or metastatic intrahepatic (inside the bile ducts of the liver) CCA 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. Within Europe, patients enrolled from France, Germany, Italy, the Netherlands, Spain and the United Kingdom.

A conditional marketing authorization in Europe is granted for medicines that fulfill an unmet medical need to treat serious diseases, and the benefits of having them available earlier outweighs any risks associated with using the medicines while waiting for further evidence. Under the specific obligation to complete post authorization measures for the conditional marketing authorization, Taiho has until October 2027 to provide additional clinical data on Lytgobi.

Lytgobi (futibatinib) is an oral, potent, selective, and irreversible tyrosine kinase inhibitor of FGFR1, 2, 3 and 4. LYTGOBI covalently binds to the ATP binding pocket of FGFR1-4 resulting in the inhibition of FGFR-mediated signal transduction pathways, reduced tumor cell proliferation in tumors with FGFR1-4 genetic aberrations.

In Japan, Lytgobi has been approved by the Japanese Ministry of Health, Labour and Welfare, as a treatment for unresectable biliary tract cancer harboring FGFR2 gene fusions that has progressed after chemotherapy, in June 2023. In September 2022, the U.S. Food and Drug Administration approved Lytgobi for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.