Pemigatinib for locally advanced or metastatic, relapsed or refractory cholangiocarcinoma with FGFR2 fusion or rearrangement

Pemigatinib is in clinical development for adults with locally advanced or metastatic bile duct cancer (cholangiocarcinoma) with FGFR2 fusion or rearrangement who have failed previous treatment. Advanced cholangiocarcinoma begins in the bile duct but spreads to other parts of the body and often is not diagnosed until a late stage. Symptoms of cholangiocarcinoma include yellowing of the skin and whites of eyes (jaundice), itchy skin, dark urine, loss of appetite, persistent tiredness, abdominal pain, fever, chills and shivering. The causes of cholangiocarcinoma are not yet fully understood but include smoking, obesity and alcohol consumption.
Pemigatinib is administered orally and it works by blocking receptors (targets) called fibroblast growth factor receptor (FGFR). FGFRs are found in many cancer cells and play a key role in the growth and spread of the cancer cells. By blocking the FGFR, pemigatinib is expected to reduce the growth and spread of the cancer. If licensed, pemigatinib may offer an additional treatment option for patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement who have failed previous therapy.