US clears first targeted treatment for cholangiocarcinoma

US regulators have approved Incyte's Pemazyre as the first targeted treatment for cholangiocarcinoma.

The decision allows physicians to prescribe the kinase inhibitor for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

CCA, also known as bile duct cancer, is a rare and aggressive type of cancer that forms in bile ducts. It is classified by anatomic origin into intrahepatic (iCCA) and extrahepatic CCA (eCCA). FGFR2 fusions have been identified as key oncogenic drivers in 10% to 16% of iCCA patients.

Although rare, the incidence of iCCA has been increasing, and patients with the condition have faced significant clinical challenges, including a poor prognosis and limited treatment options, the firm noted.

"Although cholangiocarcinoma is considered a rare disease, it has been on the rise over the past three decades," said Ghassan Abou-Alfa, Memorial Sloan Kettering Cancer Center. "It is encouraging to have a new targeted treatment option for patients who historically have had limited options after first-line chemotherapy or surgery, in which relapse rates remain high."

The drug is also currently under regulatory review in Europe.

COVID-19 trial 

Meanwhile, Incyte also announced the initiation of the first Phase III clinical trial (RUXCOVID) evaluating the efficacy and safety of Jakafi (ruxolitinib) plus standard-of-care in patients 12 years of age or older with COVID-19 associated cytokine storm.

The JAK1/JAK2 inhibitor was discovered by Incyte scientists. Overactive signalling through the JAK-STAT pathway has been associated with many types of cancer, as well as other serious immune-mediated conditions such as GVHD.

Because many patients with severe respiratory disease due to COVID-19 have features consistent with cytokine storm and increased activation of the JAK-STAT pathway, it is thought that the drug may be able to play a role in treating these patients, the firm noted.

The study will enrol around 400 patients globally.
 

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