The Italian Medicines Agency has approved the reimbursement of pemigatinib monotherapy in Italy for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with fusion or rearrangement of the fibroblast growth factor receptor 2 (FGFR2) who have progressed of the disease after at least one previous line of systemic therapy.
Following approval by the European Medicines Agency (EMA) in March 2021, the Italian Medicines Agency approved the reimbursement of monotherapy with pemigatinib in Italy through the Official Journal no. 125 dated 30.05.22 for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with fusion or rearrangement of the fibroblast growth factor receptor 2 (FGFR2) who have shown disease progression after at least one previous line of systemic therapy.
“For a long time we have had no viable options for the treatment of locally advanced or metastatic cholangiocarcinoma; chemotherapy and surgery do not guarantee a lasting response and the overall survival at 5 years for patients with this type of neoplasms is less than 20%: 17% in men and 15% in women “says Giordano Beretta, Director of UOC Medical Oncology ASL Pescara, President of the AIOM Foundation and Past President of AIOM. “In recent years, however, our molecular knowledge has grown: today we know which gene mutations drive the growth of this tumor. In particular, about half of intrahepatic cholangiocarcinomas have at least one target mutation that can be affected by molecularly targeted drugs such as pemigatinib for cholangiocarcinoma with FGFR2 fusions or rearrangements ”.
“The data from the FIGHT study demonstrate the efficacy and safety of pemigatinib, which represents the best and most effective therapeutic option for Italian FGFR2 rearranged cholangiocarcinoma patients with disease not controlled by chemotherapy”, Filippo de Braud points out. Full Professor of Medical Oncology and Director of the School of Medical Oncology at the University of Milan. Director of the Department of Oncology and Hematoncology of the National Cancer Institute of Milan.
“We are proud to be able to make the first treatment option for cholangiocarcinoma available in Europe and for eligible patients in Italy after a decade. Offering a treatment where there was no effective ‘”standard of care’ ‘is a testament to our ongoing commitment to the research and development of new drugs, which fill therapeutic areas where there are high unmet medical needs”, says Onofrio Mastandrea , General Manager Incyte Biosciences Italy.
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Data from the FIGHT-202 study demonstrated that in patients with FGFR2 fusions or rearrangements (Cohort A [108 pazienti]), pemigatinib monotherapy resulted in an overall objective response rate (ORR) of 37% (primary endpoint) and a median duration of response (DoR) of 8 months (secondary endpoint) based on centralized radiographic review of an independent committee. Pemigatinib was generally well tolerated. Pemigatinib warnings and precautions include high and low blood phosphate levels, vision or eye problems, increased blood creatinine and for pregnant women, a risk of fetal harm.
What is cholangiocarcinoma
Cholangiocarcinoma is a rare cancer that forms in the bile ducts. It is classified according to its origin: intrahepatic cholangiocarcinoma (iCCA) originates in the bile ducts inside the liver and extrahepatic cholangiocarcinoma originates in the bile ducts outside the liver. Patients with cholangiocarcinoma are often diagnosed in an advanced or late stage, when the prognosis is poor. In Europe, the incidence of cholangiocarcinoma is between 6,000 and 8,000. FGFR2 fusions or rearrangements occur almost exclusively in the ICCA, where they are seen in 10-16% of patients. In Italy, the CCA affects around 2,000 people every year.
“It is very important to speak and inform correctly about cholangiocarcinoma, because the percentage of patients who arrive at the diagnosis when the disease is at an advanced stage is still high, with negative consequences on the quality of life. Information and education to help patients during their illness and their therapeutic path are fundamental, because it is scientific research that can guide the discovery of new therapeutic options “says Giovanni Brandi, Associate Professor of medical oncology, Director of the school of specialization in medical oncology IRCCS Policlinico Sant’Orsola, University of Bologna, Founder of APIC (Italian Association of Cholangiocarcinoma Patients) and GICO (Italian Cholangiocarcinoma Group).