On May 28, 2021 PCI Biotech (OSE: PCIB), a cancer focused biopharmaceutical company reported that the Ministry of Food and Drug Safety (MFDS) in South Korea has granted Orphan Drug Designation to its lead product candidate, fimaporfin, for combination treatment with gemcitabine in patients with inoperable locally advanced or metastatic bile duct cancer (cholangiocarcinoma) (Press release, PCI Biotech, MAY 28, 2021, View Source [SID1234585150]). No approved treatment alternatives exist today for the first-line treatment of patients with this disease and fimaChem (fimaporfin) has the potential to play a role in this area of high unmet medical need.

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"The RELEASE trial has in general shown promise with good recruitment activity in South Korea despite the Covid-19 pandemic. Receiving orphan status from the South Korean authorities is a key step in the development of this important new medicine for Asian cancer patients in need of better local treatments. PCI Biotech’s fimaChem treatment is well suited for treatment of bile duct cancer, with easy light access through routine endoscopic methods." said Dr. Per Walday, CEO of PCI Biotech. "Orphan designation in South Korea, in addition to the already granted orphan designations in EU and USA, are all significant regulatory milestones and recognises the therapeutic benefits we seek to bring to the patients."

About bile duct cancer and the fimaChem technology
The bile duct drains bile from the liver into the small intestine. Bile duct cancer (cholangiocarcinoma) is a cancer that affects the cell lining of the bile duct and represents a patient population with a high unmet medical need. Surgery is the only potential curative option for these patients, but most patients are inoperable at presentation. Inoperable patients are treated with stenting to keep the bile duct open and with chemotherapy. Median survival of inoperable patients is between 11 and 12 months with the current standard of care treatment, which is a chemotherapy combination of gemcitabine and cisplatin. Biliary tract sepsis, liver failure and/or malnutrition and cachexia due to locoregional effects of the disease are the most important causes of death, so there is a high need for better locoregional treatments. The locoregional anti-cancer effect of gemcitabine is significantly enhanced by the fimaChem technology in preclinical studies, and early clinical treatment data suggest encouraging tumour response and survival in bile duct cancer patients.

Bile duct cancer is a rare disease with an incidence rate of 1-2 per 100,000 in the western world. The incidence rates are increasing worldwide and are generally higher in Asian countries. The immediate target for PCI Biotech is first-line treatment of inoperable patients with extrahepatic disease. The fimaChem treatment regimen consists of an intravenous injection of fimaporfin, followed four days later by an intravenous infusion of gemcitabine and a laser light application in the bile duct easily administered through endoscopic methods used routinely in these patients. The patients then follow the standard background treatment with up to 8 chemotherapy cycles of gemcitabine + cisplatin. The fimaChem treatment may be repeated during the background chemotherapy treatment cycles.