The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to our portfolio company PCI Biotech. This is a major regulatory milestone for the development of the company´s lead product candidate named fimaporfin for the treatment of patients suffering from bile duct cancer.
This patient population has no approved treatment alternatives today and fimaCHEM (fimaporfin) has the potential to play a role in this area of high unmet medical need.
“Receiving orphan status from the FDA is a crucial step in the development of this important new medicine for 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 is a significant regulatory milestone and recognises the therapeutic benefits we seek to bring to the patients. It supports our further development of fimaChem in this indication and provides important development and commercialisation benefits.”
About Orphan Drug Designation
Under the U.S. Orphan Drug Act, the FDA’s Office of Orphan Products Development provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan drug designation conveys up to seven years of marketing exclusivity if the drug receives regulatory approval from the FDA and offers various development incentives, including an exemption from the FDA user fee and FDA assistance in clinical trial design.
The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval and designation does not mean that marketing approval will be granted.
About bile duct cancer
The bile duct drains bile from the liver into the small intestine. Biliary tract sepsis, liver failure and/or malnutrition and cachexia due to locoregional effects of the disease are the most important causes of death.
Currently, surgery is the only curative option for these patients; yet the majority of the tumors are inoperable at presentation. Inoperable patients are treated with stenting to keep the bile duct open and with chemotherapy.
The combination of gemcitabine and cisplatin has shown promising results and has become standard treatment in some regions, but there is still a need for better treatments to increase overall survival and quality of life.
About PCI Biotech
PCI Biotech is a biopharmaceutical company focusing on development and commercialisation of novel therapies for the treatment of cancer through its innovative photochemical internalisation (PCI) technology platform. PCI is applied to three distinct anticancer paradigms: fimaCHEM (enhancement of chemotherapeutics for localised treatment of cancer), fimaVACC (T-cell induction technology for therapeutic vaccination), and fimaNAc (nucleic acid therapeutics delivery).
Photochemical internalisation induces triggered endosomal release that is used to unlock the true potential of a wide array of therapeutic modalities. The company’s lead fimaCHEM programme consists of a clinical Phase I/II clinical study in bile duct cancer, an orphan indication with a high unmet need and without approved products. fimaVACC applies a unique mode of action to enhance the essential cytotoxic effect of therapeutic cancer vaccines, which works in synergy with several other state-of-the-art vaccination technologies. fimaNAc utilises the endosomal release to provide intracellular delivery of nucleic acids, such as mRNA and RNAi therapeutics, thereby addressing one of the major bottlenecks facing this emerging and promising field.