InnoPharmax Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to gemcitabine for the treatment of cholangiocarcinoma.
“The Orphan Drug Designation represents an important milestone in the development and regulatory strategy for InnoPharmax’s oral formulation of gemcitabine in a new treatment option of cholangiocarcinoma” said Dr. CS Hsu, president of InnoPharmax. "Evidence in animal models suggests that our new oral formulation of gemcitabine has the potential to provide benefit in cholangiocarcinoma patients. We look forward to continuing an upcoming phase II clinical trial.”
About Orphan Drug Designation
Orphan Drug Designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
About Gemcitabine Hydrochloride Oral Formulation
Gemcitaine (2',2'-difluorodeoxycytidine) is a fluorine-substituted deoxycytidine analog with a broad spectrum of antitumor activity. It is rapidly and extensively deaminated by cytidine deaminase to the inactive metabolite, 2',2'-difluorodeoxyuridine (dFdU). It has been reported in a reported clinical study that the systemic exposure to oral gemcitabine was low due to extensive first pass metabolism to dFdU.
A new oral formulation of gemcitabine was developed based on InnoPharmax’s proprietary formulations to overcome the problem of low bioavailability of oral gemcitabine. In dog pharmacokinetic study, gemcitabine was well absorbed from InnoPharmax’s oral formulation of gemcitabine with oral bioavailability over 70%. Preclinical studies have shown that InnoPharmax’s oral formulation of gemcitabine shows efficacy in cholangiocarcinoma, pancreatic as well as other selected tumor models.
About InnoPharmax
InnoPharmax is a specialty pharmaceutical company focused in the development and commercialization of products for the treatment of infectious diseases, immunology and oncology. It develops and acquires potential drug candidates for further development as well as commercialization, and uses internally developed drug delivery technology platforms for improving the safety and effectiveness of currently available drugs to provide better medical choices in these specific disease categories.
