Zarix Inc announced that the U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation to Thymitaq (nolatrexed dihydrochloride) for the treatment of hepatocellular carcinoma (HCC).
Thymitaq, a thymidylate synthase inhibitor, is a novel investigational oncology agent that is being developed to treat several different cancers. THYMITAQ is currently being studied in a multi-center clinical trial for the treatment of unresectable hepatocellular carcinoma (liver cancer) in the U.S., Canada, Europe and South Africa.
The term "orphan drug" refers to a product that treats a rare disease affecting fewer than 200,000 individuals in the US. The intent of the Orphan Drug Act is to stimulate the research, development and approval of products that treat rare diseases. The designation enables the sponsor to obtain: seven years market exclusivity after approval of the New Drug Application (NDA); probable waiver of fees at the time of NDA submission; tax incentives for clinical research; and possible clinical grants.
In addition to the orphan drug designation, the Thymitaq development program has been designated Fast Track for the treatment of unresectable hepatocellular carcinoma (HCC) by the U.S. Food and Drug Administration. "Fast Track" status is reserved for those drugs in development that display the potential to address unmet medical needs for fatal or life-threatening conditions. Typically under this designation, the FDA plays an active role in advancing the drug's development. The designation may enable the sponsor to obtain priority review of the drug for this condition. Priority review reduces the time for review of a market application significantly.
"Zarix has initiated the pivotal Phase III ETHECC(C) trial ("Evaluation of Thymitaq in hepatocellular carcinoma) and there are currently 49 centers in North America, Europe and South Africa participating in the trial. We expect to complete enrollment by the end of 2002," said Dr. Elizabeth Corsi, Chief Executive Officer and President. "We believe that Thymitaq has the potential to be an important treatment for patients with HCC, who otherwise have very few treatment options today in a disease which is rapidly fatal."
Zarix is a specialty pharmaceutical company focused on the development and marketing of novel cancer drugs. The company licenses advanced oncology, hematology and related drug products and manages their clinical development and registration. Zarix is building a commercial infrastructure to market products in the US. Thymitaq is the company's flagship product, which was licensed from Agouron Pharmaceuticals Inc in late 1998.