The U.S. Food and Drug Administration (FDA) has granted TiGenix orphan drug designation for its lead candidate Cx601, which aims to treat perianal fistulas caused by Crohn’s disease in patients who have not improved with conventional treatments.
About 10 to 15 percent of Crohn’s patients develop such fistulas. These are small abnormally formed channels that connect the area close to the anus and the outside skin area, causing constant, throbbing pain.
Current treatments for this secondary manifestation of Crohn’s include antibiotics, immunosuppressants, anti-TNFs and surgery. However, none produce long-lasting benefits, and all have potentially severe complications and safety concerns.
Cx601 is based on adipose-derived stem cells collected from a donor and previously expanded in a laboratory.
Results of the European Phase 3 ADMIRE-CD clinical trial (NCT01541579) showed that a single dose of Cx601 could induce a long-term, sustained healing effect of complex perianal fistulas in Crohn’s patients. These benefits were further confirmed upon completion of 52 weeks and 104 weeks follow-up extension of the trial.
Cx601 has received similar orphan drug designation by Swiss regulators as well as the European Commission. The positive ADMIRE-CD data has supported a Marketing Authorization Application to the European Medicines Agency (EMA), with expected initial evaluation by year’s end.
TiGenix is now conducting a global pivotal Phase 3 clinical trial (NCT03279081) likely to yield additional clinical data to support the filling of a Biologics License Application (BLA) with the FDA for Cx601. The Belgian pharmaceutical company is also exploring other regulatory pathways to expedite the development and U.S. regulatory approval of its new therapy.
“The granting of orphan drug status by the FDA is a significant step forward in the Cx601 development program” Dr. María Pascual, vice president of regulatory affairs and corporate quality at TiGenix, said in a press release. “The FDA’s recognition of Cx601 as an orphan drug brings a number of potential financial benefits and is aligned with our ongoing work seeking expedited pathways towards product approval in the U.S.”
The ADMIRE-CD II trial is a randomized, placebo-controlled study that aims to enroll 326 Crohn’s patients with perianal fistulas that have not adequately responded to prior lines of treatment.
In July 2016, TiGenix and Japan’s Takeda established a licensing agreement that gave Takeda the exclusive right to develop and commercialize Cx601 outside the United States.