TiGenix NV, an advanced biopharmaceutical company focused on exploiting the anti-inflammatory properties of allogeneic, or donor-derived, stem cells to develop novel therapies for serious medical conditions, today announces that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Cx601 for the treatment of patients with fistulizing Crohns disease.
TiGenix started a global pivotal Phase III clinical trial in the first half of 2017 with the aim of submitting a future U.S. Biologics License Application (BLA) to the FDA for Cx601, a first-in-class allogeneic cell therapy product for the treatment of complex perianal fistulas in patients with Crohn’s disease who have had an inadequate response to at least one conventional or biologic therapy. In parallel, TiGenix is exploring expedited pathways to accelerate the submission and review process for U.S. regulatory approval.
“The granting of orphan drug status by the FDA is a significant step forward in the Cx601 development program” said Dr. María Pascual, Vice President Regulatory Affairs and Corporate Quality at TiGenix. “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 FDA grants orphan status for novel products to treat conditions affecting fewer than 200,000 people in the U.S. Orphan designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including seven years of market exclusivity following marketing approval, tax credits for clinical research costs, eligibility for orphan product grants and the waiver of certain administrative fees.
