The US Food and Drug Administration (FDA) has granted orphan drug designation to Belgian biopharmaceutical firm TiGenix’s Cx601 to treat patients with fistulizing Crohn’s disease.
Crohn’s disease is a chronic inflammatory condition characterised by the intestine and complex perianal fistulas.
Formulated for local administration, Cx601 comprises allogeneic (or donor-derived) expanded adipose-derived stem cells (eASCs) for treating complex perianal fistulas in patients who failed standard therapy.
TiGenix Regulatory Affairs and Corporate Quality vice-president Dr María Pascual said: “The granting of orphan drug status by the FDA is a significant step forward in the Cx601 development programme.
“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 US.”
Earlier this year, TiGenix commenced Cx601’s Phase III clinical trial to support a future submission of a US biologics licence application (BLA) to the FDA.
With orphan designation granted by European Commission in 2009, Cx601 is reported to have demonstrated favourable safety and efficacy profile in a Phase III trial completed in August 2015.
A follow-up analysis of the trial further confirmed the sustained safety and efficacy of Cx601.
TiGenix has already submitted a marketing authorisation application to the European Medicines Agency (EMA) and expects a CHMP opinion by the end of this year.
Under a licensing agreement formed with the firm in July last year, Takeda obtained exclusive development and commercialisation rights of Cx601 for complex perianal fistulas outside the US.