FDA Grants Belgian Crohn’s Disease Candidate Orphan Designation

TiGenix’s candidate for a nasty Crohn’s disease complication received orphan drug designation after agreeing on a global Phase III trial.

TiGenix exploits the anti-inflammatory properties of donor-derived stem cells to treat diseases. One of its candidates, Cx601, is targeted at a complication of Crohn’s disease – a nasty inflammatory condition affecting the intestine. Cx601 met efficacy and safety endpoints during a European Phase III trial, prompting Japanese pharma giant, Takeda, to snap up its licensing rights for up to €380M. TiGenix will hope to repeat these findings in a larger study across Europe and North America. Having been awarded Orphan Designation by the FDA, the candidate’s journey to the US market should be accelerated.

Cx601 is a cell therapy for the treatment of complex perianal fistulas – a severe, debilitating complication of Crohn’s disease. Current treatment options include antibiotics, anti TNFs and surgery, but these have limited long-term efficacy and considerable safety concerns.

Boehringer Ingelheim is targeting Crohn’s disease – a market expected to be worth up to $4.2B (€3.6B) by 2022 – with its TNFɑ inhibitor, adalimumab biosimilar, Cyltezo, while TxCell is developing a T cell therapy for the condition. Also, keep an eye out for approaches using the microbiome like that of Enterome, which targets the FimH adhesin bacterial gene.

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