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TiGenix’s Crohn's fistulas treatment wins orphan drug status

FDA designation follows positive trial results

TiGenixBelgian biopharmaceutical group TiGenix is one step closer to getting its first-in-class Crohn’s fistulas disease treatment to the US market following an orphan drug designation from the Food and Drug Administration (FDA).

The drug - currently known as Cx601 - could potentially treat ‘complex’ perianal fistulas in patients with Crohn’s disease who have had an inadequate response to at least one conventional or biologic therapy.

Dr Maria Pascual, vice president regulatory affairs and corporate quality, TiGenix, said: “The granting of orphan drug status by the FDA is a significant step forward in the Cx601 development programme.”

The designation follows results of the phase III ADMIRE-CD II trial, a randomised, double-blind, placebo-controlled study designed to confirm the efficacy and safety of Cx601 - a suspension of allogeneic adipose-derived stem cells.

It was found that a single dose of Cx601 was significantly more likely to heal perianal fistulas in Crohn’s patients and stop them coming back for at least a year compared to placebo.

Pascual added: “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.”

The move follows the FDA’s plan to eradicate its orphan designation backlog within 90 days, which was announced by commissioner Scott Gottlieb back in July this year.

Meanwhile, TiGenix and partner Takeda has said it will explore expedited pathways to accelerate the submission and review process for US regulatory approval of Cx601.

Article by
Gemma Jones

24th October 2017

From: Regulatory



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