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On August 2015, TiGenix received the FDA endorsement of a Special Protocol Assessment (SPA) for a pivotal Phase III study for Biologics License Application (BLA) in the U.S. Since the granting of the SPA, further confirmatory positive results have been obtained from the European Phase III study of Cx601 (ADMIRE-CD), the largest randomized study performed so far in Crohn’s disease patients with complex perianal fistulas. Subsequently, TiGenix submitted a Marketing Authorization Application to the European Medicines Agency in March 2016. Following the publication of this additional data, TiGenix submitted a series of technical improvements to be considered by the FDA regarding the previously endorsed protocol.
Based on the minutes from a meeting with the FDA on January 19, 2017, the Agency has agreed to an improved protocol for the global Phase III trial of Cx601. With these current amendments, the FDA has agreed that the BLA could be filed based on the efficacy and safety follow-up of patients assessed at week 24, instead of week 52. Furthermore, the FDA has agreed to accept fewer patients than originally planned in the study, and has endorsed a broader target population that will ultimately facilitate the recruitment process. With these adjustments, the study will benefit from an expedited recruitment process that should lead to shorter timelines, an earlier filing, and the possibility of an earlier approval in the U.S. As a result of these modifications, the trial design is even more similar to the European ADMIRE-CD than it was before.
“We are pleased to have had the opportunity to meet with the FDA and receive the Agency endorsement for our proposals for this improved protocol,” said Dr. Maria Pascual, VP Regulatory Affairs & Corporate Quality of TiGenix. “We continue to be focused and committed to bringing Cx601 to U.S. patients as soon as possible. We believe that this revised protocol will allow us to file for approval one year earlier than we had originally planned,” concluded Dr. Pascual.
The global pivotal Phase III trial for the U.S. registration of Cx601 is expected to begin in the first half of 2017. In parallel, TiGenix is exploring further expedited pathways to accelerate the submission and review process for its future BLA.
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