France-based MaaT Pharma (Euronext: MAAT) received a second negative trend vote from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) for MaaT013 (Xervyteg), a pooled-donor microbiome enema therapy for steroid- and ruxolitinib-refractory gastrointestinal acute graft-versus-host disease (aGvHD), following an Oral Explanation meeting on September 14, 2026. A formal CHMP opinion is expected on September 18, 2026.
The CHMP maintained its position that the available clinical data package does not allow sufficient characterization of MaaT013's benefit-risk profile, with concerns centering primarily on the absence of a randomized controlled trial. The committee's objection is unchanged from its initial June 2026 negative opinion: that the single-arm, open-label ARES trial design, combined with the use of concomitant therapies to manage aGvHD, prevents adequate attribution of the observed clinical effects to MaaT013 alone.
The ARES trial enrolled 66 adults with severe gastrointestinal aGvHD refractory to corticosteroids and ruxolitinib across 50 European sites. The study met its primary endpoint, reporting a gastrointestinal overall response rate (GI-ORR) of 62% at Day 28 and a 54% one-year overall survival rate. MaaT Pharma submitted its marketing authorization application (MAA) to the EMA in June 2025 under the conditional marketing authorization pathway, which enables approvals with less-complete data where unmet need and benefit-risk justify it, subject to post-authorization obligations. The company also included real-world data from more than 300 patients treated under an Early Access Program (EAP) active in 13 countries since 2019, as well as the CHRONOS retrospective study as contextual benchmarking evidence.