MaaT Pharma receives negative trend opinion from European Medicines Agency for Xervyteg

Edited by Robert McTiernan··3 min read

MaaT Pharma (Euronext: MAAT), based in Lyon, France, received a “negative trend” opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) regarding its conditional Marketing Authorization Application (MAA) for MaaT013 (Xervyteg), a pooled allogeneic full-ecosystem microbiome restoration therapy intended for the treatment of acute graft-versus-host disease (aGvHD). The opinion was communicated to the company during a CHMP oral explanation session ahead of the committee’s formal vote, expected at the June 2026 meeting.

The CHMP’s negative trend opinion does not constitute a final regulatory decision. A formal vote is pending at the June meeting, and the company has indicated it intends to request a re-examination procedure if that vote is negative. Under EMA rules, the CHMP is required to complete its re-examination within 60 calendar days of receiving an official request. MaaT Pharma attributed the committee’s concerns to challenges the company described as typical for first-in-class therapies assessed under a conditional marketing authorization pathway, particularly those relying on a single-arm pivotal trial as the primary basis for the application. The conditional marketing authorization pathway is designed to allow earlier patient access to medicines addressing unmet medical needs, with the expectation that confirmatory data will be generated post-approval.

About MaaT013

MaaT013, marketed under the proposed brand name Xervyteg, is a full-ecosystem, off-the-shelf microbiome restoration therapy administered by enema. It is derived from pooled, healthy donors and is characterized by high microbial diversity and richness, including a group of bacterial species the company refers to as ButyCore, which are associated with the production of anti-inflammatory metabolites. The therapy is designed to restore the balance of the gut microbiome and modulate immune function, with the goal of reducing steroid-resistant gastrointestinal aGvHD. Unlike small molecule or antibody-based therapies, MaaT013 acts by reconstituting a disrupted microbial ecosystem rather than targeting a single molecular pathway.

Acute graft-versus-host disease occurs within 100 days of allogeneic stem cell or bone marrow transplantation, when donor immune cells attack the recipient’s tissues. Gastrointestinal involvement is associated with severe complications including diarrhea, intestinal bleeding, and elevated mortality risk. First-line treatment relies on systemic corticosteroids, and patients who do not respond are classified as steroid-resistant, representing a population with limited therapeutic options.

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MaaT013 has received orphan drug designation from both the US FDA and the EMA, reflecting the recognized unmet need in aGvHD. The pivotal clinical data supporting the MAA comes from the ARES study, a single-arm trial. MaaT Pharma also cited real-world data from an Early Access Program active in 13 countries, with more than 300 patients treated globally since 2019. Data from the program has been presented at major medical congresses and published in peer-reviewed journals.

Competitive and clinical context

The only agent currently approved for steroid-resistant aGvHD in the EU is ruxolitinib, a JAK1/JAK2 inhibitor marketed by Incyte and Novartis, which received CHMP approval for this indication in March 2022. In the US, ruxolitinib received FDA approval for steroid-refractory aGvHD in 2019. The limited number of approved options in this setting underscores the unmet need MaaT013 is intended to address. The CHMP’s concerns about the single-arm trial design reflect a broader regulatory tension in rare disease development, where randomized controlled trials are difficult to conduct but remain the preferred evidentiary standard for regulators.

MaaT Pharma’s chief executive, Hervé Affagard, said the company remains committed to the EMA process and expressed confidence in the therapy’s potential for registration. The company stated it plans to request a re-examination procedure following a negative formal vote, which would initiate a new independent scientific assessment by a different set of CHMP reviewers within 60 days. MaaT Pharma also disclosed cash management measures intended to extend its financial runway to November 2026, compared to a prior estimate of August 2026, to cover the costs associated with the re-examination process and continued pipeline activities. The outcome of the June CHMP vote and the subsequent re-examination, if pursued, will determine the near-term trajectory of the MaaT013 marketing authorization in Europe.

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