MaaT Pharma's Xervyteg Hits Negative CHMP Trend Vote in Re-Examination for aGvHD Approval
核心洞察
MaaT Pharma (搜索) received a negative trend opinion from the EMA's CHMP during a September 14, 2026 Oral Explanation in its re-examination of Xervyteg (搜索) for acute graft-versus-host disease (搜索).
CHMP maintained that the clinical data package, lacking a randomized controlled trial, does not sufficiently characterize the benefit/risk of MaaT013 (Xervyteg (搜索)).
MaaT argued a single-arm pivotal study is appropriate for a first-in-class microbiome therapy, citing 54% one-year overall survival in ARES versus 29% in the CHRONOS external comparator.
MaaT Pharma (搜索) has been informed by the European Medicines Agency (搜索)'s Committee for Medicinal Products for Human Use (搜索) (CHMP) of a "negative trend" opinion following an Oral Explanation held on September 14, 2026, dealing a setback to the company's bid to overturn the committee's earlier refusal of its conditional Marketing Authorization Application (MAA) for MaaT013 (Xervyteg (搜索)®) in acute graft-versus-host disease (搜索) (aGvHD).
The Lyon, France-based clinical-stage biotechnology company said the feedback received after the Oral Explanation indicates that the CHMP maintained its view that the available clinical data package — primarily in the absence of a randomized controlled trial — does not allow sufficient characterization of the benefit/risk profile of MaaT013 (Xervyteg (搜索)®). The remaining concerns relate primarily to the attribution of clinical benefit and safety without randomized controlled trial data.
A formal CHMP opinion is expected on September 18, 2026. MaaT Pharma (搜索) said it will provide additional details following that opinion, assess the implications of the decision, evaluate all available options, and update the market as appropriate.
Regulatory History and the Single-Arm Evidence Dispute
The negative trend follows a re-examination procedure requested by MaaT after the company received a "negative trend" opinion from the CHMP in May 2026 and confirmation of the negative opinion in June 2026. The company sought European approval for Xervyteg (搜索), described as a human allogeneic fecal microbiota therapy, for the treatment of aGvHD.
Because Xervyteg (搜索) is a first-in-class therapy, MaaT has argued that a single-arm pivotal study is appropriate evidence for approval. The CHMP has continued to question whether it is possible to adequately characterize the therapy's risk-benefit profile without data from a randomized controlled trial. According to the company, the disagreement has potential implications for developers of microbiome therapies and other novel modalities that plan to seek EMA approval based on single-arm studies.
Survival Data and External Comparator
MaaT sought to show that Xervyteg (搜索) is more effective than existing treatments for third-line aGvHD by comparing its pivotal data to an external control. In the company's ARES trial, the one-year overall survival rate in patients who received Xervyteg was 54%, compared with 29% in the CHRONOS trial that MaaT used as a comparator.
Company Response
"We are deeply disappointed by this outcome. Patients suffering from gastrointestinal-aGvHD continue to face a life-threatening condition with significant unmet medical need and limited treatment options," said Hervé Affagard, Chief Executive Officer and co-founder of MaaT Pharma (搜索). "Together with the hematology community, we remain committed to these patients and will evaluate all available options to make this potentially important treatment accessible to patients worldwide."
Financial Position
The confirmation of CHMP's continued resistance to approval would intensify pressures on the cash-strapped company. MaaT ended March with €18.1 million in cash and cash equivalents. By cutting costs earlier this year, the company extended its cash runway from August to November to fund operations beyond the CHMP re-examination.
The aGvHD Treatment Landscape
Acute Graft-versus-Host Disease (搜索) occurs in patients within 100 days of undergoing a stem cell or bone marrow transplant, where the transplanted cells initiate an immune response and attack the transplant recipient's organs, causing inflammation of the skin, liver and/or gastrointestinal tract and leading to significant morbidity and mortality. Gastrointestinal involvement is associated with severe complications such as profound diarrhea, abdominal pain, intestinal bleeding, and death. These complications are often life-threatening, with increased mortality risk, due to the challenges of managing severe GI inflammation and the associated risks of infection, malnutrition, and organ failure.
The standard first-line therapy for treating aGvHD is the use of systemic steroids. Patients who do not respond to steroids are considered steroid resistant (SR), and other agents can be administered. Currently the only agent approved for treating SR aGvHD after failure of steroid treatment is ruxolitinib, which is approved for this indication in the USA and received approval from the CHMP on March 25, 2022.
About MaaT013 (Xervyteg®)
MaaT Pharma (搜索)'s Microbiome Ecosystem Therapies (MET) are designed to leverage a full microbiome ecosystem to restore balance and maximize clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases. MaaT013 (Xervyteg (搜索)®) is a full-ecosystem, off-the-shelf, standardized, pooled-donor, enema Microbiome Ecosystem Therapy™ for acute, hospital use. It is characterized by a consistently high diversity and richness of microbial species and the presence of Butycore™, a group of bacterial species known to produce anti-inflammatory metabolites.
Xervyteg (搜索)® (MaaT013) aims to restore the symbiotic relationship between the patient's functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal (GI)-aGvHD. It has been granted Orphan Drug Designation by the US Food and Drug Administration (搜索) (FDA) and the European Medicines Agency (搜索) (EMA).
MaaT Pharma (搜索), founded in 2014 and listed on Euronext Paris (ticker: MAAT) since 2021, describes itself as a late-stage clinical company focused on developing gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival, using proprietary pooling and co-cultivation technologies to develop high-diversity, standardized drug candidates.