MaaT Pharma, a clinical-stage biotechnology company at the forefront of microbiome-driven therapies, has received a positive opinion from the European Medicines Agency’s Pediatric Committee (PDCO) regarding its Pediatric Investigation Plan (PIP) for MaaT013. This marks a critical regulatory milestone for the company and paves the way for MaaT013’s clinical development in pediatric patients suffering from acute graft-versus-host disease (aGvHD). The approval also positions MaaT013 on track for a Marketing Authorization Application (MAA) submission to the EMA in June 2025.
MaaT013, a Microbiome Ecosystem Therapy™ (MET), is designed to restore the gut microbiome in pediatric patients aged 6 to 18 years with steroid-resistant aGvHD. The approval of the PIP allows MaaT Pharma to proceed with a single-arm Phase 2 trial expected to begin in 2026, evaluating the safety and efficacy of MaaT013 in third-line treatment for 18 pediatric patients.
This milestone underscores MaaT Pharma’s commitment to improving treatment options for pediatric patients who currently have limited therapeutic alternatives for this life-threatening condition.
A pioneering step in pediatric medicine: the first microbiome-driven therapy for aGvHD
Acute Graft-versus-Host Disease (aGvHD) occurs when transplanted stem cells or bone marrow attack the recipient’s tissues, causing severe inflammation, particularly in the skin, liver, and gastrointestinal (GI) tract. GI involvement, which can lead to profound diarrhea, abdominal pain, intestinal bleeding, and sepsis, is particularly deadly and increases mortality risk.
The current treatments for steroid-resistant aGvHD (SR-aGvHD) are limited, with ruxolitinib being the most common second-line therapy. MaaT013 offers a novel approach, leveraging the gut microbiome to modulate the immune system and potentially reduce inflammation, a breakthrough particularly crucial for pediatric patients who have limited treatment options.
With this approval, MaaT013 could become the first microbiome-based therapy to gain market authorization in Europe, making it a landmark development in the field of microbiome-driven treatments for cancer and immune disorders.
Commitment to pediatric patients: early access success and clinical development
MaaT Pharma’s efforts to address unmet pediatric needs are already underway, as the company has successfully treated two pediatric patients with MaaT013 through its Early Access Program, providing safe and effective treatment for children with aGvHD before regulatory approval. These early experiences underscore the therapeutic potential of MaaT013 and the company’s commitment to delivering life-saving treatments to pediatric patients suffering from severe aGvHD.
“We are thrilled with the EMA Pediatric Committee’s approval of our PIP,” said Gianfranco Pittari, MD, PhD, Chief Medical Officer at MaaT Pharma. “The pediatric aGvHD patient population has limited options, and we are proud to be at the forefront of developing a microbiome-based solution that could improve survival rates and quality of life for these vulnerable patients.”
Paving the way for market authorization: a dual regulatory path
Based on the approval of the PIP, MaaT Pharma is now on track to submit its Marketing Authorization dossier for MaaT013 in June 2025. If approved, the therapy could reach the European market as early as late 2026, offering a new hope for pediatric patients with aGvHD.
MaaT013’s successful development would also make it eligible for up to two additional years of marketing exclusivity in Europe, on top of the ten-year market exclusivity granted for orphan drugs. This would provide MaaT Pharma with a robust pathway to commercial success and enable the company to expand access to its innovative microbiome therapy across a broader patient population.
MaaT013: a microbiome ecosystem therapy™ for aGvHD
MaaT013 is a standardized, pooled-donor microbiome therapy that aims to restore a healthy gut microbiome ecosystem in patients with aGvHD. It is administered as an enema and is designed to harness the anti-inflammatory metabolites produced by a diverse range of bacteria, including Butycore™, a specific group of bacterial species known to exert beneficial effects on immune modulation.
The therapy is designed to address GI-related aGvHD, which remains a significant challenge in clinical practice. By leveraging the power of the gut microbiome to regulate immune responses, MaaT013 represents a novel, non-steroidal approach that could dramatically change the treatment landscape for both pediatric and adult aGvHD patients.
A vision for the future: microbiome-driven immunotherapies in oncology
MaaT Pharma is leading the charge in the field of microbiome-driven immunomodulators, especially in oncology, where the gut microbiome plays a crucial role in modulating immune responses. The company’s Microbiome Ecosystem Therapies™ are designed to enhance immune function, improve patient outcomes, and extend survival in cancer patients. The company is advancing therapies in both oncology and immune-related disorders, with the aim of improving survival for patients with cancer through innovative approaches to immune modulation.
“We are poised to lead the way in the development of microbiome-based therapies, with MaaT013 setting the stage for a new era in immune system modulation for pediatric and adult patients alike,” said Hervé Affagard, CEO and Co-Founder of MaaT Pharma. “Our commitment to pediatric care is paramount, and we are excited to bring life-changing therapies to patients in need.”
Looking ahead: MaaT Pharma’s regulatory and clinical roadmap
With Phase 2 trials set to begin in 2026, MaaT Pharma’s approach to microbiome-driven immune modulation will likely become a standard of care for a variety of immune-related conditions. The company is rapidly advancing its clinical pipeline, and the approval of MaaT013’s Pediatric Investigation Plan is just the beginning.
As MaaT Pharma moves closer to its goal of bringing MaaT013 to market, the company remains committed to innovative treatments that harness the power of the microbiome to transform the future of immune therapies and improve the lives of pediatric and adult patients worldwide.
