MaaT’s bid to overturn microbiome approval refusal suffers setback

Why it matters: Initial feedback from EMA’s Committee for Medicinal Products for Human Use (CHMP) suggests the biotech’s attempt to get the agency to overturn its original decision has failed. Confirmation of CHMP’s continued resistance to approval would intensify pressures on cash-strapped MaaT. 

• 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.

Backstory: MaaT requested European approval for Xervyteg, a human allogeneic fecal microbiota therapy, for the treatment of acute graft-versus-host disease (aGvHD).  

• The biotech received a “negative trend” opinion from CHMP in May. After receiving confirmation of the negative opinion in June, MaaT asked CHMP to re-examine its decision.

Big picture:  Because Xervyteg is a first-in-class therapy, MaaT has argued that a single-arm pivotal study is appropriate evidence for approval. However, CHMP has continued to question whether it is possible to adequately characterize Xervyteg’s risk-benefit profile without data from a randomized controlled trial. 

• 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.

What they’re saying: “Patients suffering from gastrointestinal-aGvHD continue to face a life-threatening condition with significant unmet medical need and limited treatment options,” said MaaT CEO Hervé Affagard in a statement. “We remain committed to these patients and will evaluate all available options to make this potentially important treatment accessible to patients worldwide.”

By the numbers: MaaT sought to show that Xervyteg is more effective than existing treatments for third-line aGvHD by comparing its pivotal data to an external control.

• 54%: The one-year overall survival rate in patients who received Xervyteg in MaaT’s ARES trial. 
• 29%: The one-year overall survival rate in the CHRONOS trial MaaT used as a comparator. 

What’s next: MaaT will assess the implications of the formal CHMP opinion that is expected on Sept. 18. The biotech plans to evaluate all available options and update the market as appropriate.