Mesoblast hit by FDA rejection, requested to run another trial

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The FDA met with Mesoblast to determine their ability to produce their treatment (Ryoncil) remestemcel-L. While the Phase III trial of this treatment showed positive results, it was not clear if the patient outcomes were directly connected to this treatment.

The FDA responded to Mesoblast regarding their therapy remestemcel-L. The agency wants to see data from an additional clinical trial before any authorization. The agency identified shortcomings in the claims of a briefing document from Mesoblast. The Phase III trial took more than 2 years.

FDA suggested that Mesoblast conduct an additional randomized clinical trial in adults or children with steroid-resistant graft-versus-host disease. Mesoblast may need to refile for approval with additional clinical data. It seems Mesoblast will be forced to allocate more time and money to bring remestemcel-L into clinical use.

Much of the briefing document includes remarks from the FDA: “in the absence of evidence of that relationship, the FDA said the attributes “may not be sufficient to ensure the manufacturing process consistently produces remestemcel-L lots of acceptable quality.” 

Mesoblast articulated in their response letter: “assays measuring the potency of remestemcel-L will continue to be refined to provide further scientific rationale for its use in severe inflammatory diseases with high mortality risk.”

In February 2018, Mesoblast showed important data from a Phase 3 trial in pediatric GVHD.  69% of patients in this study benefited from a response to the treatment after 28 days. Moreover, Japan approved this therapy under the name Temcell, there it is licensed to JCR Pharma. 

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