Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA re­ject­ed Mesoblast’s remestem­cel-L, or Ry­on­cil, its lead can­di­date for pe­di­atric acute graft-ver­sus-host dis­ease (aGVHD) that didn’t re­spond to steroids. The FDA raised a num­ber of con­cerns, first ob­ject­ing to Mesoblast’s sin­gle arm, open-la­bel tri­al, though reg­u­la­tors strug­gled to de­scribe how a ran­dom­ized tri­al would work, since pe­di­a­tri­cians and par­ents were re­luc­tant to put chil­dren with aGVHD in a place­bo arm.

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