FDA Shoots Down Mesoblast’s Pediatric GVHD Drug

The FDA has rejected Mesoblast’s biologics license application for its stem-cell treatment Ryoncil in a Complete Response Letter and called on the Australia-based company to conduct another clinical trial.

The unexpected move came after the FDA’s Oncologic Drugs Advisory Committee had recommended that the agency approve the drug as a therapy for pediatric steroid-refractory acute graft vs. host disease, which affects an estimated 50 percent of bone marrow transplant patients.

Mesoblast CEO Silviu Itescue said the agency’s rejection has delayed the drug’s launch by “three to five months.”