Mesoblast Limited, an Australian-based regenerative medicine company, announced that the US FDA issued a Complete Response Letter to its Biologics License Application for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD).
Back in August, the Oncologic Drugs Advisory Committee voted 9:1 that the available data supported the efficacy of the drug, branded as Ryoncil, in pediatric patients with SR-aGVHD. But the FDA recommended that Mesoblast conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of effectiveness.
GVHD can occur after an allogeneic stem cell transplant when the donated cells initiate an immune response and attack the transplant recipient’s organs. The condition, which can be acute or chronic, can affect multiple organ systems including the skin, gastrointestinal tract, and liver. There are currently no approved treatments for the life-threatening condition in children under 12. Last year, the FDA approved Incyte's Jakafi to treat the disease in in adult and pediatric patients 12 years and older.
Due to the lack of pediatric treatments, Mesoblast said the company will urgently request a Type A meeting with the FDA, expected within 30 days, to discuss a potential accelerated approval with a post-approval condition for an additional study.
Read the press release