Stem cell treatment producer Mesoblast has recorded a significant win after the US FDA’s Oncologic Drugs Advisory Committee voted in favour of its flagship product for treating children with severe responses to bone marrow transplants.
The planned meeting of the US regulator had sent Mesoblast shares on a rollercoaster this week. The multi-billion company’s share price dropped more than 30 per cent in a single session on Tuesday after briefing papers released ahead of the meeting revealed the watchdog would have serious questions about the company’s research.
The expert panel had been appointed to consider whether Mesoblast’s remestemcel-L product, which is also being tested on COVID-19 patients with severe respiratory distress, was effective in treating acute graft-vs-host disease in children. Graft-versus-host is a severe immune response which can occur after bone marrow transplant.
Over eight hours, the panel heard presentations from the company and experts, then grappled with the question of whether the drug was shown to be effective. The medical experts focused in particular on whether Mesoblast’s study, which was “single arm” and did not use a placebo, was enough to show the treatment was efficacious.