Mesoblast’s lead drug candidate achieves major regulatory milestone after US FDA vote
Mesoblast aims to launch remestemcel-L on the US market later this year.
Shares in Australian biotech midcap Mesoblast (ASX: MSB) have hit their highest point in more than six years today, following the US Food and Drug Administration’s Oncological Drugs Advisory Committee’s positive vote for remestemcel-L.
The FDA voted nine-to-one in favour of data supporting the efficacy of remestemcel-L in children with steroid-refractory acute graft versus host disease (SR-AGVHD).
It is warm relief for the company, which lost a third of its value this week upon news the FDA wanted to “discuss issues related to the characterisation and critical quality attributes of remestemcel-L”.
Advancing Ryoncil towards commercialisation
Remestemcel-L, to be branded Ryoncil, is Mesoblast’s lead drug candidate and the company has certainly been busy with it this year.
In May, the company revealed it was using fresh capital to enable its US commercial launch, which is planned for later this year.
The $138 million in capital was also channelled into clinical trials using remestemcel-L in critically ill COVID-19 patients who have developed acute respiratory distress syndrome.
Today’s news, however, is squared solely on its use in children with graft versus host disease.
“Steroid-refractory acute graft versus host disease is an area of extreme need, especially in vulnerable children under 12 years old where there is no approved therapy,” Mesoblast’s chief medical officer Dr Fred Grossman told investors.
“We are very encouraged by today’s outcome and are committed to working closely with the FDA as they complete their review of our submission regarding approval of Ryoncil for this life-threatening complication of an allogeneic bone marrow transplant.”
Dr Joanne Kurtzberg, a paediatric transplant physician at Duke University Medical Center, was bullish on remestemcel-L’s potential.
“This devastating condition has an extremely poor prognosis and there are no FDA-approved options for children under the age of 12. The clinical studies I have directed have demonstrated the potential for this treatment to fill a significant unmet medical need,” she said.
The next steps require the FDA to approve the drug under the Prescription Drug User Fee Act by the end of September.
The company is well-funded with $188 million cash on hand, following a major $138 million global capital raise in May.