FDA puts pediatric aGVHD drug on priority review lane — will they go virtual with the adcomm?
Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.
With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.
Australian biotech Mesoblast is the developer of the treatment, utilizing allogeneic mesenchymal stem cells as a broad anti-inflammatory agent to tame the cytokines that run amok in patients who quickly develop GvHD following a transplant. In particular, the biotech is aiming for pediatric use in its initial BLA.
“There is nothing that’s approved to treat children with GvHD,” CEO Silviu Itescu told Endpoints News. “So we would be standard of care once approved for all children with GvHD who failed frontline steroids. Everybody will get steroids. And 50% of people fail steroids.”
It’s unclear when or how the FDA, which has limited travel and postponed meetings through April, plans to conduct the adcomm. Speaking with Mark Senak — who runs the Eye on FDA blog — a few days ago, the agency said it would consider leveraging technology to host virtual meetings allowing for remote participation.
Itescu also sees much broader application for the “highly industrialized” process that his biotech has come up with, citing everything from adult GvHD (where he promises to treat sicker patients than Jakafi and improve survival more dramatically) and Crohn’s disease to Covid-19.
Even though Mesoblast has studied its therapy in lung disease before, it was still pleasantly surprising to see a paper out of China suggesting that similar mesenchymal stem cells “could cure or significantly improve the functional outcomes of seven patients without observed adverse effects.”
It makes sense, he said, as acute respiratory distress syndrome (ARDS) is a leading cause of mortality for severe Covid-19 patients. By focusing on the inflammatory immune response caused by the viral infection, they are essentially pursuing a similar strategy as Sanofi and Regeneron with their IL-6 drug Kevzara — though Itescu contends that remestemcel-L should be more effective because it blocks more cytokines.
The theory will be tested in a trial that Mesoblast is launching in Australia, China, the US and Europe.
Currently its cells are manufactured in a facility in Singapore run by Lonza. We’ve asked the company about any site inspection by the FDA and will report back when we know.