MSB Trading 2021 - paradigm shift, page-799

https://www.bioworld.com/articles/496720-mesoblasts-blazes-adcom-trail-for-msc-therapy


In opening the meeting, Wilson Bryan, director of the Office of Tissues and Advanced Therapies at the FDA’s Center for Biologics Evaluation and Research, set the agency’s negative tone, saying, “We have substantial concerns regarding this application.” The biggest concerns had to do with uncertainties about the therapy ’s mechanism of action, potential variability in an off-the-shelf cell therapy and the lack of a randomized control trial (RCT).

The article then goes on to support Mesoblast’s application...

While the FDA asked for advice on requiring another study, pediatric specialists presenting for Mesoblast and testifying in the public hearings stressed the urgency of getting the therapy approved to meet the unmet medical need. Incyte Corp.’sJakafi(ruxolitinib) has been approved to treat GVHD in adults, but it can’t be used in young children because of its side effects. So not only is there no approved treatment for children younger than 12, there is no standard of care using an off-label therapy, said Joanne Kurtzberg, director of the pediatric blood and marrow transplant program at Duke University School of Medicine and a principal investigator in Mesoblast’s 001 pediatric study.

Kurtzberg said she has been using remestemcel-L for more than a decade with good results. Because of that experience and the positive results she’s seen with the therapy, Kurtzberg argued against requiring a controlled study, saying she
and other clinicians wouldn’t risk putting their young patients into a control arm when remestemcel-L works so well. She pointed out that without the therapy, the survival rate for children with GVHD who don’t respond to steroids is 35%.

Given Kurtzberg’s remarks, panelist Mark Walters, director of blood and marrow transplantation at the University of California San Francisco, questioned the feasibility of requiring Mesoblast to conduct an RCT. Such a study would be ethical, Walters said, but it wouldn’t be practical because the already established belief in the therapy’s efficacy would create a barrier to getting trial participation.

But Pledge, this is the bit that grates with me...
I did a little search on Dr Wilson Bryan ... he stated back in 2018 that I thought the 9-1 Yes vote in favour of making Remestemcel-L available confirmed safety & efficiency. Sounds to me a case of the current FDA team saying “don’t confuse me with the facts, I have already made up my mind”. But I’ve change my mind since 2018.

IMO, SI deserves a medal for interacting with the FDA in such a civil manner!