MSB to dispute FDA finding in Type A meeting, page-161

Interesting interview SI gave to Tamra Saml Bioworld today. I'll include some snippets, but please click on the article to read to support the author.

https://www.bioworld.com/articles/498477-mesoblast-stock-tanks-on-news-of-fda-crl-for-ryoncil-bla-in-pediatric-gvhd

In its CRL, the FDA recommended that Mesoblast conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of Ryoncil for SR-aGVHD.“We’re going to have a meeting with the FDA in the next 30 days to discuss an accelerated approval, which is a conditional pathway, and we will agree to do another trial but after the product gets approved,”

Mesoblast CEO Silviu Itescu told BioWorld.That trial would be conducted in adults, which would allow the product to be available to children under 12 who have no treatment options, the CEO said.“The good news about the CRL is that most CRLs are related to safety and often efficacy. Here, efficacy was voted on by the ODAC panel, and the FDA has acknowledged no safety concerns,” Itescu said.

He said he believes the FDA wants to see a confirmatory study in a randomized population, but he remains unflappable that the product will be approved.“The ODAC panel voted 9:1. Of course, it’s going to happen. It’s just a matter of navigating the bureaucracy,” he said. “We expect to have a discussion around accelerated approval on the basis of existing data with commitment for a post-approval randomized controlled trial in adults predominantly that continues to support the efficacy evidence of the product in this high-risk patient population as was discussed and voted on by the ODAC panel,”

Itescu said in a same-day conference call with analysts.“In the ODAC panel meeting, we initiated a discussion about what a post-approval trial would look like in adults with steroid refractory graft-vs.-host disease. We have a protocol design that is well underway comparing high-risk adults against standard of care. The randomized controlled trial is well powered for outcome measurements we are proposing to the FDA.”