Per your post 77137619 late last year, there is every chance the...

Per your post 77137619 late last year, there is every chance the FDA will green light a label extension for Ryoncil to adults, while requiring a concurrent P3 trial.

“The approach we have now witnessed is likely the new Marks-FDA version of the Japanese Sakigake designation:
1. Is it safe? Yes.
2. Is there an alternative therapy on the market? Yes, but it doesn’t have a strong safety or efficacy profile, and this therapy is being offered after the existing treatment fails.
3. Are there signs that this therapy COULD help these patients? Yes.”

As a third line therapy there is little downside and some upside from approving a label extension. A label extension, a P3 trial, or both are likely to impact Cynata’s ability to recruit patients in the US. Under CYP’s patient requirements, they will not be eligible for therapies outside of steroids and Ruxolitinib. As MSB are working with the BMT CTN, who manage 80% of all bone marrow transfers, they will have make a persuasive argument for patients to enter their trial/receive their product instead of Cynata’s. So that is the risk IMO.

The good news is that:
1. No one knows when either the trial will commence or a label extension will be granted.
2. It would only impact 8 out of 29 sites CYP is recruiting from.