Ryoncil: ODAC/FDA Meeting Discussion, page-1002

Dr Bindu George

First Speaker Afternoon Session

This session will “focus on the clinical aspects of the BLA for the treatment of pediatric patients”

“The FDA generally agrees with the applicants conclusion regarding the safety of remestemcel-L”

“Our presentation will focus on the products efficacy.”

“As discussed this morning the mechanism of action is unclear and it has been difficult to identify and characteristics that correlates with the efficacy outcome in GVHD.”

“Will rely heavily on the chemical trials to provide evidence of efficacy.”

“The efficacy data comes from GVHD study end point day 28 overall response rate.

The results from the study was pretty significant.

Dr George raised many issues with the studies.

Outlined that there were two randomized controlled trials, with data coming from trials which rolled pediatric and adult patient data together and has negative implications for results in examining efficacy for treating pediatric patients.

“We are concerned about the limitations of the study particularly the challenges that may stem from bias”