An FDA advisory committee Thursday voted 9-1 to recommend that the agency approve a stem cell therapy developed by MesoBlast as a treatment for acute graft-versus-host disease (aGVHD) in children. The panel weighed the need for a new way to address the potentially fatal condition against shortcomings of the clinical trial the Australian biotech conducted to evaluate the investigational cell therapy.
Shares in (NASDAQ: MESO), which fell more than 30 percent earlier in the week after the FDA released briefing documents ahead of the committee’s meeting, closed up 51 percent Friday at $17.88 apiece compared to $11.81 at market close Wednesday. (Trading was halted on Thursday.)
The MesoBlast cell therapy, remestemcel-L (Ryoncil), is made from mesenchymal cells taken from healthy donors. The properties of these cells, which don’t prompt an immune reaction, allow them to be used as an “off-the-shelf” treatment without accompanying immunosuppressants that put patients at greater risk for infection, among other side effects.
Some patients with blood cancer are treated with a stem cell transplant, in which cells from a healthy donor are infused into their bloodstream with the intent that those cells will travel to the bone marrow and form new healthy blood cells. Frequently, however, when donors are unrelated, their cells identify the recipient’s as foreign, prompting them to attack