The Mesoblast limited (ASX: MSB) share price has been the best performer on the S&P/ASX 200 Index (ASX: XJO) on Friday.
In morning trade the biotechnology company's shares rocketed as much as 57% higher to a record high of $5.30.
Why is the Mesoblast share price rocketing higher?
Investors have been fighting to get hold of the company's shares after a very successful outcome from its meeting with the Oncologic Drugs Advisory Committee (ODAC) overnight.
That meeting was to discuss its remestemcel-L (RYONCIL) product candidate as a treatment for paediatric steroid-resistance acute graft versus host disease (paediatric SR-aGvHD).
The ODAC is an independent panel of experts that evaluates efficacy and safety of data and makes appropriate recommendations to the United States Food and Drug Administration (FDA).
What happened at the meeting?
This morning the company revealed that the ODAC voted overwhelmingly in favour that the available data supports the efficacy of remestemcel-L in paediatric patients with SR-aGvHD.
Mesoblast's Chief Medical Officer, Dr Fred Grossman, commented: "Steroid-refractory acute graft versus host disease is an area of extreme need, especially in vulnerable children under 12 years old where there is no approved therapy."
"We are very encouraged by today's outcome and are committed to working closely with the FDA as they complete their review of our submission regarding approval of RYONCIL for this life-threatening complication of an allogeneic bone marrow transplant."
What now?
While the FDA will seriously consider the recommendation of the panel, the final decision regarding the approval of the product is made solely by the regulator. This means the recommendations by the panel are non-binding.
The RYONCIL product has been accepted for Priority Review by the FDA, with an action date of 30 September 2020. If approved, Mesoblast plans to launch RYONCIL in the United States in 2020.
Paediatric transplant physician Dr Joanne Kurtzberg appears hopeful this will go to plan. She sees a major need for a treatment for this condition.
Dr Kurtzberg commented: "This devastating condition has an extremely poor prognosis and there are no FDA-approved options for children under the age of 12. The clinical studies I have directed have demonstrated the potential for this treatment to fill a significant unmet medical need."
