Mesoblast (NASDAQ:MESO, ASX:MSB) received a complete response letter to its biologics license application resubmission for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD) and requires more data to support marketing approval.
To obtain the data required, Mesoblast will conduct a targeted, controlled study in the highest-risk adults with the greatest mortality. This adult study is in line with the company’s overall commercial strategy, which envisioned a sequenced progression from pediatric to adult SR-aGVHD indications. Adults comprise 80% of the SR-aGVHD market.
“We remain steadfast in making remestemcel-L available to both children and adults suffering from this devastating disease, and have received substantial clarity in how to bring this much-needed product to these patients,” Mesoblast CEO, Silviu Itescu, said in a statement.
The company will seek alignment with the FDA on the trial design for the adult study at a Type A meeting within 45 days.
Prior to the resubmission, the FDA guided Mesoblast to resolve outstanding chemistry, manufacturing and controls issues before initiating any additional clinical trial.
In morning trading on Aug. 4, shares of Mesoblast were quoted at $1.52, down $2.47. or 62%.
