FDA Issues Complete Response Letter for Remestemcel-L to Treat Pediatric GVHD

Mesoblast Limited announced that the FDA issued a complete response letter regarding its biologics license application for remestemcel-L to treat pediatric patients with SR-aGVHD after the ODAC voted 9:1 in favor of the available efficacy and data.

The FDA issued a complete response letter to Mesoblast Limited regarding its biologics license application (BLA) for remestemcel-L to treat pediatric steroid-refractory acute graft versus host disease (SR-aGVHD), according to a press release from Mesoblast Limited.

Even though the agency’s Oncologic Drugs Advisory Committee (ODAC) voted 9:1 in support of the available data on the efficacy of remestemcel-L in pediatric patients with SR-aGVHD, the FDA preferred at least 1 more additional randomized, controlled study to further prove the effectiveness of remestemcel-L for SR-aGVHD.

“The phase 3 trial results showed that remestemcel-L provides a meaningful treatment for children with SR-aGVHD who have a very dismal prognosis,” Joanne Kurtzberg, MD, director for the Pediatric Blood and Marrow Transplant Program and co-director at the Stem Cell Transplant Laboratory Duke University Medical Center, said in a press release. “I look forward to having this much-needed therapy available to our patients.”

As for the study voted in favor for by the ODAC, patients received twice weekly intravenous (IV) infusions of 2 x 106 cells/kg over 4 weeks. The data regarding the safety and efficacy of remestemcel-L was collected from 3 different clinical trials, with remestemcel-L being well tolerated in each of the studies and no identified safety concerns.

More, the study data evaluated overall response rate (ORR) and found that survival through 100 days was significantly greater in patients who experienced a response by day 28 (82%) compared with those who did not (39%), with a 100-day overall survival of 67%. ORR by day 28 was the study’s primary end point.

Mesoblast Limited is currently conducting a randomized, controlled phase 3 trial examining up to 300 ventilator-dependent adults with moderate to severe acute respiratory distress syndrome (ARDS) due to the coronavirus disease 2019 (COVID-19). The inflammatory disease, COVID-19 ARDS, has a similar profile to that of SR-aGVHD.

Currently, there are no approved treatments for this disease in children under the age of 12, therefore Mesoblast expects to request a Type A meeting with the FDA to discuss the potential for accelerated approval with a post-approval study condition.

“We are working tirelessly to bring remestemcel-L to patients with life threatening inflammatory conditions, including SR-aGVHD and COVID-19 ARDS,” Mesoblast chief executive Silviu Itescu, said in the release.


MESOBLAST RECEIVES COMPLETE RESPONSE LETTER FROM THE FDA FOR BIOLOGICS LICENSE APPLICATION FOR STEROID-REFRACTORY ACUTE GRAFT VERSUS HOST DISEASE IN CHILDREN [news release]. Melbourne, Australia and New York, New York. Published October 1, 2020. http://investorsmedia.mesoblast.com/static-files/1e259fcb-77ba-470c-91af-1c71e5fa32e0. Accessed October 2, 2020.