After Almost 4 Years, FDA Says Mesoblast Phase 3 Therapy Data Can Support Resubmission For Serious Complications in Children Following Stem Cell Transplant

Monday, the FDA informed Mesoblast Limited MESO that following additional consideration, the available clinical data from its Phase 3 study MSB-GVHD001 appears sufficient to support submission of the proposed Biologics License Application (BLA) for remestemcel-L for pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD).

“We thank the agency for their collaborative approach. The responses and guidance from FDA are clear and provide us with a high level of confidence to refile our BLA for remestemcel-L in children with SR-aGVHD,” said Mesoblast CEO Dr. Silviu Itescu.

Mesoblast intends to file the resubmission during the next quarter, seeking to address all remaining product characterization issues.

In August 2023, the FDA issued a complete response letter to Mesoblast’s resubmission for remestemcel-L for pediatric SR-aGVHD.

The agency required more data, and Mesoblast said it would conduct a targeted, controlled study in the highest-risk adults with the greatest mortality. 

While the Oncologic Drugs Advisory Committee of the FDA in August 2020 voted 9:1 in favor of remestemcel-L’s efficacy in a pediatric patient population, in September 2020, the FDA recommended further steps be undertaken to obtain approval. 

The BLA resubmission of January 2023 included long-term follow-up data from the Phase 3 trial by the Center for International Blood and Marrow Transplant Research, showing 50% survival through more than four years of follow-up for remestemcel-L treated patients.

Earlier this month, the FDA supported an accelerated approval pathway for rexlemestrocel-L for end-stage ischemic heart failure with reduced ejection fraction (HFrEF) and a left ventricular assist device (LVAD). 

Price Action: MESO shares are up 75.90% at $3.87 on the last check Tuesday.