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.