The FDA stated that no significant deficiencies were identified in its mid-cycle review of deramiocel. From a regulatory standpoint, how reassuring is this feedback in terms of probability of approval at the August 31 PDUFA date?

The FDA plans to convene an advisory committee prior to the PDUFA action. What are the typical reasons for such a meeting in the context of a first-in-class cell therapy, and what kinds of questions or concerns might the committee focus on?

Cardiomyopathy remains the leading cause of death in DMD, yet treatment options are extremely limited. Where does deramiocel potentially fit in the standard of care, and how might it complement existing skeletal muscle therapies like exon-skipping or gene therapy?

The BLA submission includes comparisons to an FDA-supported natural history database rather than traditional placebo-controlled endpoints. How robust is this strategy for demonstrating efficacy in rare, progressive diseases like DMD?

Deramiocel uses cardiosphere-derived cells (CDCs) to modulate inflammation and fibrosis. How compelling is the mechanistic data supporting CDCs' immunomodulatory and anti-fibrotic effects in the heart, and what distinguishes this approach from gene-targeted therapies?

Given that the BLA excludes HOPE-3 interim data, how persuasive are the cardiac outcomes from HOPE-2 and its extension in suggesting real-world benefit to patients and caregivers?

Capricor highlights the role of CDC-secreted exosomes in deramiocel’s mechanism. How developed is the science behind exosome-mediated signaling in cardiac tissue, and what are the implications for scaling or long-term efficacy?

Deramiocel holds orphan drug, RMAT, and rare pediatric disease designations, potentially leading to priority review vouchers. How do these regulatory pathways influence the approval strategy, pricing expectations, and competitive landscape post-approval?