Who is eligible?
The approved indication is severe heart failure caused by ischemic cardiomyopathy that remains refractory to standard therapy, including pharmacological and invasive treatment.
How it works
Three sheets of allogeneic iPSC-derived cardiomyocytes are placed sequentially on the heart surface, generally through left thoracotomy. The regimen includes post-procedure immunosuppression and long-term follow-up.
What evidence supported approval?
The pre-approval clinical study included eight patients. At week 26, 2 of 8 had an LVEF improvement of at least five percentage points. MHLW also cited a 10% or greater Peak VO2 increase at week 52 in four patients when determining that efficacy could be inferred. The study was small and uncontrolled, so efficacy is not yet confirmed.
What happens next?
An all-patient post-marketing survey will target 75 patients across 20 institutions and assess Peak VO2, LVESVI, heart-failure hospitalization, survival, six-minute walk distance and LVEF. The conditional approval period is seven years.