Mesoblast says US FDA feedback indicates support for accelerated approval pathway

Australian cell medicine company Mesoblast (ASX:MSB) has announced that the US FDA supported an accelerated approval pathway for the company’s rexlemestrocel-L.

Mesoblast’s rexlemestrocel-L is an allogeneic mesenchymal precursor cell (MPC) product for patients with end-stage ischemic heart failure with reduced ejection fraction (HFrEF) and a left ventricular assist device (LVAD).

The company said the FDA provided the feedback in formal minutes following the Type B meeting it held with the regulator in February for rexlemestrocel-L (REVASCOR) under the existing Regenerative Medicine Advanced Therapy (RMAT) designation.

“We are very pleased with FDA’s feedback that the presented results from our pivotal study of rexlemestrocel-L in end-stage HFrEF patients with LVADs may support an accelerated approval,” said Mesoblast CEO Dr Silviu Itescu.

“We intend to request a pre-Biologics License Application (BLA) meeting to discuss data presentation, timing and FDA expectations for an accelerated approval filing.”

Every year in the US, over 100,000 patients progress to end-stage HFrEF. In these patients, more than 2,500 life-prolonging LVADs are implanted in the US annually, of whom approximately 80 per cent undergo the procedure as a destination or permanent therapy. Most patients receiving LVADs as destination therapy have an ischemic HFrEF etiology.

Compared to patients with non-ischemic HFrEF, patients with ischemic HFrEF have a 76 per cent lower likelihood of LV functional recovery following LVAD implantation and increased mortality over the initial one to two years.

Resistance to functional recovery in ischemic HFrEF patients is thought to be due to excessive inflammation and microvascular insufficiency in the ischemic myocardium. In the placebo-controlled LVAD-MPC Study, 70 patients with end-stage ischemic HFrEF were randomised during LVAD implantation surgery to either a single intervention with rexlemestrocelL or placebo injected directly into the left ventricular myocardium.

Mesoblast said that in its feedback, the FDA indicated that the presented results may support a reasonable likelihood of clinical benefit of MPCs against mortality in LVAD patients, consistent with the criteria for accelerated approval.

/Public Release. View in full here.