Intramyocardial Cell Therapy Revascor Gets Orphan Drug Designation

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Revascor (rexlemestrocel-L; Mesoblast), a potential therapy for the prevention of post-implantation mucosal bleeding in end-stage chronic heart failure patients who require a left ventricular assist device (LVAD). 

Revascor is a cell therapy that consists of 150 million allogeneic mesenchymal precursor cells and is delivered as a single intra-myocardial injection. In a phase 2b trial involving 159 patients, treatment with Revascor resulted in a 76% reduction in major GI bleeding events and 65% reduction in hospitalizations in end-stage heart failure patients implanted with an LVAD.

In 2017, the treatment was awarded Regenerative Medicine Advanced Therapy (RMAT) designation, which aims to expedite the development of regenerative medicine therapies. “We look forward to our upcoming meeting with the FDA to discuss a potential approval pathway under the product’s existing Regenerative Medicine Advanced Therapy designation for this life-threatening condition,” said Dr Silviu Itescu, CEO of Mesoblast.

Revascor is also being investigated in a phase 3 trial involving patients with New York Heart Association Class II/III moderate to advanced heart failure. The trial is assessing whether Revascor treatment can reduce recurrent heart failure related major adverse cardiac events in patients with left ventricular dysfunction, and also whether the treatment delays or prevents disease progression to terminal cardiac events (ie, death, LVAD implantation, cardiac transplant).  

For more information visit mesoblast.com.