Mesoblast Ltd (ASX: MSB) shares have been catching the eye on Thursday.
At one stage, the ASX 300 healthcare stock was up 13% to 31 cents.
Why is this ASX 300 healthcare stock jumping?
Investors have been snapping up the biotechnology company's shares this morning after it released an update on its Revascor product.
Revascor is being trialled as a treatment for children with hypoplastic left heart syndrome (HLHS), which is a potentially life-threatening congenital heart condition.
According to the release, the United States Food and Drug Administration (FDA) has granted the allogeneic cell therapy an Orphan-Drug Designation following the submission of results from a randomised controlled trial.
What does this mean?
Orphan-Drug Designation comes with a number of benefits for the ASX 300 healthcare stock. These include tax credits for qualified clinical trials, the exemption from user fees, and a potential seven years of market exclusivity after approval.
Speaking of trials, in the HLHS trial conducted in 19 children, a single intramyocardial administration of Revascor at the time of staged surgery resulted in the desired outcome.
That outcome was significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared with controls as measured by 3D echocardiography. These changes are indicative of clinically important growth of the small left ventricle, facilitating the ability to have a successful surgical correction.
Mesoblast's Chief Executive, Silviu Itescu, was pleased with the news. He commented:
We are very pleased to have now been granted both Orphan-Drug Designation and Rare Pediatric Disease Designation by FDA for REVASCOR in the treatment of children with this often-fatal congenital heart condition. The designations were granted on the back of the results from children in a randomized controlled trial indicating that REVASCOR may increase the ability to successfully accomplish life-saving surgery. We plan to meet with FDA to discuss the pathway for approval in this indication.
