ASX Health Stocks: Mesoblast rockets 20pc after being granted Rare Pediatric Disease Designation by FDA
Health & Biotech
Health & Biotech
Mesoblast (ASX:MSB) jumped as high as 25% this morning after announcing that the US FDA has granted its allogeneic cell therapy Revascor (rexlemestrocel-L) a Rare Pediatric Disease (RPD) Designation.
An RPD Designation is granted by the FDA for certain serious or life-threatening diseases which primarily affect children.
The decision follows the submission of results from the randomised controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life threatening congenital heart condition.
Earlier, results from the REVASCOR trial conducted in the US in children with HLHS were published in the December 2023 issue of the peer-reviewed, “The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open).”
In that trial conducted in 19 children, a single intramyocardial administration of REVASCOR resulted in the desired outcome.
This included significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months, compared with controls.
As noted earlier by Mesoblast:
“The fact that 100% of REVASCOR-treated children compared with 57% of controls had large enough LVs to accommodate the full BiV conversion suggests that REVASCOR treatment may help increase the ability to ‘better grow’ the HLHS LV after LV recruitment surgery.”
Mesoblast chief executive Silviu Itescu added today:
“Given the impressive enlargement of the left chamber we have seen in these children treated with REVASCOR in the randomised controlled trial and the increased ability to successfully accomplish life-saving surgery, we plan to meet with FDA to discuss the potential for this trial to support accelerated approval in this indication.”
Upon approval of a Biologics Licensing Application (BLA) for REVASCOR, Mesoblast says it may be eligible to receive an FDA Priority Review Voucher (PRV) that can be redeemed for any subsequent marketing application, or may be sold or transferred to a third party.
Visioneering Technologies (ASX:VTI) announced the full one-year results of its ongoing multi-center, double-masked, randomised controlled trial (RCT).
The trial assessed the safety and effectiveness of VTI’s NaturalVue Multifocal Contact (NVMC) lenses for myopia progression control in children.
The one-year data shows that 45% of subjects in the test group had no myopia progression at all, and nearly two-thirds of subjects in the test group, or 64%, experienced no meaningful progression (defined as 0.25D or less per year).
Further, 71% (or 0.41D reduction) in refractive error progression versus the control group was observed.
Children wearing NVMC lenses showed an average refractive error change of 61% (0.17mm) average reduction in axial elongation, versus the control group.
The average axial length change in children wearing NaturalVue Multifocal Contact lenses was 0.11mm.
VTI says the PROTECT study is ongoing and data will continue to be reviewed, analysed, and shared as available.