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- Mesoblast (ASX:MSB) has been designated as a provider of a medicine for a rare children’s disease by the FDA
- That designation could afford MSB priority status within the FDA’s regulatory ecosystem with a view towards sped-up reviews
- This could ultimately mean a fast track towards commercialisation and revenue
- The drug in question treats a congenial condition where the heart’s left valve fails to form properly
- Shares are up 20.8 per cent in the first hour of trade to 32 cents
Dual NASDAQ and ASX-listed healthcare player Mesoblast (ASX:MSB) has received a designation from the US FDA as a provider of medicine that treats Rare Paediatric Disease (RPD).
Shares rocketed 20.76 per cent to 32 cents in the first hour of trade this morning.
Mesoblast’s products are all “derived from bone marrow” – in other words, stem cells. A key product line was rejected by the FDA in July of 2023, leading to massive losses in Mesoblast’s share price. It is yet to recover.
Regarding today’s news, according to the US regulator, RPD designation awards priority status within the bureaucratic ecosystem, particularly with a view to product and research reviews.
What drug does Mesoblast make?
The product in question that’s got the regulator’s attention is Revascor (Rexlemestrocel-L), a drug that can treat children with a specific heart disease.
That particular disease is called hypoplastic left heart syndrome (HLHS), which can be life-threatening.
HLHS ultimately sees the heart fail to form morphologically correct in the womb and so children are born with an inability to flow blood through the heart properly.
While unusual – thus the RPD status – the results of a randomised trial showed that Revascor is a meaningful solution to the disease.
Management comment
In a study with 19 participants Revascor administered in addition to heart surgery showed that patients receiving Revsacor showed better surgery outcomes. Mesoblast specialises in anti-inflammatory applications.
“Given the impressive enlargement of the left chamber we have seen in these children treated with REVASCOR … we plan to meet with FDA to discuss the potential for this trial to support accelerated approval in this indication,” MSB chief Silviu Itescu said.
In Itescu’s words, Revascor offers “an increased ability to successfully accomplish life-saving surgery.”
Shares were up 20.8 per cent in the first hour of trade to 32 cents.
