After sealing imminent inclusion onto the NASDAQ’s biotech index yesterday, Mesoblast (ASX: MSB) shares have again rocketed up the charts, this time on news its groundbreaking Ryoncil stem cell treatment has been approved by the U.S. Food & Drug Administration (FDA).
That approval is going to provide access to some 10,000 patients across the U.S. that, instead of requiring chemotherapy, can use Mesoblast’s mesenchymal stem cell (MSC) therapy to treat steroid-refractory acute graft-versus-host disease (SR-aGvHD).
Crucially, the therapy is approved for children from the age of two months up to teenagers.
More than 15% of those with the condition are under 18.
The company has been around for two decades at the forefront of stem cell research, turning founder and CEO Dr Silvia Itescu into a billionaire and positioning itself as a leader in allogeneic cell medicines to treat complex diseases.
Leading the pack
It is the first cell therapy approved in the U.S. for SR-aGvHD and the first FDA tick for the Aussie biotech business, opening up a portal into the American market for its other offerings, such as Revascor, which treats heart disease and rexlemestrocel, an anti-inflammatory.
Itescu said Ryoncil’s immunomodulatory effects created the potential for the drug to treat other indications in diseases, such as SR-aGvHD in adults and biologic-resistant inflammatory bowel disease.
“With Ryoncil approval by the FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market,” Itescu said.
“We will continue to work closely with FDA to obtain approval of our other late-stage products.”
It is the first time the FDA has approved this type of drug for rollout in the U.S.
“Today’s decision marks an important milestone in the use of innovative cell-based therapies to treat life-threatening diseases with devastating impacts on patients, including children,” FDA Center for Biologics Evaluation and Research Dr Peter Marks said.
“This first mesenchymal stromal cell therapy approval demonstrates the FDA’s commitment to supporting the development of safe and effective products that could improve the quality of life for patients with symptoms that are unresponsive to other therapies.”
Index exposure
On Wednesday, Mesoblast confirmed its addition to the NASDAQ Biotechnology Index, which comes into effect on 23 December.
The move gives the $3.3 billion biotech innovator increased exposure to markets and further credibility with institutional investors.
Shares in Mesoblast are flying today, with an increase of more than 40% in early trade to $2.87, reaffirming it as one of the ASX’s best performing stocks of 2024 with a year-to-date rise of more than 800%.