Mesoblast has received FDA approval for its drug Ryoncil

The company has received US Food and Drug Administration approval for its drug Ryoncil, in a stunning turnaround from a year ago, when the same drug tanked the share price.

Mesoblast CEO Silviu Itescu.

Mesoblast shareholders could enjoy up to tenfold gains for the year after the US Food and Drug Administration approved its drug Ryoncil for use with infants and children.

It is a stunning turnaround for the company, which raised $36m at just 30c per share this time last year after an FDA query for more data on Ryoncil smashed the share price in mid-2023.

Mesoblast chief executive Dr Silviu Itescu spoke to The Australian earlier this year, explaining how the company ran through the data with the FDA in person and managed to turn the situation around.

The company’s representatives travelled to the US in September 2023 to put its case.

“Nothing beats face-to-face meetings,’’ Dr Itescu told The Australian.

“We met with them at FDA headquarters in September, we re-presented our data to a group of the leadership around a table, and presented to them both the clinical data and the manufacturing data, the science behind how the product works. And that was clearly a positive meeting.”

Mesoblast was asked for additional data, and went back to the FDA in January, expecting to be told what more needed to be done on the study front.

“And instead, what they said was ‘no, we’ve thought this through, and you’ve convinced us that the clinical data are very strong’,” Dr Itescu said.

“You’ve got enough to get approved for children – go ahead and file, and then consider how to take the product into adults.” Mesoblast on Thursday announced it had subsequently received FDA approval to sell Ryoncil, which is used to treat steroid-refractory acute graft versus host disease (SR-aGVHD).

“Annually in the US approximately 10,000 patients undergo an allogeneic bone marrow transplant, 1500 of whom are children,” Mesoblast said in a statement to the ASX.

“Approximately 50 per cent develop aGvHD and almost half of those do not respond to steroids, the recognised first-line treatment.”

Mesoblast shares were up 32 per cent at $2.61 in late morning trade on the ASX. That compares to a 12-month low of just 25.5c.

Dr Itescu told The Australian earlier this year that data supported the lifesaving potential of the drug, showing that 50 per cent of children treated were alive five years on, compared with just 20 per cent for other treatments.

He said the FDA had already signed off its manufacturing processes, and Mesoblast already had a two to three year supply of the drug on hand.

Ryoncil counteracts inflammatory processes associated with SR-aGVHD.

Mesoblast will also be looking at getting the compound approved for use in adults.

The company said on Thursday that Ryoncil was the first mesenchymal stromal cell therapy approved in the US for any indication, and the only approved treatment for steroid-refractory acute graft versus host disease (SR-aGVHD) in children two months and older.

“We are very pleased that the FDA has granted approval of Ryoncil and are proud of the company’s commitment to the GVHD community in bringing this important new treatment to children and families with no other acceptable options,” Dr Itescu said.

“With Ryoncil approval by FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market.

“We will continue to work closely with FDA to obtain approval of our other late-stage products, including Revascor for cardiovascular diseases and rexlemestrocel-L for inflammatory pain indications, as well as expanding the indications for Ryoncil in both children and adults with inflammatory conditions.”

Originally published as Mesoblast has received FDA approval for its drug Ryoncil