It's hard to believe this article was from three months ago—it feels much longer. On re-reading, the stark candour of the quotes stand out even more, especially compared to the subdued tone of the conference call. Tick tock.
Mesoblast tips big year after leaping US FDA hurdleThe drug developer, which has a platform which produces compounds targeting inflammatory disorders, received bad news from the US Food and Drug Administration last August when the regulator said it wanted more data to support marketing approval for the company’s Ryoncil compound.
However, Mesoblast managed to turn that setback around. Prof Itescu said that after the company ran through the data with the FDA in person it was able to win over the regulatory body.
The company will now be able to submit a Biologics License Application for Ryoncil for the treatment of pediatric patients with steroid-refractory acute graft versus host disease (GVHD).
Once that is submitted, the approval process which would enable Mesoblast to start selling the drug was likely to be between two and six months, Prof Itescu said.
The turnaround in the company’s fortunes is reflected in the share price, which over the past 12 months has made a U turn, dropping precipitously as the FDA rejection approached, and recovering in recent times to close at $1.04 on Friday. The range over the past year has been from highs of $1.35 last July to as low as 25.5c.
Prof Itescu said Mesoblast representatives visited the US in September to put their case.
“Nothing beats face-to-face meetings,’’ he said. “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’,’’ Prof 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.’’
Prof Itescu said the data supported the lifesaving potential of the drug, which showed that 50 per cent of children treated were alive five years later, compared with just 20 per cent for other treatments.
Given the manufacturing processes have already been inspected and signed off on, the path to market from here, pending approval, could be rapid, and the company would have two to three years worth of inventory on hand.
The next steps then would be marketing and building relationships with hospitals and insurance companies, and progressing the treatment for adult use.
Prof Itescu said the positive response from the FDA also augured well for the company’s development platform as a whole.
The Revascor compound, for use in patients with end-stage ischaemic heart failure, is also looking good. The FDA has allowed the company to file for accelerated approval in a subset of patients at the more serious end of the disease spectrum.
Prof Itescu said the company would then seek to do a confirmatory study to demonstrate the efficacy of the drug in other patients, and a wider market.
“So, that’s a big growth opportunity for the company, and I think the value proposition there will become much more evident next year when we when we file for accelerated approval,’’ he said.
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