By Emma Koehn and Jessica Yun
Stem cell biotech Mesoblast has suffered another setback in its bid to get its flagship product, remestemcel-L, approved in the US, with the US Food and Drug Administration (FDA) again asking for more data from the company before signing off on it.
The FDA’s latest guidance is another blow to Mesoblast’s long-running efforts to launch remestemcel-L to the US market. More than a decade in the making, the product has been developed as a treatment for graft versus host disease, a severe immune reaction which can occur after a bone marrow transplant.
Mesoblast, which has long championed the treatment as a first-of-its-kind stem cell product to fight the disease in children, will now conduct a controlled study of high-risk adults with the greatest mortality to get the data requested by the FDA.
US Food and Drug Administration issued a “complete response letter” to Mesoblast in October 2020, asking for more data on its treatment before it could be approved in the US market. Credit: Al Drago/AP Images
The biotech’s shares crashed on the news, sliding 56.8 per cent to close at 47 cents. The company’s share price touched highs of $5.22 in August 2020, but dipped below the 70 cent mark in 2022.
Mesoblast shareholders have been on a rollercoaster for the past three years, after the market was blindsided by the US regulator’s decision in October 2020 that the company needed to provide additional information about the drug and its manufacturing processes before it could be approved.
An expert review committee had voted nine to one in favour of a green light for the treatment, but the FDA did not follow the committee’s advice and instead said more data was needed.
In March of this year, Mesoblast confirmed that it had re-submitted its application to get the drug approved, and had provided a raft of new information to the regulator. It presented long-term survival data from the children in its original phase 3 trial, and data which compared the outcomes of the children who received Mesoblast’s treatment to others who had been treated differently.
Mesoblast chief executive Silviu Itescu remains optimistic remestemcel-L will be approved by US regulators, highlighting the FDA’s inspection had raised no safety concerns about more than 1300 patients who had received remestemcel-L so far.
Mesoblast chief executive Silviu Itescu is confident the US FDA will approve the biotech’s flagship treatment.Credit: Josh Robenstone
“We remain steadfast in making remestemcel-L available to both children and adults suffering from this devastating disease, and have received substantial clarity in how to bring this much-needed product to these patients,” Itescu said in a statement on Friday.
Itescu holds an 8.3 per cent stake in the company, according to Bloomberg records accessed Wednesday, which was worth $73.8 million on Thursday evening, though the value of his stake slipped to $33.2 million just before midday on Friday as the share price fell.
The stem cell biotech will seek approval from the US FDA on the adult study’s trial design within 45 days.
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