Mesoblast rallies as it submits new information to FDA for Remestemcel-L approval

The biotech hopes to reward shareholders as its submits fresh information in its long-running bid to receive approval for its aGVHD drug after it was rejected in 2020.

Mesoblast CEO Silviu Itescu said approval could comes as soon as early next year. Picture: Stuart McEvoy.

Mesoblast shares rose almost 8 per cent after the company submitted new information to the US healthcare regulator as part of its long-running bid for the approval of one of its marquee treatments.

The stem-cell focused biotech hopes that approval from the Food and Drug Administration could come as soon as the first quarter of 2023 after its Remestemcel-L therapy was granted a fast track designation in 2020.

In its submission, Mesoblast said there was an urgent need for approval of its drug which treated children with steroid-refractory acute graft versus host disease, with a 90 per cent mortality rate.

“The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes in children,” the company said.

The company noted that survival outcomes of aGVHD had not improved over the past two decades for children or adults with the most severe disease.

Mesoblast chief executive Silviu Itescu said that the company’s new submission provided further evidence that Remestemcel-L had the ability to save lives.

“Additionally, the improved process controls we have put in place to assure robust and consistent commercial product, together with a potency assay that predicts consistent survival outcomes, makes Remestemcel-L a compelling treatment for these children,” Dr Itescu said.

Mesoblast shares rose 7.7 per cent on Monday to close up 6c at 84c. They have slid 40 per cent since the start of the year. It is down 84 per cent since it hit a record high of $5.22 in August 2020 after the ODAC voted.

Dr Itescu last month toldThe Australian that the stem-cell focused biotech had “regrouped” after its application for approval of the drug was rejected by the FDA in October 2020.

The rejection had stunned Mesoblast and shareholders after the US Oncologic Drugs Advisory Committee voted nine to one in favour of the Remestemcel-L therapy two months prior to the decision. The FDA later recommended that Mesoblast conduct at least one additional randomised, controlled study in adults and/or children to provide further evidence of the effectiveness of Remestemcel-L for aGVHD.

Since that rejection two years ago, Mesoblast said it had maintained an active dialogue with the FDA since it received the complete response letter.

“The substantial new information submitted to the Investigational New Drug file for Remestemcel-L in the treatment of children with SR-aGVHD, as guided by FDA, represents a major milestone in the Company’s complete response to the FDA,” Dr Itescu said.

Mesoblast hopes that any approval for its latest application will reward investors who have weathered the share price volatility, which saw the company removed from the ASX 200 index earlier in the year.