Ann: FDA Accepts Mesoblast's BLA Resubmission for Remestemcel-L, page-114

Mesoblast could be selling its flagship drug within months after key FDA milestone
CAMERON ENGLAND
55 minutes ago.
Updated 38 minutes ago

The Australian Business Network

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Mesoblast is hoping to be able to sell Ryoncil within months.
Mesoblast shares soar after the US Food and Drug Administration accepted the company’s resubmission of its biologics license application for its Ryoncil compound for use in children.
The acceptance sets the company up for first sales of the compound by no later than January 7, 2025, with Mesoblast already having two to three years of inventory on hand.
The company suffered a setback in the approval process for the drug last year, when the FDA said it wanted more data to support marketing approval for the compound, which treats steroid-refractory acute graft versus host disease (SR-aGVHD).
Mesoblast managed to turn that setback around, with chief executive Professor Silviu Itescu telling The Australian earlier this year that after the company ran through the data with the FDA in person, it was able to win over the regulatory body.
Mesoblast said on Tuesday its BLA resubmission had been accepted.
“Mesoblast’s resubmission on July 8, 2024 addressed remaining CMC (Chemistry, Manufacturing, and Control) items after being informed by FDA at the end of March 2024 that, following additional consideration, the available clinical data from the Phase 3 study … appears sufficient to support submission of the proposed BLA for remestemcel-L for treatment of pediatric patients with SRaGVHD,’’ the company said.
Dr Itescu said in the statement: “We are pleased that FDA has accepted our BLA resubmission for review, and look forward to the potential approval of Ryoncil for children with SR-aGVHD’’.
Dr Itescu later told The Australian that “thing are very much on track’’, with the company well-positioned to start marketing and distributing Ryoncil once it was full approved for sale.
Mesoblast shares traded as high as $1.32 – a 12-month high – before settling to be trading at $1.30 in afternoon trade, up more than 12 per cent.
The company raised $60.3m at 30c a share in December, with shareholders who participated in that raise now up more than 300 per cent.
Ryoncil counteracts the inflammatory processes that are implicated in SR-aGVHD by inhibiting activation and proliferation of effector T cells.
Trial data has shown that 50 per cent of children treated with the compound were alive five years later, compared with just 20 per cent for other treatments.
The manufacturing processes for the compound have already been inspected and signed off on by the FDA.
Mesoblast will also be looking at getting the compound approved for use in adults.

CAMERON ENGLANDBUSINESS EDITOR