Mesoblast Limited (NASDAQ:MESO) on Monday provided an update on the use of Ryoncil (remestemcel-L-rknd) since commercially available in March 2025 for the approved label in children 2 months and older with steroid-refractory acute graft-versus-host disease (SR-aGvHD).
(SR-aGvHD) is a complication following allogeneic hematopoietic cell transplantation, defined by progression after 3 days or no improvement after 7 days of high-dose systemic steroids.
Ryoncil is the first mesenchymal stromal cell (MSC) product approved by the U.S. Food & Drug Administration (FDA) for any indication.
Data
Of the first 25 patients treated with Ryoncil in a ‘real-world’ clinical setting post-launch, 21 were alive (84%) and completed the initial 28-day treatment regimen as per the FDA approval label.
The four patients who did not complete the 28-day treatment course had been offered and failed other therapies before use of Ryoncil and died of severe SR-aGvHD within 28 days.
These early data are consistent with the prior clinical experience with Ryoncil.
The outcomes highlight the company’s focus on getting patients on Ryoncil therapy as early as possible following steroid resistance to enable completion of an initial 28-day treatment course and maximize survival.
To date, 45 transplant centers have been onboarded, with a target of 64 centers, which account for 94% of transplants performed in the U.S. Ryoncil coverage by government and commercial payers already extends to over 260 million U.S. lives with Federal Medicaid coverage by U.S. Centers for Medicare & Medicaid Services (CMS) and mandatory fee-for-service Medicaid coverage in all U.S. states.
Why It Matters?
Issuance on October 1, 2025, of a specific Healthcare Common Procedure Coding System J-Code by CMS for billing and reimbursement resulted in greater usage of Ryoncil under CMS coverage versus commercial coverage in the last quarter.
These commercial activities will continue to serve the company well as it seeks to expand the FDA label for Ryoncil to adults with severe SR-aGvHD, a market size approximately three times that of the pediatric SR-aGvHD population.
A pivotal trial of Ryoncil in adults with severe SR-aGvHD will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network and is expected to commence site enrollment this quarter.
Mesoblast posted $35.1 million in gross revenue from Ryoncil for the quarter ended on December 31, 2025, representing a 60% sequential jump.
Last week, Mesoblast provided feedback received from the U.S. Food & Drug Administration (FDA) on the potential filing of a Biologics License Application (BLA) for its allogeneic cell therapy product rexlemestrocel-L for chronic discogenic low back pain (CLBP).
Comparing outcomes between rexlemestrocel-L and placebo from the MSB-DR003 trial, the FDA acknowledged that the effects on pain intensity appear to favor the active arm.
MESO Price Action: Mesoblast shares were up 1.77% at $19.01 at the time of publication on Tuesday, according to Benzinga Pro data.
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