Mesoblast Limited (ASX: MSB) – A Two-Pronged Regulatory Advance in Cellular Therapies.
- Noel Ong
- 5 days ago
- 5 min read
Updated: 3 days ago
Announcement
Mesoblast Limited (ASX:MSB; Nasdaq:MESO), the recognised global developer of allogeneic cellular medicines, has released an important update that reflects both regulatory momentum and commercial progress across its core programs in inflammatory diseases.
The company has reaffirmed its strategic positioning with the U.S. Food and Drug Administration (FDA), achieving key milestones across two flagship products—Revascor® and Ryoncil®.
FDA Alignment on Accelerated Pathway for Revascor®
In early June, Mesoblast engaged the FDA in a Type B meeting under the Regenerative Medicines Advanced Therapy (RMAT) designation for Revascor® (rexlemestrocel-L). This discussion centred on progressing toward a potential Biologics License Application (BLA) for Revascor in patients with ischemic heart failure with reduced ejection fraction (HFrEF) and inflammation.
There was general alignment with FDA on critical filing elements, including:
Chemistry, manufacturing & controls (CMC).
Potency assays required for commercial product release.
Confirmatory trial design and primary endpoints post-approval.
Mesoblast is now awaiting formal minutes from the FDA meeting to determine its next steps and timeline for potential BLA submission.
Ryoncil®: Expansion into Adult GvHD Market Underway
Ryoncil® (remestemcel-L-rknd), already FDA-approved for paediatric use in steroid-refractory acute graft versus host disease (SR-aGvHD), is now progressing toward label extension into the adult patient population. A pivotal clinical trial is planned in collaboration with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN), with meetings scheduled for early July.
This advancement, if successful, would make Ryoncil® the only FDA-approved mesenchymal stromal cell (MSC) therapy for both paediatric and adult SR-aGvHD indications.
Commercial Uptake and Coverage Milestones for Ryoncil®
Since its commercial availability on 28 March 2025, Ryoncil® has exceeded internal expectations across all key uptake metrics:
Over 20 transplant centres are expected to be onboarded by quarter-end.
The therapy is now covered for over 220 million insured lives across the U.S.
37 of 51 states already provide Medicaid coverage via orphan drug lists or medical exception pathways.
By 1 July 2025, Ryoncil® will be covered across all 24 million remaining Medicaid lives, ensuring nationwide reimbursement access.
Chief Executive Dr. Silviu Itescu commented,
“We are very pleased with the momentum of interactions with FDA on both our cardiac and GvHD programs. We are also encouraged by the strength of the Ryoncil® commercial launch, the rate of hospital onboarding, physician adoption, and payor coverage exceeding our expectations in the ten weeks since commercial launch.”
An update on product sales is expected in the company’s quarterly activities report at the end of next month.
About Mesoblast – Platform, Manufacturing and Global Reach - Cellular Therapies.
Mesoblast continues to lead in developing off-the-shelf cellular therapies based on mesenchymal lineage cells. These products are designed to address severe inflammation by releasing targeted anti-inflammatory signals that regulate multiple immune pathways.
RYONCIL® is the first and only FDA-approved MSC therapy, currently indicated for paediatric SR-aGvHD and under further development for adult GvHD and inflammatory bowel disease.
Revascor® is in advanced development for ischemic heart failure and chronic low back pain.
The company has established strategic partnerships across Japan, Europe, and China.
Mesoblast also maintains a robust global IP portfolio of over 1,000 patents and applications, ensuring commercial protection into at least 2041. Their proprietary manufacturing enables industrial-scale production of cryopreserved, globally deployable, cell-based medicines.
Samso Concluding Comments
What’s worth noting is how Mesoblast has quietly navigated two highly demanding regulatory and commercial landscapes simultaneously. The alignment with the FDA on Revascor® is not a green light, but it is certainly a promising signal. When regulators and companies find common ground on CMC and endpoint design, it’s often a sign of substantial groundwork already in place.
The early uptake and payer support for Ryoncil® speak to both a clear medical need and execution from the Mesoblast team. That 37 states already provide Medicaid support, and full U.S. state-level coverage is expected by July, shows the company’s discipline in working through reimbursement channels—a process that many underestimate in terms of difficulty and time.
While the actual sales figures for Ryoncil® remain to be seen, the groundwork has been laid for commercial traction. If Mesoblast can deliver continued uptake and demonstrate durable outcomes across both adult and paediatric GvHD cases, it may significantly strengthen its standing not only with the FDA but in global therapeutic markets.
For investors and followers of the cellular medicine space, this latest update from Mesoblast may be one to bookmark. As always, the true story will be told in data and uptake—but for now, there’s tangible progress to acknowledge.
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