Mesoblast and FDA Align on Key Items for Revascor® Biologic License Application in Ischemic Heart Failure

Rhea-AI Summary

Mesoblast (NASDAQ:MESO) has announced a significant regulatory advancement for its Revascor® (rexlemestrocel-L) treatment. The company has achieved alignment with the FDA on key requirements for filing a Biologics License Application (BLA) for treating patients with ischemic heart failure with reduced ejection fraction (HFrEF) and inflammation.

Following a successful Type B meeting under the existing Regenerative Medicine Advanced Therapy (RMAT) designation, where the FDA indicated that presented studies could support accelerated approval, Mesoblast met with the FDA on June 3, 2025. The FDA's formal minutes confirmed alignment on critical aspects including chemistry, manufacturing & controls (CMC), potency assays for commercial product release, and the proposed design and primary endpoint for the post-approval confirmatory trial.

The company plans to file for accelerated approval by the end of 2025, specifically targeting patients with end-stage ischemic HFrEF who have been implanted with a left ventricular assist device (LVAD).

Positive

• FDA alignment achieved on key BLA filing requirements for Revascor®
• Existing RMAT designation supports potential accelerated approval pathway
• FDA confirmed previous studies could support accelerated approval
• Clear regulatory pathway established with planned BLA filing by end of 2025

Negative

• None.

NEW YORK, June 30, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced alignment with the United States Food and Drug Administration (FDA) on items required for filing a Biologics License Application (BLA) for Revascor^® (rexlemestrocel-L) in the treatment of patients with ischemic heart failure with reduced ejection fraction (HFrEF) and inflammation.

In follow-up to the successful Type B meeting in early 2024 under the existing Regenerative Medicine Advanced Therapy (RMAT) designation for REVASCOR in end-stage HFrEF patients with a left ventricular assist device (LVAD), where FDA stated that the results of the presented studies could support accelerated approval, Mesoblast met with FDA on June 3, 2025 to align on key items for BLA filing. FDA provided formal minutes to the company following the most recent Type B meeting. The minutes confirm alignment with FDA on items regarding chemistry, manufacturing & controls (CMC), potency assays for commercial product release, and proposed design and primary endpoint for the confirmatory trial post-approval.

Mesoblast intends to file by the end of the year for accelerated approval in patients with end-stage ischemic HFrEF implanted with an LVAD.

About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast’s RYONCIL^® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.

Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.

About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications are expected to provide commercial protection extending through to at least 2041 in major markets.

About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and X: @Mesoblast

Forward-Looking Statements
This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

For more information, please contact:

Corporate Communications / Investors
Paul Hughes
T: +61 3 9639 6036
Media – Global
Allison Worldwide
Emma Neal
T: +1 603 545 4843
E: emma.neal@allisonworldwide.com
Media – Australia
BlueDot Media
Steve Dabkowski
T: +61 419 880 486
E: steve@bluedot.net.au

FAQ

What is the status of Mesoblast's (MESO) Revascor® FDA approval process?

Mesoblast has aligned with FDA on key requirements for filing a Biologics License Application (BLA) for Revascor®, with plans to file for accelerated approval by the end of 2025.

What indication is Mesoblast (MESO) seeking approval for with Revascor®?

Mesoblast is seeking approval for Revascor® to treat patients with end-stage ischemic heart failure with reduced ejection fraction (HFrEF) who have been implanted with a left ventricular assist device (LVAD).

What regulatory designations does Mesoblast's Revascor® currently have?

Revascor® has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for end-stage HFrEF patients with a left ventricular assist device.

When does Mesoblast (MESO) plan to submit the BLA for Revascor®?

Mesoblast intends to file the Biologics License Application (BLA) for Revascor® by the end of 2025.

What key agreements were reached between Mesoblast and the FDA for Revascor®?

The FDA and Mesoblast aligned on chemistry, manufacturing & controls (CMC), potency assays for commercial product release, and the proposed design and primary endpoint for the confirmatory trial post-approval.