United States Food & Drug Administration (FDA) Grants Mesoblast Orphan-Drug Designation for Revascor® (Rexlemestrocel-L) in Children With Congenital Heart Disease
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Insights
The Orphan-Drug Designation (ODD) granted to Mesoblast's Revascor® can potentially unlock several financial benefits for the company, such as tax credits for clinical testing, exemption from FDA application fees and market exclusivity for seven years post-approval. These incentives are designed to encourage the development of treatments for rare diseases, which might otherwise not be profitable due to the small patient populations. The market exclusivity is particularly valuable as it protects Mesoblast's investment in Revascor® by preventing similar drugs from entering the market, thus potentially enhancing revenue projections and positively influencing investor sentiment.
However, investors should consider the high costs and long timelines associated with bringing a drug through clinical trials to market. While the ODD is a positive step, the actual impact on Mesoblast's financials will depend on successful approval and commercialization of the treatment. Additionally, the pediatric market for this drug is limited, which may cap the revenue potential despite the granted exclusivity.
Revascor®'s progress in clinical trials underscores its potential as a significant medical advancement for children with hypoplastic left heart syndrome (HLHS). The positive trial results indicating that Revascor® may facilitate successful surgeries for HLHS patients represent a substantial step forward in pediatric cardiology. The ability of Revascor® to increase left ventricular volumes is crucial for the success of biventricular conversion surgeries, which can ultimately improve survival rates and quality of life for these children.
From a research perspective, the data suggesting a 100% success rate in Revascor®-treated children versus 57% in controls is statistically significant and could signal a breakthrough in treatment efficacy. However, the small sample size of 19 children should be taken into account when projecting the broader applicability of the therapy. Further research and larger trials will be necessary to validate these findings and ensure the safety and efficacy of Revascor® for a wider patient population.
The Orphan-Drug and Rare Pediatric Disease Designations by the FDA reflect not only the potential clinical importance of Revascor® but also the economic implications for the healthcare system. Treatments for conditions like HLHS are often associated with high medical costs due to the complexity of surgeries and long-term care required. If Revascor® proves to be effective in facilitating successful surgical outcomes, it could lead to a reduction in overall healthcare expenditures for this patient group by potentially decreasing the need for multiple surgeries or long-term medical management.
It's important to consider the cost-effectiveness of new treatments like Revascor®. While the drug might come at a high price due to development costs and the ODD's market exclusivity, the long-term savings from improved surgical outcomes and reduced healthcare utilization could justify the investment. However, these economic benefits must be weighed against the affordability and accessibility of the treatment for patients and healthcare providers.
NEW YORK, Feb. 14, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food and Drug Administration (FDA) has granted its allogeneic cell therapy Revascor® (rexlemestrocel-L) an Orphan-Drug Designation (ODD) following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life threatening congenital heart condition. This follows the Rare Pediatric Disease Designation (RPDD) granted by FDA last month.
Mesoblast Chief Executive Silviu Itescu said: “We are very pleased to have now been granted both Orphan-Drug Designation and Rare Pediatric Disease Designation by FDA for REVASCOR in the treatment of children with this often-fatal congenital heart condition. The designations were granted on the back of the results from children in a randomized controlled trial indicating that REVASCOR may increase the ability to successfully accomplish life-saving surgery. We plan to meet with FDA to discuss the pathway for approval in this indication.”
Results from a blinded, randomized, placebo-controlled prospective trial of REVASCOR conducted in the United States in children with HLHS were published in the December 2023 issue of the peer reviewed The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open).1
In the HLHS trial conducted in 19 children, a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in the desired outcome of significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared with controls as measured by 3D echocardiography, (p=0.009 & p=0.020 respectively).
These changes are indicative of clinically important growth of the small left ventricle, facilitating the ability to have a successful surgical correction, known as full biventricular (BiV) conversion, which allows for a normal two ventricle circulation with the surgically repaired left ventricle taking over circulatory support to the body. Without full BiV conversion the right heart chamber is under excessive strain with increased risk of heart failure and death.
As noted in our recent publication, “The fact that
About Orphan Drug Designation
The FDA’s Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the United States. Orphan designation qualifies the sponsor of the drug for various development incentives, including eligibility for seven years of market exclusivity upon regulatory approval, exemption from FDA application fees, tax credits for qualified clinical trials, and other potential assistance in the drug development process.
About Rare Pediatric Disease Designation
FDA awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. Under this program, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a Priority Review Voucher (PRV) that can be redeemed to receive a priority review of a subsequent marketing application for a different product or may be sold or transferred to a third party.
About Hypoplastic Left Heart Syndrome (HLHS)
HLHS is a severe congenital heart disease in which the left side of the heart does not fully develop and effective pumping of oxygenated blood by the left ventricle to the rest of the body is reduced. Without immediate surgery after birth, the prognosis is dismal with HLHS overall being responsible for
About Revascor® (rexlemestrocel-L) in Heart Disease
REVASCOR is an allogeneic preparation of immunoselected and culture-expanded mesenchymal precursor cells which have been shown previously to have multiple mechanisms-of-action that may be beneficial to children with HLHS including neovascularization, anti-fibrosis, anti-apoptosis, immunomodulation, reduction in inflammation, and reversal of endothelial dysfunction. In the DREAM-HF randomized sham-placebo controlled prospective trial of REVASCOR in 565 randomized adult patients with heart failure with low ejection fraction (HFrEF), a single intramyocardial administration of REVASCOR into the left ventricle resulted in significant improvement in LV ejection fraction at 12 months,3 indicative of strengthened overall LV systolic function.
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 Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of late-stage product candidates which 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 has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. 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 is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease, biologic-resistant inflammatory bowel disease, and acute respiratory distress syndrome. Rexlemestrocel-L is in development for advanced chronic heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblast’s licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.
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 Twitter: @Mesoblast
References / Footnotes
- Wittenberg RE, Gauvreau K, Leighton J, Moleon-Shea M, Borow KM, Marx GR, Emani SM, Prospective randomized controlled trial of the safety and feasibility of a novel mesenchymal precursor cell therapy in hypoplastic left heart syndrome, JTCVS Open Volume 16, Dec 2023, doi: https://doi.org/10.1016/j.xjon.2023.09.031
- Kritzmire, S. M, et al. (2022). Hypoplastic left heart syndrome. https://www.ncbi.nlm.nih.gov/books/NBK554576/#
- Perin EC, Borow KM, Henry TD, et al. Randomized Trial of Targeted Transendocardial Mesenchymal Precursor Cell Therapy in Patients With Heart Failure. Journal of the American College of Cardiology. 2023;81(9):849-863. doi:10.1016/j.jacc.2022.11.061
Forward-Looking Statements
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FAQ
What is the significance of the Orphan-Drug Designation granted by the FDA to Mesoblast Limited?
What were the results of the clinical trial conducted on children with hypoplastic left heart syndrome using REVASCOR?
Where were the results of the clinical trial published?
What is the aim of REVASCOR treatment in children with HLHS?
