FDA Provides Seven Years of Orphan-Drug Exclusive Approval for Ryoncil®
Mesoblast (MESO) has secured a significant regulatory milestone as the FDA granted seven years of orphan-drug exclusive approval for Ryoncil® (remestemcel-L), their treatment for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients. The exclusivity prevents FDA approval of competing mesenchymal stromal or stem cell products for this indication until 2032.
Additionally, the company's biologic exclusivity extends until December 2036, preventing biosimilar competition. Mesoblast's intellectual property protection for SR-aGvHD treatments extends through 2044, creating a robust commercial barrier against competitors.
Mesoblast (MESO) ha raggiunto un importante traguardo regolatorio: la FDA ha concesso sette anni di esclusiva come farmaco orfano per Ryoncil® (remestemcel-L), il loro trattamento per la malattia da trapianto contro l’ospite acuta refrattaria agli steroidi (SR-aGvHD) nei pazienti pediatrici. Questa esclusiva impedisce l'approvazione da parte della FDA di prodotti concorrenti a base di cellule stromali mesenchimali o staminali per questa indicazione fino al 2032.
Inoltre, l'esclusiva biologica dell'azienda si estende fino a dicembre 2036, impedendo la concorrenza di biosimilari. La protezione della proprietà intellettuale di Mesoblast per i trattamenti SR-aGvHD dura fino al 2044, creando una solida barriera commerciale contro i concorrenti.
Mesoblast (MESO) ha alcanzado un hito regulatorio significativo, ya que la FDA otorgó siete años de exclusividad como medicamento huérfano para Ryoncil® (remestemcel-L), su tratamiento para la enfermedad de injerto contra huésped aguda refractaria a esteroides (SR-aGvHD) en pacientes pediátricos. Esta exclusividad impide la aprobación por parte de la FDA de productos competidores basados en células estromales mesenquimales o células madre para esta indicación hasta 2032.
Además, la exclusividad biológica de la compañía se extiende hasta diciembre de 2036, evitando la competencia de biosimilares. La protección de la propiedad intelectual de Mesoblast para tratamientos SR-aGvHD se extiende hasta 2044, creando una fuerte barrera comercial contra competidores.
Mesoblast (MESO)는 중요한 규제 성과를 달성했습니다. FDA가 소아 환자의 스테로이드 불응성 급성 이식편대숙주병(SR-aGvHD) 치료제인 Ryoncil®(remestemcel-L)에 대해 7년간 희귀의약품 독점 승인을 부여했습니다. 이 독점권은 2032년까지 이 적응증에 대해 경쟁하는 중간엽 줄기세포 또는 줄기세포 제품의 FDA 승인을 막습니다.
또한, 회사의 생물학적 독점권은 2036년 12월까지 연장되어 바이오시밀러 경쟁을 방지합니다. Mesoblast의 SR-aGvHD 치료제에 대한 지적 재산권 보호는 2044년까지 이어져 강력한 상업적 장벽을 형성합니다.
Mesoblast (MESO) a franchi une étape réglementaire importante puisque la FDA a accordé sept ans d'exclusivité en tant que médicament orphelin pour Ryoncil® (remestemcel-L), leur traitement contre la maladie du greffon contre l'hôte aiguë réfractaire aux stéroïdes (SR-aGvHD) chez les patients pédiatriques. Cette exclusivité empêche l'approbation par la FDA de produits concurrents à base de cellules stromales mésenchymateuses ou de cellules souches pour cette indication jusqu'en 2032.
De plus, l'exclusivité biologique de la société est prolongée jusqu'en décembre 2036, empêchant la concurrence des biosimilaires. La protection de la propriété intellectuelle de Mesoblast pour les traitements SR-aGvHD s'étend jusqu'en 2044, créant une barrière commerciale solide contre les concurrents.
Mesoblast (MESO) hat einen bedeutenden regulatorischen Meilenstein erreicht, da die FDA sieben Jahre Exklusivität als Orphan-Drug für Ryoncil® (remestemcel-L), ihre Behandlung der steroid-refraktären akuten Graft-versus-Host-Erkrankung (SR-aGvHD) bei pädiatrischen Patienten, gewährt hat. Diese Exklusivität verhindert die Zulassung konkurrierender mesenchymaler Stromazell- oder Stammzellprodukte für diese Indikation bis 2032 durch die FDA.
Zusätzlich erstreckt sich die biologische Exklusivität des Unternehmens bis Dezember 2036, wodurch eine Konkurrenz durch Biosimilars verhindert wird. Der geistige Eigentumsschutz von Mesoblast für SR-aGvHD-Behandlungen gilt bis 2044 und schafft eine starke kommerzielle Barriere gegen Wettbewerber.
- Seven years of FDA orphan-drug exclusivity for Ryoncil, preventing competing MSC products
- Biologic exclusivity until December 2036, blocking biosimilar market entry
- Strong IP protection through 2044 for SR-aGvHD treatments
- Multiple layers of market protection securing commercial advantage
- None.
NEW YORK, May 14, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has received seven years of orphan-drug exclusive approval from U.S. Food and Drug Administration (FDA) for Ryoncil® (remestemcel-L) for treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older.
This period of statutory exclusivity means that the FDA will not approve another mesenchymal stromal or stem cell (MSC) products for this indication during the 7-year period from the approval of Ryoncil®. Separately, Mesoblast has biologic exclusivity preventing another sponsor from referencing the Ryoncil® biologic license application (BLA) until December 2036, twelve years from its first approval which would prevent market entry by a biosimilar.
These statutory exclusivities are in addition to Mesoblast’s strong U.S. intellectual property position on MSC composition of matter, manufacturing and indications, including SR-aGvHD, that provide a commercial barrier to entry against competitors through 2044.
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 2044 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 Twitter: @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.
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| Media – Global | |
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| Emma Neal | |
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| E: emma.neal@allisonworldwide.com | |
| Media – Australia | |
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| Steve Dabkowski | |
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| E: steve@bluedot.net.au |
FAQ
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