FDA为Ryoncil®提供七年的孤儿药独家批准-动脉网

FDA Provides Seven Years of Orphan-Drug Exclusive Approval for Ryoncil®

PHARMA FOCUS ASIA 等信源发布 2025-05-15 14:35

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Mesoblast, 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..

Mesoblast，炎症性疾病同种异体细胞药物的全球领导者，今天宣布已获得美国食品和药物管理局 (FDA) 授予的七年孤儿药独占权，用于 Ryoncil®（remestemcel-L）治疗两个月及以上年龄的类固醇难治性急性移植物抗宿主病 (SR-aGvHD) 的儿科患者。

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..

这段法定独占期意味着，FDA 在 Ryoncil® 获批后的 7 年内不会批准其他用于该适应症的间充质基质或干细胞 (MSC) 产品。此外，Mesoblast 拥有生物制品独占权，防止其他申请者在 2036 年 12 月之前参考 Ryoncil® 的生物制品许可申请 (BLA)，这一独占权自其首次获批起持续十二年，可阻止生物类似药进入市场。

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.

这些法定的独占权是对 Mesoblast 在 MSC 成分、制造和适应症（包括 SR-aGvHD）方面强大的美国知识产权地位的补充，为公司提供了直至 2044 年的商业进入壁垒以对抗竞争对手。

About Mesoblast

关于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（公司）是开发用于治疗严重和危及生命的炎症性疾病的同种异体（现成）细胞药物的世界领导者。公司专有的间充质谱系细胞治疗技术平台的疗法通过释放抗炎因子来应对严重炎症，这些因子能够对抗并调节免疫系统的多个效应分支，从而显著减轻破坏性的炎症过程。

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公司的RYONCIL®（remestemcel-L）用于治疗两个月及以上的儿童类固醇难治性急性移植物抗宿主病（SR-aGvHD），是首个获FDA批准的间充质基质细胞（MSC）疗法。请访问www.ryoncil.com查看完整的处方信息。

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.

Mesoblast致力于基于其remestemcel-L和rexlemestrocel-L同种异体基质细胞技术平台，开发针对不同适应症的其他细胞疗法。RYONCIL正被开发用于治疗其他炎症性疾病，包括成人SR-aGvHD和生物抵抗性炎症性肠病。

Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China..

Rexlemestrocel-L 正在被开发用于治疗心力衰竭和慢性下背痛。公司已在日本、欧洲和中国建立了商业合作伙伴关系。

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..

关于Mesoblast的知识产权：Mesoblast拥有强大而广泛的全球知识产权组合，已获得或申请了1000多项专利，涵盖间充质基质细胞成分、制造方法及适应症。这些已授权的专利和专利申请预计将在主要市场提供至少到2044年的商业保护。

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.

关于Mesoblast的生产：公司专有的生产流程产出的是工业规模、冷冻保存、可立即使用的细胞药物。这些细胞疗法具有明确的药物释放标准，计划在全球范围内让患者随时可用。

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.

Mesoblast在澳大利亚、美国和新加坡设有分支机构，并在澳大利亚证券交易所（MSB）和纳斯达克（MESO）上市。欲了解更多信息，请访问www.mesoblast.com、LinkedIn：Mesoblast Limited 和 Twitter：@Mesoblast。

Forward-Looking Statements

前瞻性声明

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.

本新闻稿包含前瞻性陈述，这些陈述涉及未来事件或我们的未来财务表现，并包含已知和未知的风险、不确定性以及其他可能导致我们的实际结果、活动水平、表现或成就与这些前瞻性陈述明示或暗示的任何未来结果、活动水平、表现或成就存在重大差异的因素。

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.

我们根据《1995年私人证券诉讼改革法案》及其他联邦证券法的安全港条款作出这些前瞻性陈述。前瞻性陈述不应被解读为对未来业绩或结果的保证，实际结果可能与这些前瞻性陈述中预期的结果不同，且差异可能会重大且不利。

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 c.

前瞻性声明包括但不限于以下方面的声明：Mesoblast公司临床前和临床研究的启动、时间、进展和结果，以及Mesoblast的研发项目；Mesoblast将候选产品推进至临床研究、招募患者并成功完成临床研究（包括多国临床试验）的能力；Mesoblast提升其制造能力的能力；监管文件提交和批准、生产活动及产品营销活动的时间或可能性（如有）；Mesoblast的RYONCIL针对儿童SR-aGVHD及其他任何获批准的候选产品的商业化；围绕干细胞疗法使用的监管或公众认知及其市场接受度；如果Mesoblast的候选产品获得批准，可能因患者不良事件或死亡而被撤市的风险；战略合作可能带来的潜在益处。

Release authorized by the Chief Executive.

行政长官授权发布。

For more information, please contact:

更多信息，请联系：

Corporate Communications / Investors

企业传播／投资者

Paul Hughes

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T: +61 3 9639 6036

电话：+61 3 9639 6036

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