Vericel Announces Submission of Biologics License Application to the FDA for NexoBrid for the Treatment of Severe Thermal Burns

Vericel 宣布向 FDA 提交生物技术许可申请,用于治疗严重热烧伤

2020-06-30 21:31:15 BioSpace

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CAMBRIDGE, Mass., June 30, 2020 -- Vericel Corporation today announced the submission of a Biologics License Application to the U.S. Food and Drug Administration seeking the approval of NexoBrid® for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. NexoBrid is approved in the European Union and other international markets and has been designated as an orphan biologic in the United States, European Union and other international markets. Vericel holds an exclusive license for North American commercial rights to NexoBrid. Funding and technical support to MediWound Ltd. for development of NexoBrid is provided by the U.S. Biomedical Advanced Research and Development Authority . The BLA submission for NexoBrid is based on multiple preclinical and clinical studies including the pivotal Phase 3 U.S. clinical study (DETECT) of NexoBrid in adult patients with deep partial-thickness and full-thickness thermal burns up to 30% of total body surface area. The study met its primary endpoint of complete eschar removal as well as all secondary endpoints, including shorter time to eschar removal, a lower incidence of surgical eschar removal, and lower blood loss during eschar removal compared to standard of care (SOC), including both surgical and non-surgical debridement methods. A key safety endpoint, non-inferiority in time to complete wound closure compared with patients treated with SOC, was also achieved. Long-term follow-up data to assess cosmesis, function, and quality of life, including 12-month results from the DETECT study, were also included in the submission. “The BLA submission for NexoBrid marks an important milestone in our partnership with MediWound, bringing us one step closer to providing NexoBrid as an innovative treatment for the thousands of patients admitted to burn units each year with deep partial-thickness and full-thickness burns who would benefit from rapid and selective eschar removal,” said Nick Colangelo, President and CEO of Vericel. “We look forward, together with MediWound, to working with the FDA during the BLA filing and review process as we seek marketing approval for NexoBrid in the United States.” Sharon Malka, CEO of MediWound added, “Submitting the NexoBrid BLA has been a team effort and we thank all of the investigators, their teams, our employees and all our partners, especially BARDA and Vericel, for their commitment to the program. This is a major milestone for MediWound and it is gratifying to know NexoBrid is one step closer to being available to help burn victims in the U.S.” About NexoBrid NexoBrid is a topically administered biological product that enzymatically removes nonviable burn tissue, or eschar, in patients with deep partial and full-thickness thermal burns within four hours of application without harming viable tissue. NexoBrid is approved in the European Union and other international markets and has been designated as an orphan biologic drug in the United States, European Union and other international markets. Vericel holds an exclusive license for North American commercial rights to NexoBrid. In January 2019, MediWound announced positive top-line results from the acute phase of the pivotal Phase 3 U.S. clinical study (DETECT) of NexoBrid in adult patients with deep partial-and full-thickness thermal burns up to 30 percent of total body surface area. The study met its primary endpoint of complete eschar removal compared to gel vehicle as well as all secondary endpoints compared to standard of care (SOC), including shorter time to eschar removal, a lower incidence of surgical eschar removal and lower blood loss during eschar removal. Safety endpoints, including the key safety endpoint of non-inferiority in time to complete wound closure compared with patients treated with SOC, were also achieved. In addition, the twelve-month follow-up safety data of cosmesis and function were found to be comparable between the treatment and SOC arms, and no new safety signals were observed. Additional twenty-four-month long term safety follow up data will be submitted as a safety labeling update as part of a post-approval commitment. NexoBrid is currently an investigational product in the United States. About Vericel Corporation Vericel is a leader in advanced therapies for the sports medicine and severe burn care markets. The company markets two cell therapy products in the United States. MACI® (autologous cultured chondrocytes on porcine collagen membrane) is an autologous cellularized scaffold product indicated for the repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults. Epicel® (cultured epidermal autografts) is a permanent skin replacement for the treatment of patients with deep dermal or full-thickness burns greater than or equal to 30% of total body surface area. The company also holds an exclusive license for North American commercial rights to NexoBrid®, a registration-stage biological orphan product for debridement of severe thermal burns. For more information, please visit the company’s website at www.vcel.com. Epicel® and MACI® are registered trademarks of Vericel Corporation. NexoBrid® is a registered trademark of MediWound Ltd. and is used under license to Vericel Corporation. © 2019 Vericel Corporation. All rights reserved. Cautionary Note Regarding Forward-Looking Statements Vericel cautions you that all statements other than statements of historical fact included in this press release that address activities, events or developments that we expect, believe or anticipate will or may occur in the future are forward-looking statements. Although we believe that we have a reasonable basis for the forward-looking statements contained herein, they are based on current expectations about future events affecting us and are subject to risks, assumptions, uncertainties and factors, all of which are difficult to predict and many of which are beyond our control. Actual results may differ materially from those expressed or implied by the forward-looking statements in this press release. These statements are often, but are not always, made through the use of words or phrases such as “anticipates,” “intends,” “estimates,” “plans,” “expects,” “continues,” “believe,” “guidance,” “outlook,” “target,” “future,” “potential,” “goals” and similar words or phrases, or future or conditional verbs such as “will,” “would,” “should,” “could,” “may,” or similar expressions. Specifically, this press release contains forward-looking statements concerning the anticipated progress, development, objectives, expectations and commercial potential of NexoBrid. Among the factors that may cause results to be materially different from those stated herein are the inherent uncertainties associated with the timing and conduct of clinical trial and product development activities; the timing or likelihood of regulatory approvals; the ability to successfully develop and commercialize NexoBrid, including its commercial growth potential and the market demand for the product; the availability of funding from BARDA under its agreement with MediWound for use in connection with NexoBrid development activities; competitive developments; whether FDA will accept all or part of the BLA and provide marketing approval for NexoBrid in the United States; the risks related to the timing and conduct of our NEXT Study; the impact of applicable laws and regulations; and the uncertainties associated with the scope, scale and duration of the impact of the COVID-19 pandemic. For example, we are unable to predict how the pandemic will affect the overall healthcare infrastructure, including the pace with which governmental agencies, such as the FDA, will review and approve regulatory submissions. Additional government-imposed quarantines and requirements to “shelter at home” or other incremental mitigation efforts also may impact our ability to source supplies for our operations or our ability to sell and support the use of NexoBrid in the future. These and other significant factors are discussed in greater detail in Vericel’s Annual Report on Form 10-K for the year ended December 31, 2019, filed with the Securities and Exchange Commission (“SEC”) on February 25, 2020, Quarterly Reports on Form 10-Q and other documents filed by the Company with the SEC from time-to-time. These forward-looking statements reflect management’s current views as of the date hereof and Vericel does not undertake, and specifically disclaims, any obligation to update any of these forward-looking statements to reflect a change in our views or events or circumstances that occur after the date of this release except as required by law. Investor Contact: Lee Stern Solebury Trout lstern@troutgroup.com +1 (646) 378-2922
马萨诸塞州.,2020年6月30日—— Vericel 公司今天宣布向美国食品药品监督管理局(Food and Drug Administration)提交生物技术许可申请,寻求批准 NexoBrid ®用于在深度局部厚度和/或厚度完全热烧伤的成年人中去除 escha 。NexoBrid 在欧盟和其他国际市场获得批准,并被指定为美国、欧盟和其他国际市场的孤儿生物制剂。Vericel 拥有 NexoBrid 北美商业权利的独家许可。为 MediWound Ltd .开发 NexoBrid 提供资金和技术支持,由美国生物医学高级研究与开发管理局提供。 提交给 NexoBrid 的 BLA 是基于多项临床前和临床研究的,包括 NexoBrid 的关键3期美国临床研究( DETET )在成人患者深部分厚度和全厚度热烧伤高达总体表面积的30%。该研究满足了其主要终点,即完全清除 escha 和所有次要终点,包括较短的时间去 escha 清除,较低的发生率手术 escha 清除,和较低的血液损失在 escha 清除标准护理( SOC ),包括手术和非手术的去杠杆化方法。与使用 SOC 治疗的患者相比,一个关键的安全终点,即完成伤口闭合的时间非劣后性也得到了实现。提交材料还包括评估宇宙学、功能和生活质量的长期后续数据,包括 DETECT 研究的12个月结果。 “ BLA 提交给 NexoBrid 标志着我们与 MediWound 合作伙伴关系的一个重要里程碑,使我们更接近于提供 NexoBrid 作为一种创新的治疗方法,为每年被承认使用深度局部厚度和全厚度烧伤的数千名患者提供这种治疗方法,他们将受益于快速和选择性的 escha 切除。Vercel 总裁兼首席执行官 NickColangelo 说。“我们期待着与 MediWound 一起,在 BLA 备案和审查过程中与 FDA 合作,以寻求 NexoBrid 在美国的上市许可。” MediWound 首席执行官 Sharon Malka 补充说:“提交 NexoBrid BLA 是一个团队的努力,我们感谢所有调查人员、他们的团队、我们的员工以及我们的所有合作伙伴,特别是 BARDA 和 Vericel ,感谢他们对该计划的承诺。这是 MediWound 的一个重要里程碑,了解 NexoBrid 是帮助美国烧伤受害者的更近一步是令人欣慰的。” 关于 NexoBrid NexoBrid 是一种局部给药的生物制品,它能在应用4小时内酶解不能生存的烧伤组织,或 escha ,用于深度局部和全厚度的热烧伤患者,而不会损害可行的组织。NexoBrid 在欧盟和其他国际市场获得批准,并被指定为美国、欧盟和其他国际市场的孤儿生物药物。Vericel 拥有 NexoBrid 北美商业权利的独家许可。2019年1月, MediWound 公布了 NexoBrid 的美国临床研究( DETECTT )的关键阶段3期急性阶段的正面顶线结果,该阶段的成人深度局部和全厚度热烧伤患者的体表面积高达30%。该研究满足了其主要终点的完整 eshar 清除相比于凝胶车辆以及所有次要终点相比护理标准( SOC ),包括较短的时间去 eshar 清除,较低的发病率手术 eshar 清除和较低的血液损失在 eshar 清除。安全终点,包括关键安全终点的非劣后性及时完成伤口愈合相比,患者治疗 SOC ,也取得了。此外,12个月的美容和功能的后续安全数据被发现是可比的治疗和 SOC 武器,没有观察到新的安全信号。作为批准后承诺的一部分,额外的24个月长期安全后续数据将作为安全标签更新提交。NexoBrid 目前是美国的一种研究产品。 关于 Verizel 公司 Vercel 是运动医学和严重烧伤护理市场的先进疗法的领导者。公司在美国市场销售两种细胞治疗产品。MACI ®(猪胶原膜上的自体培养软骨细胞)是一种自体细胞组织支架产品,用于修复有症状的、单独的或多个完整的膝关节软骨缺损,并伴有或不伴有成骨。表皮®(培养的表皮自体移植物)是一种永久性皮肤置换,用于治疗深度皮肤或厚度烧伤大于或等于全身表面面积30%的患者。该公司还拥有 NexoBrid ®的北美商业权利的独家许可, NexoBrid ®是一种注册阶段的生物孤儿产品,用于重热烧伤的清创。详情请访问公司网站 www.vcel.com 。 Epidecel ®和 MACI ®是 Vercel Corporation 的注册商标。NexoBrid ®是 MediWound Ltd .的注册商标,许可 Vericel Corporation 使用。©2019 Verizel Corporation 。保留所有权利。 有关前瞻性陈述的注意事项 Vercel 提醒您,除了包含在本新闻稿中的历史事实陈述以外,所有涉及我们预期、相信或预期未来将或可能发生的活动、事件或发展的陈述均为前瞻性陈述。尽管我们相信,我们对本文件所载的前瞻性陈述有合理的基础,但该等陈述乃基于目前对影响我们的未来事件的预期,并受限于风险、假设、不明朗因素及因素,所有该等风险、假设、不明朗因素及因素均难以预测,且其中许多不受我们控制。实际结果可能与本新闻稿中前瞻性陈述所表达或暗示的结果存在重大差异。这些陈述通常是,但并非总是,通过使用诸如“预期”、“意图”、“估计”、“计划”、“预期”、“继续”、“相信”、“指导”、“前景”、“目标”、“未来”、“潜力”、“目标”和类似的词语或短语,或者诸如“ will ”、“ will ”、“ should ”、“ should ”、“ should ”、“ should ”、“ should ”、“ should ”等未来或条件动词来作出。“可以,”或者类似的表达。 具体而言,本新闻稿载有关于 NexoBrid 的预期进展、发展、目标、预期和商业潜力的前瞻性陈述。可能导致结果与本文所述结果存在重大差异的因素包括与临床试验和产品开发活动的时间和开展相关的固有不确定性、监管批准的时间或可能性、成功开发和商业化 NexoBrid 的能力、包括其商业增长潜力和产品的市场需求;根据与 MediWound 达成的协议, BARDA 提供的资金可用于与 NexoBrid 开发活动有关的用途;竞争性开发; FDA 是否将接受 BLA 的全部或部分内容,并为 NexoBrid 在美国提供营销批准;与我们 NEXT 研究的时间和开展有关的风险;适用法律和法规的影响;与 COVID-19大流行影响的范围、规模和持续时间相关的不确定性。例如,我们无法预测大流行将如何影响整体医疗基础设施,包括 FDA 等政府机构审查和批准提交的监管文件的速度。政府实施的额外隔离措施和“在家里避难”或其他渐进缓解措施的要求,也可能影响我们为我们的运营采购供应的能力,或我们未来销售和支持使用 NexoBrid 的能力。 这些和其他重要因素在 Vericel 于2020年2月25日向美国证券交易委员会(“ SEC ”)提交的截至2019年12月31日的10-K 年度报告、10-Q 季报和公司不时向 SEC 提交的其他文件中有更详细的讨论。这些前瞻性陈述反映了管理层截至本协议签署之日的当前观点, Vericel 不承担更新这些前瞻性陈述的任何义务,以反映本协议发布之日之后发生的我们的观点或事件或情况的变化,除非法律要求。 投资者联系方式: 李斯特恩 Solebury Trout lstern @ curtgroup.com +1(646)378-2922

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