Ascendis Pharma A/S Announces U.S. Food and Drug Administration Accepts Biologics License Application for TransCon™ hGH for Pediatric Growth Hormone Deficiency

Ascendis Pharma A / S 宣布美国食品药品监督管理局(Food and Drug Administration)接受用于治疗儿童生长激素缺乏症的 Transcon ™ hGH 生物技术许可申请

2020-09-05 05:00:11 BioSpace

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– Prescription Drug User Fee Act target action dateof June 25, 2021 – – FDA indicated that it is currently not planning to hold an Advisory Committee Meeting – COPENHAGEN, Denmark, Sept. 04, 2020 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S A/S (Nasdaq: ASND), a biopharmaceutical company that uses its novel TransCon technologies to address unmet medical needs, today announced that the FDA has accepted the company’s BLA for TransCon hGH (lonapegsomatropin), an investigational long-acting prodrug of somatropin (human growth hormone or hGH) for the treatment for pediatric GHD. A PDUFA date is set for June 25, 2021. The FDA also has indicated that it is currently not planning to hold an Advisory Committee Meeting to discuss the application at this time. “We look forward to engaging with the FDA during its review of our BLA submission for TransCon hGH in pediatric GHD,” said Dana Pizzuti, M.D., Ascendis Pharma’s Senior Vice President of Development Operations. “Based on data from our clinical development program, we believe once-weekly TransCon hGH has the potential to expand treatment options for clinicians and children with GHD.”  TransCon hGH is designed to release somatropin with the same mode of action and distribution as once-daily somatropin products, but with a once-weekly injection.1  The BLA for pediatric GHD is supported by the results a clinical development program that included eight clinical trials evaluating safety and efficacy in more than 400 subjects with GHD. TransCon hGH is an investigational therapy that is not approved for use in any country.  There is no long-acting growth hormone treatment approved by the FDA for the treatment of pediatric GHD. TransCon hGH has received orphan designation in both the U.S. and Europe as a treatment for GHD. The company plans to submit a Marketing Authorisation Application for TransCon hGH in pediatric GHD to the European Medicines Agency in the third quarter of 2020. About Pediatric Growth Hormone Deficiency (GHD)2 Pediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Children with GHD are not only characterized by short stature, but they also may experience metabolic abnormalities, psychosocial challenges and poor quality of life.  For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH, which improves growth and overall endocrine health. About TransCon™ Technology TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology designed to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action may be maintained. TransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally. About Ascendis Pharma A/S  Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon™ technologies to create new and potentially best-in-class therapies. Ascendis Pharma currently has a pipeline of three independent endocrinology rare disease product candidates in clinical development and is advancing oncology as its second therapeutic area of focus. The company continues to expand into additional therapeutic areas to address unmet patient needs. Ascendis is headquartered in Copenhagen, Denmark, with additional offices in Heidelberg and Berlin, Germany, and in Palo Alto and Redwood City, California. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ PDUFA date of June 25, 2021 with respect the BLA Ascendis submitted in June 2020, (ii) the FDA’s indication that it is currently not planning to hold an Advisory Committee Meeting with respect the BLA Ascendis submitted in June 2020, (iii) Ascendis’ plans to submit its marketing application for TransCon hGH for the treatment of pediatric GHD in Europe in the third quarter of 2020, (iv) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, (v) Ascendis’ product pipeline and expansion into additional therapeutic areas, and (vi) Ascendis’ expectations regarding its ability to utilize its TransCon technologies to create new and potentially best-in-class therapies.  Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: unforeseen safety or efficacy results in its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development and potential commercialization of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs, selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for planned clinical studies; Ascendis’ ability to obtain additional funding, if needed, to support its business activities and the effects on its business of the worldwide COVID-19 pandemic. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ prospectus supplement filed on July 9, 2020 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on April 3, 2020. Forward-looking statements do not reflect the potential impact of any future in-licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma Group. © September 2020 Ascendis Pharma A/S. ________________ 1 Sprogøe K, et al. Endocrine Connections. 2017(6): R171-181. 2 Backeljauw PF, et al. Endocrine Disorders in Adolescents. 2014: 292-403.
–自2021年6月25日起,处方药使用者费法的目标行动– – FDA 表示目前没有计划召开咨询委员会会议- 丹麦 COPENHHAGEN ,2020年9月4日( GLOBE NEWSWIRE )-- Ascendis Pharma A / S A / S ( Nasdaq : ASND ),一家生物制药公司,利用其新的 TransCon 技术来满足未满足的医疗需求,今天宣布 FDA 已经接受该公司的 BLA 用于 TransCon hGH ( Loonapegsomatropin ),用于治疗儿童 GHD 的生长激素(人类生长激素或 hGH )长效前药。PDUFA 日期设定为2021年6月25日。FDA 还表示,目前还没有计划召开咨询委员会会议讨论该申请。 Ascendis Pharma 公司负责开发运营的高级副总裁 Dana Pizzuti 医学博士(M.D.)博士说:“我们期待在审查 BLA 提交的儿童 GHD 中的 Transcon hGH 时与 FDA 合作。”“根据我们临床开发计划的数据,我们相信每周一次的 TransCon hGH 有可能扩大临床医生和患有 GHD 的儿童的治疗选择。”这是一个很好的例子 Transcon hGH 的目的是释放生长激素,其作用和分布方式与每日一次的生长激素产品相同,但每周注射一次。1儿童 GHD 的 BLA 得到了一个临床开发计划的支持,该计划包括八个临床试验,评估了400多名 GHD 患者的安全性和有效性。 TransConhGH 是一种未经批准在任何国家使用的研究疗法。目前尚无 FDA 批准的用于治疗儿童 GHD 的长效生长激素治疗。TransConhGH 在美国和欧洲都获得了孤儿的称号,作为治疗 GHD 的药物。公司计划于2020年第三季度向欧洲药品管理局提交儿童 GHD 的 TransCon hGH 上市许可申请。 关于儿童生长激素缺乏症( GHD )2 儿科 GHD 是一种严重的孤儿疾病,当垂体没有产生足够的生长激素时引起。患有 GHD 的儿童不仅身材矮小,还可能经历代谢异常、心理社会挑战和生活质量低下。数十年来, GHD 的护理标准一直是每日皮下注射 hGH ,从而改善生长和整体内分泌健康。 关于 Transcon ™技术 TransCon 指的是“瞬时共轭”。专有的 TransCon 平台是一项创新技术,旨在创造优化治疗效果的新疗法,包括疗效、安全性和剂量频率。TransCon 分子有三个组成部分:一种未修饰的母体药物,一种保护它的惰性载体,以及一种暂时结合两者的连接剂。当绑定时,载体会使母体药物失去活性,并使其免于清除.当注射到体内时,生理条件(如 pH 值和温度)以可预测的方式启动活性的、未修饰的母体药物的释放。由于母药未被修饰,其原有的作用方式可能得以维持。TransCon 技术旨在广泛应用于多个治疗领域的蛋白质、多肽或小分子,并在系统或局部应用。 关于 Ascendis Pharma A / S Ascendis Pharma 正在应用其创新平台技术,打造一家领先的、完全整合的生物制药公司,专注于在患者生活中做出有意义的改变。公司以病人的核心价值、科学和热情为指导,利用 TransCon ™技术创造新的、潜在的最佳治疗方案。 Ascendis Pharma 目前拥有三个独立的内分泌罕见疾病产品候选药物在临床开发中,正在推进肿瘤治疗作为其第二个重点治疗领域。该公司继续扩大到其他治疗领域,以满足未得到满足的患者需求。 Ascendis 总部位于丹麦哥本哈根,在德国的海德堡和柏林,以及加利福尼亚州的帕洛阿尔托和雷德伍德都设有办事处。 前瞻性陈述 本新闻稿载有涉及重大风险及不明朗因素的前瞻性陈述。除历史事实陈述外,本新闻稿中有关 Ascendis 未来经营、计划和管理目标的所有陈述均为前瞻性陈述。此类声明的示例包括但不限于以下声明:㈠ Ascendis ’ PDUFA 日期为2021年6月25日,与2020年6月提交的 BLA Ascendis 有关;㈡ FDA 表示目前不打算就2020年6月提交的 BLA Ascendis 举行咨询委员会会议;( iii ) Ascendis 计划于2020年第三季度提交其用于治疗欧洲儿童 GHD 的 TransCon hGH 上市申请,( iv ) Ascendis 应用其平台技术建立领先的、完全集成的生物制药公司的能力,( v ) Ascendis 的产品管线和扩展到其他治疗领域,以及( vi ) Ascendis 对利用其 TransCon 技术创造新的和潜在的最佳治疗方法的能力的期望。Ascendis 可能无法实际实现计划、实施意图或达到前瞻性陈述中披露的预期或预测,您不应过度依赖这些前瞻性陈述。实际结果或事件可能与前瞻性声明中披露的计划、意图、预期和预测存在重大差异。各种重要因素可能导致实际结果或事件与 Ascendis 作出的前瞻性陈述存在重大差异,包括以下内容:其肿瘤项目的不可预见的安全性或有效性结果, TransCon hGH ,Transcon PTH 和 Transcon CNP 或其他开发计划;与肿瘤项目、 Transcon hGH 、 Transcon PTH 和 Transcon CNP 或其他开发计划的开发和潜在商业化相关的不可预见费用,一般销售、总务和管理费用,其他研发费用和 Ascendis 业务;延迟开发其肿瘤项目、 Transcon hGH 、 Transcon PTH 和 Transcon CNP 或其他与制造相关的开发计划。监管要求、患者招募速度或其他不可预见的延迟;依赖第三方制造商为计划中的临床研究提供研究药物; Ascendis 在必要时获得额外资金的能力,以支持其业务活动以及全球 COVID-19大流行对其业务的影响。有关可能导致实际结果与这些前瞻性声明中表达的风险和不确定性的进一步描述,以及与 Ascendis 总体业务相关的风险,请参见2020年7月9日提交的 Ascendis 招股说明书补充文件以及 Ascendis 提交或提交的当前和未来报告。美国证券交易委员会( SEC ),包括其于2020年4月3日向 SEC 提交的20-F 表格年度报告。前瞻性声明并不反映 Ascendis 可能参与或进行的任何未来许可、合作、收购、合并、处置、合资或投资的潜在影响。除法律要求外, Ascendis 不承担更新任何前瞻性陈述的义务。 Ascendis 、 Ascendis Pharma 、 Ascendis Pharma 徽标、公司徽标和 TransCon 为 Ascendis Pharma Group 拥有的商标。©2020年9月 Ascendis Pharma A / S 。 _________ 1 Sprogo ø e K 等人。内分泌连接.2017(6): R171-181。 2 Backeljauw PF 等。青少年内分泌失调.2014年:292-403。

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