Acceleron Receives FDA Orphan Drug Designation for Sotatercept in Pulmonary Arterial Hypertension


2019-09-11 10:40:00 BioSpace


Acceleron Pharma Inc. , a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that the United States Food and Drug Administration has granted Orphan Drug designation to sotatercept for the treatment of patients with pulmonary arterial hypertension . “We’re pleased that the FDA has granted this designation for sotatercept,” said Janethe de Oliveira Pena, MD, PhD, Vice President, Pulmonary Medical Research at Acceleron. “In preclinical studies, sotatercept has demonstrated an ability to target the underlying mechanisms of PAH, which is a rare disease of high unmet medical need. We believe that if similar effects are seen in a clinical setting, sotatercept has the potential to become an important addition to the standard of care in PAH.” Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website at Sotatercept is being evaluated in two Phase 2 trials in patients with PAH: the PULSAR trial, which completed its target enrollment in June of this year and the SPECTRA exploratory trial, which is currently enrolling. The Company expects to report top-line results from the PULSAR trial during the first quarter of 2020. Sotatercept is an investigational therapy that is not approved for any use in any country. About Sotatercept Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure. Sotatercept is currently being evaluated in the PULSAR and SPECTRA Phase 2 trials in PAH. For more information, please visit About PAH PAH is a rare and chronic, rapidly progressing disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant strain on the heart, often leading to limited physical activity, heart failure, and reduced life expectancy. The 5-year survival rate for patients with PAH is approximately 57%. Available therapies generally act by promoting the dilation of pulmonary vessels without addressing the underlying cause of the disease. As a result, PAH often progresses rapidly for many patients despite standard of care treatment. A growing body of research has implicated imbalances in BMP and TGF-beta signaling as a primary driver of PAH in familial, idiopathic, and acquired forms of the disease. About Acceleron Acceleron is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair. Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its neuromuscular program with ACE-083, a locally-acting Myostatin+ agent in Phase 2 development in facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease and is conducting a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension.
Aceleron Pharma Inc .,一家领先的生物制药公司,致力于发现和开发治疗严重和罕见疾病的 TGF-β超家族疗法,今天宣布,美国食品药品监督管理局(Food and Drug Administration)已批准孤儿药物指定用于治疗肺动脉高压患者。 “我们很高兴美国食品和药物管理局已经批准了这一指定的精神状态,”医学博士,博士,副总裁,肺医学研究的 Acceeleron 。“在临床前研究中,苏氨西普已经证明有能力针对 PAH 的潜在机制,这是一种罕见的高未满足医疗需求的疾病。我们相信,如果在临床环境中也能看到类似的效果,那么精神感应有可能成为 PAH 护理标准的重要补充。” 孤儿的指定是由 FDA 孤儿产品开发办公室授予的,目的是推动评估和开发安全有效的治疗罕见病或影响美国少于20万人的罕见疾病的治疗方法。根据《孤儿药物法案》, FDA 可以为临床试验费用提供资金。税收优惠, FDA 的用户费用福利,和七年的市场独占性后,在美国 FDA 的市场批准。授予孤儿指定请求不会改变获得营销批准的标准监管要求和流程。有关孤儿指定的更多信息,请访问 FDA 网站 。 在 PAH 患者的2期临床试验中, Sorecepit 正在进行评估: PULSAR 试验,该试验于今年6月完成了目标注册,目前正在进行 SPECTRA 探索性试验。公司预计在2020年第一季度报告 PULSAR 试验的顶级结果。 强迫症是一种未经批准在任何国家使用的研究疗法。 关于强迫的问题 Sostatercept 是一种研究试剂,被设计为 TGF-beta 超家族成员的选择性配体陷阱,以重新平衡 BMPR2信号传导,这是 PAH 的关键分子驱动因素。在 PAH 的临床前研究中,顺铂逆转肺血管肌肉组织,改善右心衰竭的指标。目前,在 PAH 的 PULSAR 和 SPECTRA2期试验中正在评估 Sorecepit 。有关更多信息,请访问 。 关于 PAH 肺动脉高压是一种罕见的慢性进展迅速的疾病,其特点是小肺动脉收缩和肺动脉循环血压升高。PAH 会导致心脏明显的紧张,经常导致有限的体力活动,心力衰竭和降低预期寿命。PAH 患者5年生存率约为57%。现有的治疗方法通常通过促进肺血管扩张而不解决疾病的根本原因。因此,尽管有标准的护理治疗, PAH 往往进展迅速。越来越多的研究表明 BMP 和 TGF-beta 信号不平衡是 PAH 家族、特发性和后天形成的主要驱动因素。 关于 Acceleron Aceleron 是一家临床阶段的生物制药公司,致力于发现,开发和商业化的治疗药物治疗严重和罕见的疾病。本公司在了解 TGF-beta 超家族生物学和蛋白质工程方面的领导能力产生了创新化合物,使人体能够调节细胞生长和修复。 Aceleron 专注于血液学、神经肌肉和肺部疾病的研究和开发工作。在血液学方面,本公司与其全球合作伙伴新基(Celgene)正在开发用于治疗骨髓增生异常综合征、β-地中海贫血和骨髓纤维化的慢性贫血的 lussercept 。Acceeleron 公司还在推进其神经肌肉项目, ACE-083,一种局部作用的 Myostatin +剂在第2阶段的发展,在相沉积肱肌营养不良和 Charcot-Marie-Tooth 疾病,并正在进行第二阶段的肺计划,与 sotatercip 在肺动脉高压。