Ultragenyx Announces Orphan Drug Designation for UX701 for the Treatment of Wilson Disease


2020-12-10 21:30:11 BioSpace


NOVATO, Calif., Dec. 09, 2020 -- Ultragenyx Pharmaceutical Inc. , a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to UX701 for the treatment of Wilson disease, a larger rare metabolic disease that affects more than 50,000 people in the developed world. “FDA Orphan Drug Designation is an important milestone in the development of UX701, a gene therapy for the treatment of Wilson Disease, and highlights the significant unmet medical need for people with this genetic disorder,” stated Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. “Current treatment options involve the often complicated and lifetime use of medications that block the absorption of copper from the diet or remove copper by chelation. UX701 is designed to directly address the underlying cause of disease by restoring normal copper metabolism in the liver. By correcting copper trafficking and removal, this one-time treatment has the potential to better address the many serious effects of this disease and improve the lives of patients.” UX701 is currently in late preclinical development, and an investigational new drug (IND) application is expected to be filed with the FDA by the end of the year. About Orphan Drug Designation The FDA Orphan Drug Designation program provides orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received. About Wilson Disease Wilson disease is a rare inherited disorder caused by mutations in the ATP7B gene, which results in deficient production of ATP7B, a protein that transports copper. Loss of function of this copper-binding protein results in the accumulation of copper in the liver and other tissues, most notably the central nervous system. Patients with Wilson disease experience hepatic, neurologic and/or psychiatric problems. Those with liver disease can experience such symptoms as fatigue, lack of appetite, abdominal pain and jaundice, and can progress to fibrosis, cirrhosis, life-threatening liver failure and death. Wilson disease can be treated by reducing copper absorption or removing excess copper from the body using life-long chelation therapy, but unmet needs exist because some treated patients experience clinical deterioration and severe side effects. Wilson disease affects more than 50,000 individuals in the developed world. About UX701 UX701 is an investigational AAV type 9 gene therapy designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion. It has been shown in preclinical studies to normalize copper trafficking and excretion from the body. UX701 was granted Orphan Drug Designation in the United States. About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com. Ultragenyx Forward-Looking Statements Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its business plans and objectives for UX701 and future clinical developments for UX701 are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company’s clinical development programs, business and operating results, the timing of submissions to regulatory authorities and the timing and likelihood of regulatory approvals for our product candidates, the timing of clinical trial activities and reporting results from same, the availability or commercial potential of Ultragenyx’s products and drug candidates manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. 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 the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on October 27, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission. Contacts: Ultragenyx Pharmaceutical Inc. Investors & Media Joshua Higa 415-660-0951 jhiga@ultragenyx.com
加利福尼亚州诺瓦托,2020年12月9日--Ultragenyx Pharmaceutrical Inc.,一家专注于开发和商业化治疗罕见和超罕见遗传疾病的新型产品的生物制药公司,今天宣布美国食品和药物管理局已授予UX701孤儿药称号,用于治疗威尔逊病,这是一种较大的罕见代谢疾病,在发达国家影响着50,000多人。 Ultragenyx基因疗法开发部首席医疗官Eric Crombez医学博士说:“FDA孤儿药的命名是治疗威尔逊病的基因疗法UX701开发过程中的一个重要里程碑,它突出了患有这种遗传病的人尚未得到满足的重大医疗需求。“目前的治疗方案涉及经常复杂和终身使用的药物,这些药物阻断铜从饮食中的吸收或通过螯合去除铜。UX701旨在通过恢复肝脏中正常的铜代谢来直接解决疾病的根本原因。通过纠正铜的贩运和清除,这种一次性治疗有可能更好地解决这种疾病的许多严重影响,并改善患者的生活。“ UX701目前处于后期临床前开发阶段,预计年底将向FDA提交一份研究性新药(IND)申请。 关于孤儿药的命名 FDA孤儿药品指定计划为那些用于安全有效治疗,诊断或预防在美国影响少于200,000人的罕见疾病的药品和生物制品提供孤儿地位。在美国,孤儿指定的好处包括:在FDA批准后七年的市场排他性,免除或部分支付申请费,以及在收到孤儿指定后进行的临床试验费用的税收抵免。 关于Wilson病 Wilson病是一种罕见的遗传性疾病,由ATP7B基因突变引起,导致ATP7B(一种运输铜的蛋白质)产生不足。这种铜结合蛋白功能的丧失导致铜在肝脏和其他组织中的积累,最显著的是中枢神经系统。Wilson病患者经历肝脏,神经和/或精神方面的问题。肝病患者可出现乏力,食欲不振,腹痛,黄疸等症状,并可发展为纤维化,肝硬化,危及生命的肝功能衰竭和死亡。Wilson病可以通过减少铜吸收或使用终身螯合疗法去除体内多余的铜来治疗,但存在未满足的需求,因为一些接受治疗的患者经历了临床恶化和严重的副作用。在发达国家,威尔逊病影响着5万多人。 关于UX701 UX701是一种研究性AAV 9型基因疗法,其目的是在单次静脉输注后稳定表达ATP7B铜转运体。临床前的研究表明,它可以使铜的运输和排泄正常化。UX701在美国被授予孤儿药称号。 关于Ultragenyx制药公司。 Ultragenyx是一家生物制药公司,致力于为患者带来治疗严重罕见和超罕见遗传疾病的新颖产品。该公司已经建立了一个多样化的已获批准的疗法和候选产品组合,旨在解决医疗需求很高且治疗生物学明确的疾病,对于这些疾病,通常没有已获批准的治疗潜在疾病的疗法。 该公司由一个在罕见病疗法的开发和商业化方面经验丰富的管理团队领导。UltraGenyx公司的战略是基于时间和成本效益的药物开发,目标是为最紧急的患者提供安全有效的治疗。 欲了解有关Ultragenyx的更多信息,请访问该公司的网站:www.Ultragenyx.com。 Ultragenyx前瞻性陈述 除此处包含的历史信息外,本新闻稿中阐述的事项,包括与UltraGenyx关于其UX701业务计划和目标以及UX701未来临床发展的预期和预测相关的陈述,均为前瞻性陈述,符合1995年《私人证券诉讼改革法》“安全港”条款的含义。此类前瞻性陈述涉及重大风险和不确定性,可能导致我们的临床开发计划,与第三方的合作,未来结果,表现或成就与前瞻性陈述所表达或暗示的显著不同。这些风险和不确定因素包括,其中,COVID-19大流行对公司临床开发计划,业务和经营业绩的影响,向监管部门提交报告的时间以及我们的候选产品获得监管部门批准的时间和可能性,same的临床试验活动和报告结果的时间,Ultragenyx的产品和候选药物的可用性或商业潜力制造风险,来自其他疗法或产品的竞争,以及可能影响现有现金,现金等价物和短期投资充足性以资助运营,公司未来经营业绩和财务表现的其他事项。Ultragenyx不承担更新或修订任何前瞻性陈述的义务。有关可能导致实际结果与这些前瞻性陈述中所表达的结果不同的风险和不确定性的进一步描述,以及与Ultragenyx一般业务相关的风险,请参见Ultragenyx于2020年10月27号提交给证券交易委员会的10-Q表季度报告及其随后提交给证券交易委员会的定期报告。 联系人: Ultragenyx制药公司。 投资者和媒体 约书亚·希加 415-660-0951 电子邮件:jhiga@ultragenyx.com