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Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx 向美国FDA提交了生物制品许可申请,申请针对A型桑菲利普综合症(MPS IIIA)的UX111 AAV基因治疗。
GlobeNewswire ·  12/19 21:00

If approved, UX111 would be the first approved therapy in the U.S. for Sanfilippo Syndrome Type A

如果获得批准,UX111将是美国首个批准治疗A型Sanfilippo综合症的疗法。

NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA).

加利福尼亚州诺瓦托,2024年12月19日(环球新闻)-- Ultragenyx Pharmaceutical Inc.(纳斯达克:RARE)今天宣布已向美国食品药品监督管理局(FDA)提交了一份生物制品许可申请(BLA),请求加速批准UX111(ABO-102)AAV基因疗法,作为治疗A型Sanfilippo综合症(MPS IIIA)患者的方案。

"The path to get a treatment to the point of a BLA filing has been long and perilous for the Sanfilippo community. They have had to watch their children, once thriving, lose their ability to speak and walk, and eventually die, while research programs were shelved due to regulatory and funding hurdles," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We commend the FDA's detailed evaluation and acceptance of cerebral spinal fluid (CSF) heparan sulfate (HS) as a well-characterized biomarker to support an accelerated approval pathway for mucopolysaccharidoses (MPS) disorders, including Sanfilippo syndrome. The FDA's acceptance of CSF HS, which we define as a disease-cause biomarker since it measures the underlying disease, enabled us to file our BLA and may unlock the future accelerated approvals of a host of new therapies for these devastating MPS diseases that affect the brain."

Ultragenyx的首席执行官兼总裁Emil D. Kakkis万.D.博士表示:“将治疗推进到BLA申请的道路对Sanfilippo社区来说既漫长又危险。他们不得不目睹曾经健康的孩子失去说话和行走的能力,最终死去,而研究项目由于法规和资金障碍被搁置。我们赞扬FDA对脑脊液(CSF)肝素硫酸盐(HS)作为支持加速批准通道的良好特征生物标志物进行了详细评估和接受。这意味着FDA接受CSF HS,我们将其定义为一种疾病原因生物标志物,因为它可以测量潜在疾病,使我们能够提交BLA,并可能为这些影响大脑的MPS致命疾病带来一系列新的治疗的未来加速批准铺平道路。”

Earlier this year, Ultragenyx reached agreement with the Agency that CSF HS can be used as a surrogate endpoint for accelerated approval based on the body of data presented by the company, along with a consortium of academics and other industry sponsors at a workshop hosted by the Reagan-Udall Foundation for the FDA in February 2024.

今年早些时候,Ultragenyx与FDA达成一致,认为CSF HS可以作为根据公司及其与一群学术界和其他行业赞助商在2024年2月由Reagan-Udall基金会为FDA主办的研讨会上所提供的数据支持的加速批准的替代终点。

The BLA submission for UX111 is supported by available data, including from the ongoing pivotal Transpher A study, demonstrating treatment with UX111 resulted in rapid and sustained decreased levels of HS in CSF in patients with Sanfilippo syndrome type A, and that sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development compared to the decline observed during the same period of time in natural history data. The most frequently reported treatment-related adverse events to date were elevations in liver enzymes, and the majority of these events were mild (Grade 1) or moderate (Grade 2) in severity and all resolved.

UX111的BLA提交得到了可用数据的支持,包括来自正在进行的关键Transpher A研究的数据显示,UX111治疗导致A型Sanfilippo综合症患者的CSF中的HS水平迅速且持续下降,并且随时间的推移,CSF HS暴露的持续减少与与同一时间段自然历史数据中观察到的认知发展下降相比有改善相关。迄今为止,最常报告的相关治疗不良事件是肝酶升高,这些事件中的大多数程度为轻度(1级)或中度(2级),所有事件均已解决。

About UX111
UX111 is a novel in vivo gene therapy in Phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment that primarily affects the brain. UX111 is designed to be dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of heparan sulfate, a glycosaminoglycan, in the brain that results in progressive cell damage and neurodegeneration. The UX111 program has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., and PRIME and Orphan medicinal product designations in the EU.

关于UX111
UX111是一种新型的体内基因治疗,正处于针对A型桑菲利普综合症(MPS IIIA)的1/2/3期开发中。这是一种罕见的致命溶酶体储存疾病,目前尚无批准的治疗方法,主要影响大脑。UX111旨在通过一次静脉输注的方法使用自我互补的AAV9载体向细胞传递SGSH基因的功能拷贝。该疗法旨在解决导致肝素硫酸异常积累的SGSH酶缺乏问题,肝素硫酸是一种糖胺聚糖,积累在大脑中导致细胞损伤和神经退行性变。UX111项目在美国已获得再生医学先进疗法、快速通道、罕见儿科疾病和孤儿药物的认证,在欧盟获得了PRIME和孤儿药物产品认证。

About Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the brain and is characterized by rapid neurodegeneration, with onset in early childhood. Children with MPS IIIA present with global developmental delay which eventually leads to progressive language and cognitive decline, behavioral abnormalities and early death. MPS IIIA is estimated to affect approximately 3,000 to 5,000 patients in commercially accessible geographies with a median life expectancy of 15 years. MPS IIIA is caused by biallelic pathogenic variants in the SGSH gene that lead to a deficiency in the sulfamidase (SGSH) enzyme responsible for breaking down heparan sulfate, a glycosaminoglycans, which accumulate in cells throughout the body resulting in the observed rapid neurodegeneration that is associated with the disorder.

关于A型桑菲利普综合症(MPS IIIA)
A型桑菲利普综合症(MPS IIIA)是一种罕见的致命溶酶体储存疾病,目前尚无批准的治疗方法,主要影响大脑,并且以快速的神经退行性变为特征,通常在儿童早期发病。患有MPS IIIA的儿童表现出全球发育迟缓,最终导致逐渐的语言和认知能力下降、行为异常和早逝。MPS IIIA估计影响大约3,000到5,000名在商业可及地区的患者,平均预期寿命为15年。MPS IIIA是由SGSH基因中的双等位致病变异引起的,导致负责分解肝素硫酸的硫酰酶(SGSH)缺乏,肝素硫酸是一种糖胺聚糖,广泛积聚在细胞中,导致与该疾病相关的快速神经退行性变。

About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.

关于Ultragenyx Pharmaceutical
Ultragenyx是一家生物制药公司,致力于为患者提供新型疗法,以治疗严重的罕见和超罕见遗传疾病。该公司建立了一个多样化的已批准药物和治疗候选者的组合,旨在解决具有高未满足医疗需求和明确生物学特征的疾病,这些疾病通常没有批准的疗法可以治疗其根本病因。

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.

该公司由一支有丰富罕见病治疗药物开发和商业化经验的管理团队领导。Ultragenyx 的策略基于及时高效的药品研发,旨在以最大的紧迫性向患者提供安全有效的治疗。

For more information on Ultragenyx, please visit the company's website at: .

欲了解更多有关Ultragenyx的信息,请访问公司的官方网站:。

Forward-Looking Statements and Use of Digital Media
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 future operating results and financial performance, business plans and objectives for UX111, expectations regarding the tolerability and safety of UX111, and future clinical and regulatory developments for UX111 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 uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, the ability of the company to successfully develop UX111, the company's ability to achieve its projected development goals in its expected timeframes, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company's behalf, smaller than anticipated market opportunities for the company's products and product 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, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.

前瞻性声明和数字媒体的使用
除本文所含的历史信息外,本新闻稿中所述事项,包括与Ultragenyx的预期和预计相关的关于其未来运营结果和财务表现、业务计划和UX111的目标、对UX111的耐受性和安全性的期望,以及UX111的未来临床和监管进展的声明,均属于1995年《私人证券诉讼改革法案》“安全港”条款内的前瞻性声明。这些前瞻性声明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声明中所表达或暗示的存在显著差异。这些风险和不确定性包括但不限于临床药物开发的不确定性、获得监管批准的不可预测性和漫长流程、公司成功开发UX111的能力、公司在预期时间框架内实现其预期开发目标的能力、与不良副作用相关的风险、依赖第三方合作伙伴代表公司进行某些活动的风险、公司产品和产品候选者的市场机会低于预期、制造风险、来自其他疗法或产品的竞争,以及其他可能影响现有现金、现金等价物和短期投资充足性以资助运营、公司未来运营结果和财务表现、临床试验活动的时间安排和结果报告、以及Ultragenyx产品和药物候选者的可用性或商业潜力等事项。Ultragenyx不承担更新或修订任何前瞻性声明的义务。

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 (SEC) on November 6, 2024, and its subsequent periodic reports filed with the SEC.

有关可能导致实际结果与这些前瞻性声明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2024年11月6日向证券交易委员会(SEC)提交的10-Q季度报告及其后续定期报告。

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx's Investor Relations website () and LinkedIn website ().

除了在SEC提交的文件、新闻稿和公开电话会议外,Ultragenyx还利用其投资者关系网站和社交媒体渠道发布重要信息,包括可能被视为对投资者重要的信息,并遵守其根据FD法规的披露义务。关于Ultragenyx的财务和其他信息定期发布,并可在Ultragenyx的投资者关系网站()和LinkedIn网站()上访问。

Contacts
Ultragenyx Pharmaceutical Inc.

联系人
Ultragenyx Pharmaceutical Inc。

Investors
Joshua Higa
+1-415-475-6370
ir@ultragenyx.com

投资者
约书亚·比嘉
+1-415-475-6370
ir@ultragenyx.com

Media
Carolyn Wang
+1-415-225-5050
media@ultragenyx.com

媒体
卡罗琳·王
+1-415-225-5050
media@ultragenyx.com


译文内容由第三方软件翻译。


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