Dive Brief:

简报：

The Food and Drug Administration has

食品药品监督管理局已经

declined to approve

拒绝批准

Ultragenyx Pharmaceutical’s experimental gene therapy for a rare neurodegenerative disorder, citing manufacturing concerns in a complete response letter issued weeks ahead of its August 18 decision deadline.

Ultragenyx制药公司的一种罕见神经退行性疾病实验性基因疗法，因生产问题在8月18日决定期限前数周发出的完全回应信中被提及。

Ultragenyx said on Friday that the FDA had requested additional information regarding manufacturing processes at its facility. The company said the agency’s observations are “not directly related to the quality of the product,” and it believes the issues are “readily addressable.”

Ultragenyx周五表示，FDA已要求提供有关其设施生产流程的更多信息。该公司表示，该机构的观察结果“与产品质量无直接关系”，并认为这些问题“很容易解决”。

Ultragenyx plans to resubmit its drug once the issues are resolved, which would start a new six-month review clock once completed. In a client note Friday, Leerink Partners analyst Joseph Schwartz wrote that, while the news is disappointing, “it looks like this is a speed bump to approval, rather than a roadblock.” Shares for the company fell about 5% Friday, but traded up nearly 1% to open Monday trading..

Ultragenyx计划在问题解决后重新提交其药物申请，一旦完成，将启动新的六个月审查期。Leerink Partners的分析师约瑟夫·施瓦茨在周五的一份客户报告中写道，尽管这一消息令人失望，“但看起来这只是通向批准过程中的一个小阻碍，而非完全的障碍。”该公司股价周五下跌了约5%，但在周一开盘时交易上涨了近1%。

Dive Insight:

深度洞察：

The complete response letter is another hit to Ultragenyx, which just announced a

完整的回复函对刚刚宣布这一消息的Ultragenyx来说是又一次打击，

disappointing interim update

令人失望的中期更新

from a closely watched clinical trial of a drug it is developing with partner Mereo BioPharma.

来自其与合作伙伴Mereo BioPharma共同开发的药物备受关注的临床试验。

The rejected therapy, known as UX111, is designed to treat people with Sanfilippo syndrome Type A, a rare lysosomal storage disorder that can cause a range of progressive neurological symptoms and lead to early death.

被拒绝的疗法，称为UX111，旨在治疗患有A型Sanfilippo综合征的患者，这是一种罕见的溶酶体贮积症，可引起一系列进行性神经系统症状，并导致早逝。

A one-time infusion, Ultragenyx’s gene therapy is meant to deliver a functional copy of a gene called SGSH. Cells with the corrected gene can then produce the enzyme that gene encodes, which is deficient in people with Sanfilippo Type A. Ultragenyx

一次性输注，Ultragenyx的基因疗法旨在提供一个名为SGSH的功能性基因副本。携带矫正基因的细胞随后可以产生该基因编码的酶，这种酶在患有Sanfilippo A型综合症的人中是缺乏的。

licensed the therapy from Abeona Therapeutics

从Abeona Therapeutics获得该疗法的许可

in a 2022 deal.

在2022年的一笔交易中。

Ultragenyx said the FDA did not cite any issues with the clinical data submitted in its application, although the agency did ask for updated results from current study participants to be included in any resubmission. The company will work with the FDA over the next few months to resolve the manufacturing issues observed..

Ultragenyx 表示，FDA 并未对其申请中提交的临床数据提出任何问题，但该机构确实要求在任何重新提交的申请中包含当前研究参与者的最新结果。该公司将在未来几个月内与 FDA 合作，解决所观察到的生产问题。

Kristen Kluska, an analyst at Cantor Fitzgerald, also described the rejection as a disappointment, as UX111 could have been a “near-term revenue and [priority review voucher] sale opportunity,” she wrote in a client note, referring to the transferable fast passes granted by the FDA upon certain drug approvals..

康托·菲茨杰拉德分析师克里斯汀·克鲁斯卡也在一份客户报告中形容这一拒绝令人失望，她写道，UX111 本可以成为“短期内的营收和（优先审评券）销售机会”，她指的是FDA在某些药物获批时授予的可转让快速通道券。

Still, Kluska noted how most manufacturing-related complete response letters are resolved and she expects UX111 to ultimately get approved.

尽管如此，克鲁斯卡指出，大多数与制造相关的完全回应函件都得到了解决，她预计 UX111 最终将获得批准。