FDA Investigates Data Manipulation Issue With Newly Approved Novartis Drug Zolgensma

FDA 调查新批准的诺华(Novartis)药物 Zolgensma 的数据操纵问题

2019-08-07 22:40:51 Benzinga

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Novartis AG 's Zolgensma, the first gene therapy approved for pediatric patients with spinal muscular dystrophy Type 1, has caught the FDA's attention due to a disclosure by the product's manufacturer about a data manipulation issue.  Flaw Identified In Animal Testing Data AveXis, which manufactures Zolgensma, informed the agency June 28 about a data manipulation issue that impacted the accuracy of certain data from product testing performed in animals submitted in the BLA for the drug, Dr. Peter Marks, the director of the FDA's Center for Biologics Evaluation and Research, said in a Tuesday letter. Zolgensma was approved by the FDA May 24. "The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market," Marks said. The concerns are limited to a small portion of the product testing data that was used to support the development of its production process for the product, the FDA said.  The manipulated data does not invalidate the information from the drug's human trials, according to the regulatory agency.  FDA: AveXis Knew Of Issue Ahead Of Drug Approval The integrity of the product testing data used in the development of the product's manufacturing process is "still a matter that we are continuing to evaluate and take very seriously," Marks said.  The FDA letter said AveXis was aware of the data integrity issue ahead of the approval of Zolgensma; the agency said it will take action, if appropriate, that may include civil or criminal penalties. Reacting to the statement from the FDA, Novartis shares slid 2.76% to $88.22 Tuesday. Novartis Backs Zolgensma  Novartis reiterated its commitment toward Zolgensma in a statement.  "First and foremost, we are fully confident in the safety, quality and efficacy of Zolgensma," the company said. The company said it will cooperate with the FDA to "appropriately update its submission and address any quality gaps identified." Novartis also does not expect the development to impact the timing of its ongoing Zolgensma regulatory filings and development programs. Novartis shares were trading higher by 0.35% at $88.53 at the time of publication Wednesday.  Related Links: Biotech Stock On The Radar: Veru, A Catalyst-Rich Biopharma The Week Ahead In Biotech: Focus On Earnings Deluge, Mid-Year Clinical Trial Readouts See more from Benzinga
诺华公司( Novartis AG )的佐尔甘斯曼( Zolgensma )是首个被批准用于儿童脊髓性肌营养不良1型患者的基因疗法,由于该产品制造商披露了一个数据操纵问题,引起了 FDA 的注意。 动物测试资料中的标记 生产 Zolgensma 的 AveXis 公司在周二的一封信中说,该公司于6月28日向 FDA 通报了一个数据操纵问题,该问题影响了 BLA 提交给该药物的动物进行产品测试的某些数据的准确性。 Zolgensma 于5月24日获得 FDA 批准。 “ FDA 正在仔细评估这种情况,并仍然有信心佐尔甘马应该继续在市场上,”马克斯说。 FDA 表示,这些担忧仅限于用于支持产品生产工艺开发的一小部分产品测试数据。 据监管机构称,这些被操纵的数据并不会使药物人体试验的信息失效。 FDA :药品批准前的常见问题 Marks 说,在产品制造过程的开发过程中使用的产品测试数据的完整性“仍然是我们继续评估和非常认真的问题”。 FDA 的信中说, AveXis 在 Zolgensma 获得批准之前就已经知道了数据完整性问题; FDA 表示将采取适当的行动,包括民事或刑事处罚。 作为对 FDA 声明的回应,诺华(Novartis)股价周二下跌2.76%,至88.22美元。 诺华(Novartis) Backs Zolgensma 诺华(Novartis)在一份声明中重申了对佐尔甘斯曼的承诺。 “首先,我们对 Zolgensma 的安全性、质量和有效性充满信心,”该公司说。 该公司表示,将与 FDA 合作,“适当更新其提交的文件,并解决任何发现的质量差距。” 诺华(Novartis)也不认为这一发展会影响其正在进行的 Zolgensma 监管申报和开发计划的时间。 诺华(Novartis)股价周三公布时上涨0.35%,至88.53美元。 相关链接: 生物技术股票的雷达: Veru ,催化剂丰富的生物制药 生物技术未来一周:专注于收益欺诈,年中临床试验准备 请参阅 Benzinga

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