FDA threatens criminal penalties against Novartis over faulty data used in gene therapy application

FDA 威胁对诺华(Novartis)公司在基因治疗应用中使用的错误数据进行刑事处罚

2019-08-17 01:45:06 CNBC

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The Food and Drug Administration is threatening to take criminal action against Novartis, saying Tuesday the drugmaker used data it knew was inaccurate in its application for a $2.1 million gene therapy that was approved in May. "On June 28, following the FDA's approval of the product, the agency was informed by AveXis Inc., the product's manufacturer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application and reviewed by the FDA," Peter Marks, director of FDA's Center for Biologics Evaluation and Research, said in a statement. AveXis is the gene therapy subsidiary of Novartis. The FDA said the gene therapy for spinal muscular atrophy should remain on the market, even while it's still assessing the situation. "The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties," Marks added. U.S.-traded shares of the Swiss drugmaker tumbled 3.6% in early afternoon trading. Biogen, which also produces a spinal muscular atrophy treatment, was up about 1%. In May, the FDA approved Novartis' gene therapy, making it the world's most expensive drug. The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11,000 births. In a statement, Novartis said it continues to be "fully confident in the safety, quality and efficacy of Zolgensma. "The FDA supports the continued marketing and use of Zolgensma for patients with spinal muscular atrophy (SMA) less than 2 years of age," the statement read.  "We maintain that the totality of the evidence demonstrating the product's effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile. We remain steadfast that this important treatment remain available to pediatric patients with SMA less than 2 years of age." The company added that, as noted by the FDA, "the data in question were a small portion of our overall submission and are limited to an older process no longer in use." According to Novartis, findings from the investigation did not indicate issues with product safety, efficacy or quality.  At the time the FDA approved the gene therapy, Acting FDA Commissioner Ned Sharpless lauded the approval, saying that it marked "another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases." In a tweet Tuesday, Sharpless said the agency relies "on truthful scientific data to make regulatory decisions, and we take the issue of data integrity very seriously." This story is developing. Check back for updates.
美国食品药品监督管理局(Food and Drug Administration)( FoodandDrugAdministration )威胁将对诺华( Novartis )采取刑事行动。该公司周二表示,该公司在申请210万美元基因治疗时使用的数据不准确,该申请于5月份获得批准。 "6月28日,在 FDA 批准该产品后,该机构从产品制造商 AveXis Inc .获悉了一个数据操纵问题,该问题影响了在生物制剂许可申请中提交并经 FDA 审查的动物中进行的产品测试的某些数据的准确性," Peter Marks ,FDA 生物技术评估和研究中心主任在一份声明中说。 AveXis 是诺华(Novartis)公司的基因治疗子公司。FDA 表示,尽管目前仍在评估这种情况,但脊肌萎缩症的基因疗法仍应留在市场上。 “该机构将利用其全部权力采取行动,如果适当的话,可能包括民事或刑事处罚,”马克斯补充说。 这家瑞士制药公司在美国上市的股票在下午早些时候下跌了3.6%。同时进行脊髓性肌萎缩症治疗的百健(Biogen)公司股价上涨了约1%。 五月, FDA 批准诺华(Novartis)的基因治疗,使其成为世界上最昂贵的药物。这种疗法,佐尔甘斯玛,是一种一次性治疗脊髓性肌肉萎缩,一种肌肉萎缩疾病和领先的基因导致婴儿死亡率,影响一个在每11,000个新生儿。 诺华(Novartis)在一份声明中表示,该公司继续“对佐尔甘马的安全性、质量和有效性充满信心”。 “ FDA 支持继续营销和使用 Zolgensma 的患者脊柱肌肉萎缩( SMA )不到2岁,”声明说。“我们认为,证明产品有效性和安全性的全部证据继续提供令人信服的证据,支持整体有利的利益风险状况。我们仍然坚定地认为,这种重要的治疗方法仍然适用于不到2岁的儿童 SMA 患者。” 该公司补充说,正如 FDA 所指出的,“所涉及的数据只是我们整体提交数据的一小部分,并且仅限于不再使用的较旧程序。”据诺华(Novartis)公司称,调查结果并未表明产品安全、疗效或质量方面的问题。 在 FDA 批准基因治疗的时候,代理 FDA 专员 Ned Sharples 称赞了这一批准,并表示这标志着“基因和细胞疗法治疗多种疾病的另一个里程碑”。 Sharpless 在周二的推文中表示,该机构“依赖真实的科学数据做出监管决定,我们非常认真地对待数据完整性问题。” 这个故事正在发展中。检查是否有更新。

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