US Senate seeking answers from Novartis on Zolgensma data manipulation issues

Novartis has been asked by the US Senate Finance Committee to provide details regarding possible data manipulation for its Zolgensma (onasemnogene abeparvovac-xioi) gene therapy, which was approved in May for certain patients with spinal muscular atrophy. Senate committee chairman Chuck Grassley has given Novartis until August 23 to provide access to internal reports and to explain when the drugmaker became aware of the issues in question. 

Earlier this month, the FDA stated that some data submitted as part of Novartis' submission seeking approval of the $2.1-million gene therapy may have been manipulated, resulting in possible "inaccuracies" in the filing. Nevertheless, the agency said it believes Zolgensma should remain on the market as its concerns are related to only a "small portion" of product testing data. However, the FDA indicated that it may seek criminal or civil penalties from the drugmaker as the agency was not informed of the problem until June 28, even though Novartis' AveXis unit knew about it prior to Zolgensma's approval. 

Meanwhile, Grassley is also requesting information regarding how many employees were terminated as a result of the issue. Novartis has blamed several workers of its AveXis unit, which it acquired last year for $8.7 billion to gain rights to Zolgensma, formerly known as AVXS-101. The drugmaker stated that it has received the communication from Grassley and is reviewing the request for more information on the matter. 

Last week, CEO Vas Narasimhan acknowledged that the company became aware of the potential data manipulation issue in March, but delayed informing the agency until it had completed an internal investigation (for related analysis, read ViewPoints: Data manipulation an optical nightmare for Novartis

Earlier this week, some US lawmakers called on the FDA to pursue civil, criminal and regulatory action against Novartis regarding the alleged data manipulation. Meanwhile, the Institute for Clinical and Economic Review, which conducted an analysis of Zolgensma's anticipated cost before Novartis eventually settled on the therapy's $2.1-million price tag, said it is "deeply concerned" about the data integrity allegations and is "monitoring this situation closely." 

For related analysis, see ViewPoints: Novartis' Zolgensma sets the new status quo. See also ViewPoints: Payers hopeful that Zolgensma may spark new thinking in gene therapy cost models.  

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