Novartis’ Zolgensma Under Fire Again
Novartis’ gene therapy Zolgensma is again under a cloud of doubt after Nature Biotechnology retracted a 2010 article featuring early data on the treatment over data manipulation concerns.
Two years after the FDA closed its Zolgensma data manipulation investigation, Nature Biotechnology retracted its 2010 article titled, “Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN,” noting “issues” regarding data cited in a key figure in the report. Original source data provided earlier this year showed multiple inaccuracies in mouse models that were used to indicate the therapeutic promise.
The error was in a Kaplan–Meier curve that represented the survival of mice with spinal muscular atrophy that received either Zolgensma or a control scAAV9-GFP vector.
The article’s initial data showed that six mice survived for more than 250 days. However, earlier this year, the original data showed that only one of the treated mice lived.
In its retraction statement, Nature Biotechnology said its editorial team is “of the opinion that the extent of the inaccuracies in Fig. 1e and associated text undermines full confidence in the study.”
The now-retracted paper was a foundational study that led to the SMA gene therapy treatment being greenlit for in-human studies. Those clinical studies, including the pivotal Phase III STRIVE trial, showed that Zolgensma treated SMA patients with unprecedented survival rates.
The children dosed with the gene therapy saw improvements in motor function and durable milestone achievement, including an ability to sit up without support.
Nature Biotechnology noted that most researchers listed as authors of the article disagree with the decision to retract. One researcher never responded to the publication’s repeated attempts to reach her.
https://www.biospace.com/article/publication-retracts-2010-zolgensma-paper-for-data-manipulation/
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