AveXis, a startup company with an awing success story, has now been cited by the FDA for manipulating data that contributed to their miracle drug’s approval.

AveXis was acquired in 2018 by renowned pharmaceutical giant Novartis for $8.7 billion—no small sum of change. No less impressive is their miracle drug Zolgensma: a cure for babies with a deadly neurological disorder (spinal muscular atrophy) that was also classified as the world’s most expensive drug on the market by May 2019. Just one infusion of this gene therapy costs $2.125 million.

Why the Rush?

With billions of dollars of investments at stake and pressure from potential patients and investors, AveXis was driven to move quickly towards a marketable drug therapy. But in the hurry to fulfill the drug’s promise, AveXis manipulated data that was submitted to the FDA in their application for approval, including misstated dosing figures from early-stage trials.

Since the FDA’s citation, both AveXis and Novartis have acknowledged the data manipulation, which occurred before Novartis purchased the company. Novartis did, however, learn of the mistake while the FDA was reviewing Zolgensma and neglected to inform the FDA until after it was approved.

Zolgensma is one of the first commercially available gene therapies and is considered a revolutionary treatment for SMA. It is delivered intravenously in a single dose and had promising research results. The drug’s first clinical trials were conducted in 2014 and 2015, where 15 babies were treated. 12 of the babies were treated with a higher dosage level and have since made it past their second birthday, a major milestone for those with SMA.


As reported by the Wall Street Journal, “Scientist Brian Kaspar and other researchers at Nationwide Children’s Hospital in Columbus, Ohio, had been experimenting with a possible gene-therapy treatment for SMA for years. In 2013, AveXis licensed the technology, eventually bringing on Dr. Kaspar as scientific founder.” However, in 2016, employees found that Dr. Kaspar’s calculations were imprecise and notified executives. Kaspar was later replaced in the wake of the data-manipulation issue.

Additional issues were found during the FDA’s probe into the data manipulation, such as incomplete lab records and the use of an uncertified laboratory for patient testing.

Staying on the Shelf

The FDA has allowed the drug to stay on the market, saying the data manipulation doesn’t affect its efficacy or safety. In addition, the FDA official on the call said the agency would have taken the treatment off the shelf if it had any doubts. After all, dozens of babies have been successfully treated with Zolgensma.

As relieving as it is for patients to hear that Zolgensma is proven to be safe and effective, we wonder—why was the data manipulated in the first place? Why lie? The numerous mistakes made by AveXis and Novartis have definitely caused worry and not in any way helped the controversies already surrounding the field of gene therapy. And because Novartis waited until Zolgensma’s approval to disclose the data manipulation to the FDA, we question the moral fabrics of their organization. Their dismissal of Dr. Kaspar may also prove to be a cause of concern.

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Source: The Wall Street Journal

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