Amid a fresh scandal, this time over data manipulation for the world’s most expensive med, top Novartis execs laid out a detailed account Wednesday and defended the company’s decision not to inform the FDA until after its own internal probe wrapped up.
In the end, CEO Vas Narasimhan said the company did the “best we could.”
After FDA announced Tuesday that Novartis had turned over details on data manipulation at its gene therapy outfit AveXis—which won approval in May for its rare disease med Zolgensma—the Swiss drugmaker rounded up a group of execs for a conference call Wednesday to offer its version of events.
According to Novartis’ timeline, an AveXis scientist stepped up in mid-March to report the manipulated data, which the FDA said was submitted as part of Zolgensma’s application for approval. The company launched an internal probe, and by early May had determined the scientist was correct. It didn’t tell the FDA about the allegations, however, until June 28, more than a month after Zolgensma won its FDA green light.
The company is in the process of “exiting the small number of AveXis scientists involved,” CEO Vas Narasimhan said during the call.
The data dust-up is the latest in a series of scandals at Novartis and the second major controversy to surface since Narasimhan took the reins. The CEO has said rehabbing the company’s reputation is one of his top goals as chief executive. But now Novartis has to not only spruce up its image, but regain trust at the FDA, which made its own grievances very public on Tuesday.
In this case, Narasimhan said the company wanted to dig up the details before sharing the problem with the FDA. The internal investigation that started in March aimed to confirm that the scientist’s claims were true and to determine whether the data problem would affect Zolgensma’s efficacy, safety or quality. The manipulated data had been attributed to a potency assay Novartis no longer used in testing Zolgensma during production, Narasimhan said.
Fast forward to early May, when the company confirmed the allegation and then launched a “full technical investigation,” Narasimhan said. He primarily got involved once the company confirmed the data manipulation, he said Wednesday.
Meanwhile, the FDA was reviewing Zolgensma’s application and approved it on May 24, without knowing about the scientist’s allegations or Novartis’ internal probe. The company launched its new drug, which treats babies two years or younger with spinal muscular atrophy, at a sticker price of $2.1 million, making it the most expensive drug in the world.
The FDA on Tuesday took an unusually harsh tone over Novartis’ conduct. Acting chief Ned Sharpless tweeted that the regulator is considering civil and criminal punishments, and the agency sent out a more detailed announcement to the media. The FDA and Novartis both say Zolgensma should remain on the market.
Facing questions from analysts on Wednesday, Narasimhan said the company has a deep commitment to rebuild public trust after numerous scandals around the world in recent years. He said it’s a “long road” that’s sometimes “bumpy,” but that in this case “we tried to do all of the right things.”
“We understand the agency has a different perspective which we can respect,” he added.
The company’s choice to inform regulators only after investigating them itself was essentially standard procedure. In any similar case, it would contact the appropriate agency after it fully understood the issue, head of regulatory affairs Rob Kowalski said on the call.
Novartis’ delay notifying the agency had nothing to do with the FDA’s ongoing drug review for Zolgensma, Narasimhan said. He said that if the company’s probe had wrapped up before the agency’s Zolgensma decision, it would’ve shared the results.
Novartis says the data manipulation incident was specific to certain scientists at AveXis who are now on the way out. Novartis bought AveXis last year to bolster its presence in gene therapy and has almost completely turned over the gene therapy company’s management team. AveXis president Dave Lennon said on Wednesday’s call. Lennon himself took the helm at AveXis in June 2018.
Novartis has also hired 1,000 new staffers for the unit, Lennon said.
“We are now in the process of exiting the small number of AveXis scientists involved in these data inaccuracies,” Narasimhan told analysts Wednesday. “We do not believe this issue extends beyond these individuals and does not impact our clinical data or gene therapy platform.”
Novartis doesn’t plan any other changes to its quality systems because they’re already “robust,” Narasimhan said. As far as changing procedures for reporting internal probes to authorities, Narasimhan declined to comment on Wednesday.
“We did the best we could but we will always learn and work to get better,” he said.
Another takeaway from the call: Zolgensma is now out of the accelerated review timeline in Europe due to a back-and-forth with regulators there.