Novartis AG failed to notify the U.S. Food and Drug Administration about inaccurate data for its gene therapy Zolgensma while the agency was reviewing the drug for approval, even though the Swiss pharmaceutical giant had known as early as mid-March.
"We tried to do the right thing in this instance," Novartis CEO Vas Narasimhan said Aug. 7.
As the drug was being reviewed, Novartis conducted an independent investigation with external counsel to validate the data manipulation claims. In early May, the investigation confirmed that a "small number" of scientists at AveXis had manipulated a test in mice during preclinical work to achieve certain outcomes.
Zolgensma, a $2.1 million gene therapy developed by AveXis Inc. for rare infant disorder spinal muscular atrophy, was approved by the FDA on May 24. Novartis acquired AveXis in April 2018.
In other Novartis news, Narasimhan told S&P Global Market Intelligence in an exclusive interview that RNA technologies may become a new research focus for the Basel, Switzerland-based pharmaceutical giant.
"We're still trying to decide how best to engage," the Novartis CEO said. "There are lots of things in preclinical research, but nothing I can say is at the point where it's mature enough at the moment."
Novartis says it 'tried to do the right thing' amid FDA gene therapy data probe
Novartis, under fire from the U.S. FDA due to preclinical data manipulation of its now-approved gene therapy Zolgensma, said it "tried to do the right thing" and "proactively" informed regulatory agencies — but only after the drug was FDA-approved.
US FDA opens probe into Novartis' gene therapy Zolgensma over data inaccuracy
The U.S. FDA has opened a probe into Novartis' Zolgensma after the company's drug manufacturing unit reported a data manipulation issue related to animal testing that was done to support the gene therapy's approval.
Novartis CEO eyeing RNA landscape for M&A to expand research 'gameboard'
"I think now you're starting to see, sort of like gene therapy, a maturation of the entire RNA space," Narasimhan, the Swiss drug giant's CEO, said in an interview.
Chart of the week
Story: Healthcare 2nd-worst performer among S&P 500 sectors in July
A nurse handles blood samples taken from a patient for use in CAR-T cell therapy at the Fred Hutchinson Cancer Research Center in Seattle.
Source: The Associated Press
Medicare agency accepts Novartis, Gilead gene therapies for cancer treatment
Medicare recipients will now have access to chimeric antigen receptor T cell therapies for cancer after the Trump administration cleared a final rule accepting the medicines for use in the program that provides health insurance to people with disabilities and those over 65.
US cost watchdog proposes framework changes to address cell, gene therapy prices
A U.S. pricing watchdog, the Institute for Clinical and Economic Review, has proposed changes to its evaluation process to address cell and gene therapies that could be one-time "cures" for certain diseases.
Big pharma slams US Senate drug pricing bill, outlines exposure during Q2 calls
Pharma executives have little love for the Senate Finance Committee's drug pricing legislation, with Pfizer Inc. and Bristol-Myers Squibb Co. calling the bill package "punitive" for innovators in the industry.
Of Mice Not Men: New allergy-driving T cell; an eye-on-a-chip; hepatitis B in shrews
In this week's "Of Mice Not Men," scientists from various institutions identified T cells that could drive allergic reactions, engineered an eye-on-a-chip model that replicates the human eye and discovered a new strain of hepatitis B virus in shrews.
China's tougher rules for tainted vaccines market to boost larger drugmakers
Chinese regulators approved fewer vaccines in the first half of 2019 after a series of scandals over poor-quality inoculations rocked the industry and led to the passing of a tougher vaccine law earlier this year.
Healthcare companies are reporting second quarter earnings, with Merck KGaA, Allergan PLC and Becton Dickinson and Co. in the spotlight this week. The S&P Global Market Intelligence Healthcare news team takes a look at companies' performance, and the remaining half to come.
Merck KGaA looks to tripling sales of Mavenclad as Rebif declines in MS
The Darmstadt, Germany-based company's CEO, Stefan Oschmann, is optimistic that the declining sales of Rebif — one of the company's most profitable products — will continue to be compensated by Mavenclad.
Allergan posts growth in core products Botox, Juvederm — but profits lag
Allergan's sales from the second quarter of 2019 provide a snapshot of the kind of company AbbVie Inc. is slated to acquire by early 2020.
Becton expects growth in China, focus on plug-in M&A as balloon devices stumble
Becton saw double-digit business growth in China during the third fiscal quarter, driven by high volume sales of disposable products and a lack of competition in unique areas like biosurgery and diagnostic systems.
Zoetis beats Q2 consensus ahead of pet launches, livestock's return to normal
Zoetis Inc. reported 22% growth in its companion animal segment for the second quarter and a return to growth in livestock, despite China's continued African swine fever outbreak, and underscored upcoming first-to-market pet products.
Regeneron's Q2 sales rise 32% as Eylea, Dupixent make up for flagging Praluent
U.S. sales of Eylea grew 17% to $1.16 billion amid mounting competition from Roche Holding AG and Novartis' Lucentis in the eye disease age-related macular degeneration.
Essential Healthcare is a weekly collection of critical developments across the pharmaceutical, biotechnology, healthcare provider, healthcare technology and medical equipment industries that draws on exclusive analysis and value-added content from the Healthcare News team at S&P Global Market Intelligence. Subscribe now to get Essential Healthcare delivered to your inbox every week.