The FDA said it is carefully assessing the situation.
The manipulated data was used to illustrate comparability between an
early version of Zolgensma and the later version of the treatment,
which was manufactured using a different process.
The FDA does not believe that the manipulation impacts the safety or
testing surrounding the version of the drug, which treats spinal
muscular atrophy (SMA), the leading genetic cause of death in
Novartis' Avexis unit informed the FDA of the data manipulation on
June 28, the regulator said.
Novartis acquired the therapy's maker in 2018, and the drugmaker was
aware of the manipulation as early as March - more than two months
before the treatment's approval, the FDA said.
The regulator plans to take action against the company including
possible civil or criminal penalties.
In a statement on Tuesday, Novartis said it is "fully confident" in
the safety, quality and efficacy of Zolgensma.
The issue is not expected to impact the timing of its ongoing
Zolgensma regulatory filings and development programs, the drugmaker
"The data in question were a small portion of our overall submission
and are limited to an older process no longer in use."
Zolgensma - the world's most expensive drug - was approved as a
one-time treatment for SMA in late May.
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The disease often leads to paralysis, breathing difficulty and death
within months for babies born with the most serious Type I form. SMA
affects about one in every 10,000 live births, with 50% to 70%
having Type I disease.
The FDA said its concerns were currently limited to a small portion
of product testing data included in the marketing application for
According to an inspection note released by the agency, there were
discrepancies in some of its mouse survival data results.
The manipulated data does not change the FDA's positive assessment
of information from human clinical trials, it said. The agency said
it will continue to evaluate the integrity of the product testing
data used in the development of Zolgensma's manufacturing process.
U.S.-listed shares of Swiss drugmaker Novartis closed down 2.8% at
$88.20 on Tuesday. Shares of Biogen Inc, which makes rival SMA
treatment Spinraza, closed up 2.1% at $240.17 and shares of PTC
Therapeutics Inc, which is also developing an SMA treatment, closed
4.9% higher at $44.75.
(Reporting by Michael Erman and Manojna Maddipatla; editing by Bill
Berkrot, Marguerita Choy and Cynthia Osterman)
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