Novartis Hid Manipulated Data While Seeking Approval for $2.1 Million Treatment


The drug maker Novartis concealed manipulated data from the Food and Drug Administration while applying for approval of an extremely expensive gene therapy treatment and then delayed reporting the issue, the agency said on Tuesday.

Officials said the inaccurate data, which involved testing in mice of two different strengths of the treatment, did not affect the safety or efficacy of the therapy, Zolgensma, used to treat a rare, often fatal genetic disease called spinal muscular atrophy.

Approved in May, the treatment’s price — set at $2.1 million — stoked concerns about the astronomical costs of potential cures for rare diseases and upset parents who initially could not get insurance coverage for the breakthrough treatment.

The F.D.A. said patients were not at risk, and that the treatment could still be sold.

But the news that a drugmaker had manipulated or mishandled data is an unsettling moment for the pharmaceutical industry. Many companies are racing to develop breakthrough gene therapy treatments for rare and intractable diseases.

And the F.D.A. has granted a record number of expedited approvals for novel drugs and treatments in recent years, and Zolgensma was among those given top priority.

Dr. Peter Marks, director of the F.D.A.’s Center for Biologics Evaluation and Research, said the drug’s approval probably would have been delayed had the agency known of the manipulated data. The public “expects us to have accurate data when we approve products,” he added.

Dr. Marks said the F.D.A. would continue to investigate and would determine whether Novartis should face civil or criminal penalties.

Shares of Novartis, a Swiss drugmaker that also makes the multiple sclerosis drug Gilenya, were down 2.77 percent, at $88.22 a share, on Tuesday.



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