·  Legal News, Analysis, & Commentary

Health & Medicine

Novartis Provided False Data to Win Zolgensma Approval, FDA Says

— August 16, 2019

Novartis manipulated data to win approval for its spinal muscular atrophy treatment.

The Food and Drug Administration (FDA) has announced that Novartis manipulated data when applying for approval of Zolgensma, an expensive gene therapy treatment and then delayed reporting the issue.  However, officials said the inaccurate data, involving lab testing in mice of two different strengths of the treatment, “did not affect the safety or efficacy of the therapy” and “patients are not at risk…the treatment can still be sold.”  Zolgensma is used to treat a spinal muscular atrophy.

Spinal muscular atrophy, a genetic disease, in its most severe form, results in many infant mortalities before two years of age.  In 2016, the agency approved Spinraza.  However, the medication must be taken for the rest of the patient’s life.  Zolgensma is a one-time treatment and, in clinical trials, it appeared to halt the progress of the disease.

Because Novartis concealed the data from the agency, it approved the treatment in May and its sticker price, $2.1 million, infuriated patients and advocates for affordable and accessible medical care.

Novartis Provided False Data to Win Zolgensma Approval, FDA Says
Photo by Trust “Tru” Katsande on Unsplash

Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said the drug’s approval “probably would have been delayed had the agency known about the deception.  The public expects us to have accurate data when we approve products,” he said, adding the agency “would continue to investigate and would determine whether Novartis should face civil or criminal penalties.”

Zolgensma was the second gene therapy to be approved by the FDA following behind Luxturna, a treatment for blindness.  Many more approvals are expected to follow in the coming years.  By 2025, the agency said it “expects to approve 10 to 20 new gene therapies a year.”

The FDA revealed it was notified of the data manipulation problem on June 28, more than one month after Zolgensma was approved and in the market.  An inspection report dated July 24 to Aug. 2, 2019 noted “lapses and discrepancies in record-keeping” by the company, and “improper procedures in quality control in gathering data on the mice.”  In some cases, “records stating how long the mice lived were different from the actual value,” and in four cases “discrepancies of greater than one day were noted (ranging from 2 to 19 days).”

Erin Kelly, a spokesperson for the patient group Cure SMA, said, “We fully trust the regulators to address this issue.  Cure SMA will continue to support our families and urge them to work with their clinicians to determine the best course of treatment.”

“It’s unclear to us, at this point, exactly why this occurred,” Dr. Marks said.  It was clear that the data had been modified, but he added that in cases like these, “the motive is not always clear.”

“Many times, people do things for stupid reasons, because if they would have left well enough along, everything would have been okay,” he said.

Often, a rush to get a new, much-needed drug to market before the competition is the cause.  Pharmaceutical companies are continually competing to offer the latest and greatest patient treatments for rare diseases and seeking to secure approvals as quickly as possible to maximize profits.


FDA Says Some Preclinical Data was Manipulated for Novartis’ $2.1 Million Gene Therapy Drug

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

Join the conversation!