In the face of possible fines and criminal charges being leveled against his company by the FDA, Novartis CEO Vasant Narasimhan, MD, said that he wanted to give the FDA the “best information” possible about the manipulation of data involving its new drug Zolgensma.
“We made the decision to progress our quality investigation prior to informing FDA and other regulatory authorities so that we could provide the best information and technical analysis—which we did promptly on completion on June 28,” said Narasimhan. Only problem with that is that the FDA approved the gene therapy on May 24.
Narasimhan defiantly defended how Novartis handled the problem, saying that the company can “stand proud that we tried to do the right things in this instance,” as the Hill reports. “We understand the agency has a different perspective, which we respect, but we’ve tried to be transparent, thorough, science-based and, most importantly, patient-oriented to ensure that we never compromised patient safety, efficacy, or product quality during any moment during all of this.”
Zolgensma, which at $2.1 million per single-dose treatment is the most expensive drug on the market, treats spinal muscular atrophy (SMA). Both the FDA and Novartis stressed that the drug is still effective and can still be sold. SMA makes infants’ muscles waste away, and most victims of the most severe form of the disease die before their second birthday. That makes SMA the most common genetic cause of infant death.
Novartis hid or manipulated data in tests conducted on mice that were part of what the FDA considered when it approved the drug. Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research, earlier this week said that he didn’t know why the data were manipulated. Narasimhan said that the scientists involved in the data manipulation will be let go.
About 400 U.S. babies are born with SMA every year, and around 300 have the most severe version. The 700 patients who already have the disease are still eligible for the drug. Zolgensma halts SMA’s progression and even offers hope for a cure in some cases.
Novartis “alerted European and Japanese health authorities—which are in the process of evaluating Zolgensma for approval—to the problem shortly after the FDA,” the Wall Street Journal reports. ovartis said it doesn’t anticipate any regulatory delays on the back of the disclosures.”