Zolgensma (onasemnogene abeparvovec-xioi) treats spinal muscular atrophy (SMA), which makes infants’ muscles waste away. Most victims of the most severe form of the disease die before their second birthday. Zolgensma is a breakthrough offering hope, but it also comes with a price tag of $2.125, making it the most expensive medicine in the world.
That alone creates drama. Throw in the fact that data were allegedly manipulated during one of the testing phases and that that discovery was kept from the FDA and you’ve got a scandal on your hands.
As Novartis CEO Vas Narasimhan, MD, is finding out. When news about the data manipulation broke a few weeks ago, Narasimhan took a somewhat defiant stand. Novartis did no wrong, he insisted, and only wanted to complete an internal investigation about what happened and then present that to the FDA. Novartis did so on June 28, but the FDA had already approved the drug on May 24. Both Novartis and the FDA say that the problem with the data does not compromise Zolgensma’s efficacy and the drug can still be sold.
Problem solved, right?
Brad Loncar, an investor who runs an exchange-traded fund of biotech companies, tells the Wall Street Journal (WSJ), that Novartis “handled this in just about the worst way possible.”
David Gortler, a former FDA official, explains that “submitting complete information to the FDA is sacrosanct to the agency.”
The newspaper reports that the “FDA has referred the Zolgensma matter to its Office of Criminal Investigations to begin a preliminary inquiry, according to a person familiar with the events. Submitting false data to the agency as part of a new-drug application could be a crime if investigators prove the actions were intentional and not an oversight.”