The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug.
These statements were softened somewhat when the director of the Center for Biologics Evaluation said that it is actually a small amount of data that is in dispute and that the FDA did not have concerns about the drug’s safety or efficacy.
The agency has inspected the facility that manufactures the newly-approved drug and stated that the benefits of keeping the drug on the market clearly outweigh the risks.
Novartis Has Not Been Cleared Yet
However, the director emphasized the FDA’s concern for the integrity of any data submitted and that they will continue their investigation. He also said that they would take action if it is warranted.
The claim being made against Novartis involves its submitting data on its application for the approval of a gene therapy drug. The FDA considers the data to be manipulated as it proved to be inaccurate. Novartis did not divulge the data to the FDA until after the application was approved. Therefore the agency approved the product sooner than it would have had it known otherwise.
Novartis Conducted Its Own Investigation.
After notification by the FDA, Novartis issued a statement confirming its confidence in the safety of Zolgensma. Novartis said it investigated the FDA’s allegations and claimed that the data was gathered through an older process that is no longer in use.
About Zolgensma
There is no award given for the most expensive drug in the world but if there was, with a price tag of $2.1 million, Zolgensma would be the winner.
Putting its price tag aside, Zolgensma has been successful in treating children under two years of age who have been diagnosed with spinal muscular atrophy. It is an extremely rare disease that, if untreated, causes the death of infants or requires babies to be put on life support.
Spinal muscular atrophy affects around thirty patients every month. The cause is known to be a defect in the gene that is responsible for the maintenance of motor neuron cells.
Zolgensma truly makes a life or death difference. Infants who require a ventilator can barely lift their head by the time they are a year old. But children who have undergone therapy with Zolgensma were able to walk (with some difficulty) at three or four years of age.
United Healthcare Reverses its Denials
After years of research, miraculous cures come with high price tags. Although United Healthcare initially denied two requests to cover Zolgensma, it then reversed its decision on two appeals and confirmed coverage for four more requests.
The FDA previously approved the gene therapy Luxturna by Spark Therapeutics. The drug treats a rare form of blindness. Zolgensma is now the second gene therapy to be approved by the FDA.
Do you know of anyone who prevailed in an appeal for drug coverage?
