But he stressed that the agency takes data integrity issues seriously and is continuing to investigate the situation. He said the FDA “will use its full authorities to take action, if appropriate, which may include civil or criminal penalties” against the manufacturer, AveXis Inc., a subsidiary of Novartis.
The $2.1 million treatment -- considered the world’s most expensive therapy — is called Zolgensma. It is used for children less than 2-years-old with spinal muscular atrophy, a leading genetic cause of infant death.
In a statement, Novartis said it is “fully confident in the safety, quality and efficacy of Zolgensma.” The drugmaker said AveXis investigated allegations of data manipulation and “once we had interim conclusions from our investigations, we shared our findings with the FDA.” The data, the company added, “are limited to an older process no longer in use.”
The FDA approved the gene therapy on May 24. On June 28, Marks said, AveXis told the agency about the data problem, which involved tests comparing older and newer versions of the gene therapy. The FDA said the company began investigating the data problem in March.
Spinal muscular atrophy is considered an extremely rare disease, affecting about 30 new patients a month. It is caused by a defective gene that is supposed to allow the body to maintain cells called motor neurons. Without a properly functioning gene, infants typically die or live on respirators and other life support.
Marks said he was most concerned that the manufacturer “submitted data that was inaccurate to us as part of their application and that led us to approve a product potentially sooner than we might have."
He added the agency has inspected the manufacturing facility used for the treatment and has “no concerns” about the process. “The benefit-risk ratio is clearly in favor of allowing the product to remain on the market,” he said.
The gene therapy has been hailed as “transformative” for children with the genetic defect. Left untreated, a child with the disease is likely to be on a ventilator and unable to raise his or her head by 1-years-old, Marks said. Children who have been treated appear normal three to four years after the therapy.
Zolgensma is the second gene therapy approved by the FDA. The first was Luxturna, made by Spark Therapeutics, which is used for a rare inherited type of blindness.