FDA says faulty data was used to approve new gene therapy

WASHINGTON — The Food and Drug Administration said Tuesday that data a Novartis subsidiary used as part of its application for a recently approved gene therapy was manipulated, and the manufacturer didn’t tell the agency about the inaccuracies before the treatment was cleared.

However, the agency said, the $2.1 million treatment — considered the world’s most-expensive drug — should stay on the market. The therapy, called Zolgensma, is for children less than two years old with spinal muscular atrophy, a leading genetic cause of infant death.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said the inaccurate information involved mouse data and was a “small amount of the totality” that was submitted. The faulty data didn’t raise concerns about the safety and effectiveness of the treatment, he said.

But he added 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.

Novartis and AveXis didn’t immediately respond to requests for comment.

The FDA approved the gene therapy May 24. On June 28, Marks said, AveXis told the agency about the data problem. Marks said the manipulated data involves tests comparing older and newer versions of the gene therapy.

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 their first birthday, 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.