NEW YORK, Aug 7 (Reuters) - Drugmaker Novartis on Wednesday acknowledged that there were discrepancies with data it had submitted to regulators for approval of its more than $2 million gene therapy Zolgensma, but delayed notifying authorities until it completed its internal investigation.
Novartis faces possible civil or criminal penalties from the U.S. Food and Drug Administration, which said on Tuesday that some data was manipulated from early testing of Zolgensma, the world’s most expensive drug. The FDA said the company was aware of the problems for as much as two months before the drug’s approval.
“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,” Novartis Chief Executive Officer Vasant Narasimhan said on a conference call with analysts.
Narasimhan said the decision to delay notifying regulators was not due to the timing of the drug’s approval process.
The FDA said on Tuesday it did not believe the manipulation affects the safety or later testing of the drug and it believes the treatment should remain on the market. The manipulated data was used to illustrate comparability between an early version of Zolgensma and the later version of the treatment, which was manufactured using a different process.
Zolgensma was approved in late May as a one-time treatment for spinal muscular atrophy (SMA), the leading genetic cause of death in infants..
The disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious Type I form. SMA affects about one in every 10,000 live births, with 50% to 70% having Type I disease. (Reporting by Michael Erman; Editing by David Gregorio)