The Swiss pharmaceutical firm Novartis has been asked to provide details regarding possible data inaccuracies for its human gene therapy Zolgensma – the world’s most expensive treatment.
Novartis has been accused of withholding information about manipulated data during early testing of Zolgensma, a one-time $2.1 million (CHF2.1 million) treatment for spinal muscular atrophy (SMA), the leading genetic cause of death in infants. The treatment was approved by the US Food and Drug Administration (FDA) in May.
Novartis said its subsidiary in California began investigating the alleged data manipulation for Zolgensma as soon as it learned about it. The company claims that it shared the information with the FDA as soon as it had the interim conclusions.
The chairman of the US Senate Finance Committee, Chuck Grassley, wants to have access to internal reports and to know exactly when the group became aware of the data manipulation. The Basel-based pharmaceutical giant has until August 23 to shed light on the situation. Novartis has acknowledged receipt of this request.
Despite the allegations, the FDA reported last week that it retains full confidence in the new treatment. However, it plans to take the case to court, as the pharmaceutical group was aware of the problem before receiving the green light to market it at the end of May.
“The agency will use its full authorities to take action, if appropriate, which may include civil or criminal penalties,” said an FDA statement last weekexternal link.
Novartis has blamed several employees of its subsidiary Avexis, but according to several analysts this case could cost it dearly. Zolgensma has yet to receive the green light from authorities in the European Union and Japan.