The Food and Drug Administration (FDA) on Tuesday said that a gene therapy maker for babies kept quiet about the data manipulations which caused inaccuracies and was informed to the federal regulators only after the treatment was approved.
AveXis which is a subsidiary of Novartis, submitted the manipulated data for Zolgensma. This treatment has faced many criticisms for its very high cost of $2.1 million. As per the National Institute of Neurological Disorders and Stroke, the disease is an inherited deformity arising from defective gene that causes loss of nerve cells and affects the muscles which help in talking, walking, swallowing and even breathing. Zolgensma is a one-time treatment which works by keeping a working copy of that particular gene within the nerve cells before their death and the symptoms begin to develop.
The director of Center for Biologics Evaluation and Research of the FDA, Dr. Peter Marks said that the agency will take proper action against AveXis and may include either civil or criminal punishments as they informed the FDA of the issue only after getting approval for Zolgensma. Marks said that the agency was sure that Zolgensma will remain in the market and said that even though the inaccuracies constituted only a minor portion of the testing data, still accurate and truthful submission of data was essential for protecting public health and that the law asks for it. It was in the month of May that Zolgensma was approved and FDA was informed only after a month.
Novartis, on Tuesday said in their statement that they would stand behind their multimillion-dollar therapy approved in children below the age of 2 years who have spinal muscular atrophy. The company said that its subsidiary, AveXis had voluntarily disclosed to FDA and other health authorities that the previously submitted data had some inaccuracies. Dr. Ned Sharpless, acting commissioner of FDA said that the approval of this therapy would become a milestone in cell and gene therapies.