The maker of a gene therapy for babies did not inform federal regulators about 'data manipulation that created inaccuracies' until after its treatment was approved, the US Food and Drug Administration said
The maker of a gene therapy for babies did not inform federal regulators about"data manipulation that created inaccuracies" until after its treatment was approved, the US Food and Drug Administration said Tuesday.
The manipulated data was submitted by AveXis, a subsidiary of drugmaker Novartis, for the spinal muscular atrophy therapy Zolgensma, the FDA said. The treatment has been criticized for its sky-high cost: $2.1 million.Because AveXis didn't inform the FDA of the issue until after Zolgensma was approved, the agency will"use its full authorities to take action, if appropriate, which may include civil or criminal penalties," said Dr.
The disease is an inherited disorder stemming from a defective gene that leads to the death of nerve cells, affecting the muscles that allow people to walk, talk, breathe and swallow, according to the National Institute of Neurological Disorders and Stroke.As many as 1 in 8,000 babies is estimated to be affected by one of the genetic mutations that causes it. Zolgensma, a one-time treatment, places a working copy of that gene into nerve cells before they die and symptoms develop.
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