Controversies

In the months leading up to the medication's approval by the FDA, a whistleblower informed Novartis that certain studies of the medication had been subject to data manipulation. Novartis fired two AveXis executives it determined responsible but informed the FDA of the data integrity issue only in June 2019, a month after the drug's approval. The delay drew strong condemnation of the FDA. In October 2019, the company admitted to not having informed the FDA and the European Medicines Agency (EMA) for seven months about toxic effects of the intravenous formulation observed in laboratory animals. Due to data manipulation issue, the EMA withdrew their decision to allow an accelerated assessment of the medication.

In December 2019, Novartis announced that it would donate 100 doses of onasemnogene abeparvovec per year to children outside the US through a global lottery. The decision, which has been claimed by Novartis to be based on a recommendation by unnamed bioethicists, was received with much criticism by the European Commission, some European healthcare regulators and patient groups (e.g., SMA Europe or the UK's TreatSMA) who see it as emotionally burdening, suboptimal, and ethically questionable. Novartis did not consult with families or doctors before announcing the scheme. Alan Regenberg, a bioethicist at Johns Hopkins Berman Institute of Bioethics, said that the scheme was perhaps the best available since it may be impossible to reliably establish prognosis for children under two years of age.

https://en.wikipedia.org/wiki/Onasemnogene_abeparvovec