Brazil's National Health Surveillance Agency, known as Anvisa, has suspended three experimental clinical trials run by pharmaceutical company Novartis for autoimmune diseases, after three people died during the studies. The measure was published in a resolution in the Diario Oficial da Uniao, Brazil's official government gazette, on Tuesday.
The trials had already been paused by Novartis itself earlier this month, following the three deaths among participants. The company said the cases were being evaluated as part of a safety review of the trials.
What the therapy was being tested for
The suspended trials involved rapcabtagene autoleucel, also known as rap-cel or YTB323, an experimental cell therapy being tested against autoimmune diseases. One of the studies assessed the therapy's efficacy and safety in participants with severe active granulomatosis with polyangiitis or microscopic polyangiitis. Another targeted patients with active refractory systemic lupus erythematosus or active refractory lupus nephritis.
According to Novartis, the patients who died experienced severe reactions linked to immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome, known as IEC-HS. The company said at the time that this condition is a known side effect of CAR-T cell therapies and can cause potentially fatal complications. CAR-T therapies work by re-engineering a patient's own immune cells to target disease, and have become a prominent tool in treating certain cancers and, more recently, autoimmune conditions.
Anvisa's resolution does not specify which occurrences prompted the suspension, stating only that the trials were halted as a safety measure.
Novartis responds
Responding to CNN Brasil, Novartis said it had decided to temporarily halt, at a global level, the screening, randomization and treatment of patients in clinical trials of rapcabtagene autoleucel for autoimmune diseases in the immunology and neuroscience fields. The company said this preventive and voluntary decision was duly reported to Anvisa in accordance with current regulations.
Novartis said the measure followed notification of three serious adverse events that resulted in death outside Brazil. The company said it remained in ongoing dialogue with the independent data monitoring committees and steering committees for the studies, as well as with local and international health authorities, to carry out a detailed analysis of the safety data and define additional measures to strengthen protection for participants.
Novartis added that its rapcabtagene autoleucel program in oncology has not been affected by the suspension, though that study is not being conducted in Brazil. The company stressed that no Brazilian patient died during the clinical trials in the country, and reiterated that patient safety and wellbeing remain an absolute priority for the company.
