Anvisa suspends Novartis trials after test deaths

Anvisa suspended three Novartis clinical trials in Brazil this Monday (14). The measure follows a global suspension of studies, prompted by three patient deaths during tests of the cellular therapy rapcabtagene autoleucel, known as rap-cel or YTB323.

Severe immunological reaction triggered the halt

The deaths were caused by a severe immunological reaction called immune effector cell-associated hemophagocytic syndrome (IEC-HS). This condition is a known side effect of CAR-T therapies and can lead to fatal complications.

Which diseases were the target of the trials?

The phase 2 protocols evaluated the safety and efficacy of rap-cel in patients with granulomatosis with polyangiitis, microscopic polyangiitis, active refractory systemic lupus erythematosus, active refractory lupus nephritis, and severe refractory diffuse cutaneous systemic sclerosis.

What happens to volunteers already in the study?

The suspension prevents the enrollment of new volunteers and the initiation of new procedures but does not interrupt the clinical follow-up of participants already treated. Researchers continue to monitor patients under study.

How is Novartis responding?

The company reported that it is reviewing the cases with independent safety committees to understand why the immunological response was not controlled and to identify warning signs earlier. Rap-cel trials in cancer, such as leukemia and lymphoma, were not affected, as the safety observed in these patients remains within expectations.

What did Anvisa determine?

In a resolution published in the Official Gazette, Anvisa determined the immediate halt of human trials and the enrollment of new volunteers in the country. The measure follows the regulation of advanced therapy products, aiming for participant safety.

With information from G1, Folha de S.Paulo, Estadão.