The US Food and Drug Administration (FDA) has approved updates to the prescribing information for the chimeric antigen receptor ciltacabtagene autoleucel (cilta-cel; CARVYKTI). The agency reported it added a boxed warning label to the therapy for immune effector cell-associated enterocolitis (IEC-EC).
Reports from clinical trials and postmarketing data showed patients who developed IEC-EC had severe or prolonged diarrhea, abdominal pain, and weight loss weeks to months after infusion. Some IEC-EC patient cases required total parenteral nutrition and immunosuppressive therapy including corticosteroids. Fatal outcomes occurred from gut perforation and sepsis, the agency reported.
The label now includes overall survival data from the phase 3 CARTITUDE-4 trial. The trial showed statistically significant improvement with cilta-cel versus standard therapy after median follow-up of 33.6 months. This past year marked a milestone in myeloma treatment. Five-year follow-up data presented at ASCO 2025 showed that one-third of patients treated with the therapy remained myeloma-free. Clinicians are now beginning to say the word cure, once unspoken in this disease.
The FDA determined that benefits continue to outweigh risks for approved use, according to a press release from the FDA. Patients with treatment-refractory IEC-EC should be referred to gastroenterology and infectious disease specialists. Additional workup is needed to rule out T-cell lymphoma of the gastrointestinal tract, the agency said.
