Johnson & Johnson said Monday that the U.S. Food and Drug Administration has granted approval for a supplemental New Drug Application expanding the indication for Caplyta to include prevention of relapse in adults with schizophrenia. The decision rests on long-term clinical data assessing safety and effectiveness for this specific use.
Schizophrenia affects an estimated 2.8 million adults in the United States, and an appreciable portion of this population does not receive adequate care - roughly 40 percent, according to the clinical context provided with the approval. The condition is associated with recurrent episodes: adults living with schizophrenia average nine relapse events over a six-year period. The report accompanying the approval also cited a 2024 estimate of schizophrenia's societal cost in the U.S. at $366.8 billion.
The pivotal evidence came from a Phase 3, double-blind, randomized withdrawal study. During the 26-week double-blind treatment window, patients maintained on Caplyta showed a significantly longer time to relapse compared with those switched to placebo. Study results indicate a 63 percent lower risk of relapse for patients on Caplyta versus placebo, and 84 percent of patients treated with Caplyta remained relapse-free over six months. The drug also produced a significant delay in time to all-cause treatment discontinuation, which includes discontinuation due to relapse.
Safety observations in the trial were consistent with the existing clinical database for Caplyta, and no new safety concerns were identified in the submitted data. The most commonly reported treatment-related adverse event was headache, occurring in at least 5 percent of treated patients and at least twice the rate observed with placebo.
Short-term clinical studies in schizophrenia found Caplyta to be similar to placebo with respect to weight change, metabolic effects, and extrapyramidal symptoms. In the 6-month randomized withdrawal study, there were no clinically meaningful increases in prolactin or cardiometabolic parameters at the end of the double-blind period.
Longer-term information from a 12-month open-label extension study showed mean weight change of -2.05 kg for patients treated with Caplyta over one year, alongside sustained improvement or stability in metabolic measures.
Caplyta already held approvals in adults as an adjunctive treatment with antidepressants for major depressive disorder, and for schizophrenia and depressive episodes associated with bipolar I or II disorder as both monotherapy and as adjunctive therapy with lithium or valproate. The supplemental approval specifically addresses relapse prevention in schizophrenia based on the submitted long-term data.
Key points
- FDA approved a supplemental application for Caplyta to prevent relapse in adult schizophrenia based on long-term trial data.
- Phase 3 randomized withdrawal results showed a 63 percent reduction in relapse risk and 84 percent of treated patients relapse-free at six months, with delayed time to all-cause discontinuation.
- Safety profile aligned with prior data; headache was the most common treatment-related adverse event. Sectors affected include healthcare providers, payers, and the pharmaceutical industry.
- Treatment-related adverse events occurred - the most frequent being headache, reported in at least 5 percent of patients and at least twice the rate of placebo - which may affect tolerability and clinical decision-making.
- Despite the reduction in relapse risk, a portion of patients still experienced relapse during the study period, indicating relapse is not eliminated for all treated individuals.
- All-cause treatment discontinuation was observed in the trial population; while Caplyta delayed time to discontinuation, discontinuation for various reasons remains possible.