Key Takeaways
- Caplyta shows strong efficacy in relapse prevention: Phase III trial data demonstrated a 63% reduction in relapse risk and significantly prolonged time to relapse versus placebo in patients with schizophrenia.
- Favorable safety profile supports long-term use: No new safety signals were identified with Caplyta, with headache as the only adverse event occurring at ≥5% and twice the rate of placebo.
- Significant unmet need in schizophrenia care: With up to 2.8 million US adults affected by schizophrenia and only 40% receiving adequate treatment, Caplyta may help fill a critical treatment gap.
Johnson & Johnson announced that it has submitted a supplemental New Drug Application (sNDA) to the FDA, seeking an expanded indication for Caplyta (lumateperone) to include relapse prevention in schizophrenia. According to the company, the sNDA is based on positive data from a Phase III randomized withdrawal trial, which demonstrated that Caplyta significantly prolonged time to relapse compared to placebo.1
How Effective is Caplyta in Preventing Schizophrenia Relapse?
“For people living with schizophrenia, relapses can be devastating as they disrupt lives, undo hard-earned treatment progress toward patients’ goals, and increase the risk of hospitalization with each episode,” said Christoph U. Correll, MD, clinical professor of psychiatry, Zucker School of Medicine, Hofstra/Northwell, New York, in a press release. “Caplyta substantially lowers the chance of relapse for patients compared to placebo, which is often a major source of anxiety and suffering for them and their families.”
Phase III Trial Results Support Expanded Indication
- The double-blind, multicenter, placebo-controlled, randomized withdrawal trial evaluated the safety and efficacy of Caplyta compared to placebo.
- The primary endpoint of the trial was time to relapse during the 26-week double-blind treatment phase. The key secondary endpoint was delayed time to all-cause discontinuation, including relapse.
- Results show that time to relapse was significantly longer in patients receiving Caplyta compared to those receiving placebo (p=0.0002). Patients treated with Caplyta demonstrated a 63% reduction in risk of relapse compared to placebo (hazard ratio [95% CI] = 0.37, [0.22, 0.65]).
- Caplyta also demonstrated a significantly delayed time to all-cause discontinuation compared to placebo during the double-blind phase (p=0.0007).
- Headache was the most common adverse event, occurring in at least 5% of patients and at twice the rate seen with placebo.
- The safety profile of Caplyta was consistent with previous studies and no new safety signals were identified.1
Global and US Schizophrenia Burden Underscores Unmet Need
According to the World Health Organization (WHO), an estimated 24 million people are affected by schizophrenia globally, which is equal to approximately one in every 222 people. Those affected by schizophrenia are two to three times more likely to die prematurely than the general population, primarily due to physical illnesses such as cardiovascular, metabolic, and infectious diseases. Onset is most common between late adolescence and the twenties, with men typically experiencing earlier onset than women.
Currently, half of patients in mental health facilities are living with schizophrenia; however, only 31.3% of people with schizophrenia receive specialist mental health care. WHO suggests that mental health facilities are not currently providing effective care for patients with schizophrenia.2
According to the National Alliance on Mental Illness, the prevalence of schizophrenia in the United States is estimated to be between 0.25% and 0.64%.3 According to Johnson & Johnson, this equates to 2.8 million adults, with around 40% receiving proper medical care. It is also estimated that patients with schizophrenia experience around nine relapses over the course of six years.1
Long-Term Data Reinforce Caplyta’s Role in Neuropsychiatric Care
“Relapse prevention is a critical goal for the long-term care and management of this debilitating disorder,” said Bill Martin, PhD, global therapeutic area head, neuroscience, Johnson & Johnson Innovative Medicine, in the press release. “These Phase III results provide compelling evidence of meaningful relapse prevention, which is critical in preserving long-term patient stability, breaking the cycle of hospitalization, and helping to control symptom progression. We’re committed to building on the decade of research reinforcing the robust efficacy, proven safety, and favorable tolerability of Caplyta and providing additional data to support the long-term use of this medicine in neuropsychiatric disorders.”
References
1. Supplemental new drug application submitted to U.S. FDA for CAPLYTA® (lumateperone) with data demonstrating significant schizophrenia relapse prevention compared to placebo. J&J. July 8, 2025. Accessed July 8, 2025. https://www.jnj.com/media-center/press-releases/supplemental-new-drug-application-submitted-to-u-s-fda-for-caplyta-lumateperone-with-data-demonstrating-significant-schizophrenia-relapse-prevention-compared-to-placebo
2. Schizophrenia. WHO. Accessed July 8, 2025. https://www.who.int/news-room/fact-sheets/detail/schizophrenia
3. Schizophrenia. NAMI. Accessed July 8, 2025. https://www.nami.org/about-mental-illness/mental-health-conditions/schizophrenia/?tab=overview
