FDA Approves First-In-Class Schizophrenia Drug COBENFY™ by Bristol Myers Squibb

COBENFY™ (xanomeline and trospium chloride) has been approved by the U.S. Food and Drug Administration (FDA) as a new oral treatment for schizophrenia in adults. This approval marks the introduction of the first new pharmacological approach for schizophrenia in several decades, as COBENFY targets M1 and M4 receptors in the brain without blocking D2 receptors, unlike current treatments.

Speaking about this significant advancement, Chris Boerner, PhD, the board chair and CEO of Bristol Myers Squibb, emphasized that COBENFY could revolutionize the treatment paradigm for schizophrenia, especially given the lack of new approaches in over 30 years. Boerner noted that while the company reenters the neuropsychiatry field, it aims to alter the conversation around serious mental illnesses.

Schizophrenia is a persistent and often disabling mental illness affecting approximately 2.8 million people in the United States. Symptoms generally manifest in early adulthood and vary widely among individuals, complicating diagnosis and management. Despite the effectiveness of the current standard of care, up to 60% of patients experience inadequate symptom improvement or intolerable side effects.

Gordon Lavigne, CEO of the Schizophrenia & Psychosis Action Alliance, pointed out the challenges patients face in finding effective treatments. He stressed that the approval of COBENFY provides another valuable option for managing this serious condition.

The FDA's approval is based on data from the EMERGENT clinical program, which includes three placebo-controlled efficacy and safety trials and two open-label trials assessing the long-term safety and tolerability of COBENFY for up to one year. In the Phase 3 EMERGENT-2 and EMERGENT-3 trials, COBENFY met its primary endpoint by significantly reducing schizophrenia symptoms compared to a placebo. The Positive and Negative Syndrome Scale (PANSS) was used to measure the total score change from baseline to week five, showing a 9.6-point reduction in EMERGENT-2 and an 8.4-point reduction in EMERGENT-3.

In addition to its efficacy, the safety profile of COBENFY has been extensively evaluated. The most common adverse reactions reported in the Phase 3 EMERGENT-2 and EMERGENT-3 trials included nausea, dyspepsia, constipation, vomiting, hypertension, abdominal pain, diarrhea, tachycardia, dizziness, and gastroesophageal reflux disease. Unlike many antipsychotic medications, COBENFY does not carry atypical antipsychotic class warnings and precautions and lacks a boxed warning.

Dr. Rishi Kakar, the chief scientific officer and medical director at Segal Trials and an investigator in the EMERGENT program, remarked on the transformative potential of COBENFY. He noted that previous medications for schizophrenia have relied on the same primary pathways in the brain, but COBENFY leverages a novel pathway, offering a new option for managing this challenging condition.

Bristol Myers Squibb also announced the launch of COBENFY Cares™, a support program for patients prescribed COBENFY, which will be available from late October. This program aims to assist patients in adhering to their treatment plans and managing their condition effectively.

Schizophrenia is a severe mental illness that disrupts how individuals think, feel, and behave. Symptoms are divided into three domains: positive symptoms (e.g., hallucinations, delusions), negative symptoms (e.g., lack of motivation), and cognitive dysfunction (e.g., impaired memory and decision-making). Affecting nearly 24 million people worldwide, including 2.8 million in the U.S., schizophrenia is a leading cause of disability globally.

COBENFY™ (xanomeline and trospium chloride) combines xanomeline, a dual M1 and M4 muscarinic receptor agonist, with trospium chloride, a muscarinic receptor antagonist, to treat schizophrenia. The EMERGENT clinical program has demonstrated its efficacy and safety, making it a promising new option for those affected by this debilitating condition.