On March 2, 2024, the U.S. Food and Drug Administration (FDA) approved OPSYNVI for the treatment of chronic pulmonary arterial hypertension (PAH) in adult patients of WHO functional class II-III. This is the first single-tablet treatment to combine macitentan, an endothelin receptor antagonist (ERA), and tadalafil, a phosphodiesterase 5 (PDE5) inhibitor.
Adults who have treatment-resistant PAH, or who already use an ERA, PDE5 inhibitor or both, may use OPSYNVI. Adults who are currently treated concomitantly with stable doses of macitentan 10 mg and tadalafil 40 mg (20 mg x 2) as separate tablets may use OPSYNVI.
The FDA approval is based on results from the phase 3 A DUE study, which demonstrated greater reduction in pulmonary vascular resistance (PVR) after 16 weeks versus tadalafil or macitentan monotherapy. The new treatment option supports the clinical guidelines recommendations for the early use of combination therapy to treat PAH.
“As administration of macitentan and tadalafil together are commonly prescribed for initial therapy for PAH, the introduction of a single tablet combining both is promising for clinicians treating patients as it may help bridge the gap between clinical guidelines and everyday clinical practice, while offering a patient-friendly approach to support initial combination therapy and rapid escalation for the appropriate patients,” said Kelly Chin, MD, professor of internal medicine and director of the Pulmonary Hypertension Program at UT Southwestern Medical Center, and an investigator in the A DUE study.
Individually, macitentan reduces the risk of clinical worsening events and hospitalization, and tadalafil improves exercise ability.
OPSYNVI has a boxed warning due to the risk of embryo-fetal toxicity and requires female patients to enroll in the Macitentan-Containing Products Risk Evaluation and Mitigation Strategy (REMS) program.
“People with PAH often live with the burden of taking many pills each day, which can pose challenges,” said James F. List, MD, PhD, global therapeutic area head at Johnson & Johnson. “We're thrilled to bring this single-tablet combination therapy to patients, as it has the potential to optimize disease management and fulfill a significant unmet need in supporting recently updated treatment guidelines that call for initial or early combination treatment.”
