It is the first and only once-daily single-tablet combination therapy by the US Food and Drug Administration (FDA). OPSYNVI combines macitentan, an endothelin receptor antagonist (ERA), with tadalafil, a phosphodiesterase 5 (PDE5) inhibitor, offering a streamlined treatment option for adults with PAH.
PAH, characterized by the narrowing of small pulmonary arteries and elevated blood pressure in the pulmonary circulation, poses a severe threat to patients, often leading to right heart failure. With an estimated 500 to 1,000 new cases diagnosed annually in the U.S., PAH is considered a rare and life-threatening condition.
The approval of the tablet marks a significant advancement in PAH treatment, offering a comprehensive approach to managing the disease. The medication is indicated for adults with PAH classified as World Health Organization (WHO) Group 1 and categorized as WHO functional class (FC) 2-3. It may be used as initial therapy for treatment-naïve patients or those already receiving an ERA, PDE5 inhibitor, or both.
Dr Kelly Chin, professor of internal medicine and director of the Pulmonary Hypertension Program at UT Southwestern Medical Center, is optimistic about the treatment's potential impact on patient care.
She said: “The introduction of OPSYNVI as a single-tablet combination therapy is promising for clinicians treating PAH patients. It bridges the gap between clinical guidelines and everyday clinical practice, offering a patient-friendly approach to support initial combination therapy and rapid escalation for appropriate patients.”
The FDA's approval of the tablet was based on findings from the pivotal phase 3 A DUE study, which demonstrated a superior reduction in PVR after 16 weeks compared to tadalafil or macitentan monotherapy. However, the tablet carries a Boxed Warning due to the risk of embryo-fetal toxicity, requiring female patients to enroll in the Macitentan-Containing Products Risk Evaluation and Mitigation Strategy (REMS) program.
The A DUE study was a phase 3, multicenter trial comparing the efficacy and safety of OPSYNVI to macitentan and tadalafil monotherapies in adult patients with PAH. The study demonstrated the tablet's superiority in reducing Pulmonary Vascular Resistance (PVR) after 16 weeks compared to monotherapy options.
James List, global therapeutic area head at Johnson & Johnson, highlighted the significance of the treatment in streamlining PAH management.
He said: “People with PAH often face the challenge of taking multiple pills daily, impacting their quality of life. OPSYNVI offers a promising solution to optimize disease management, aligning with updated treatment guidelines that advocate for initial or early combination treatment.”
The tablet represents a pivotal addition to Johnson & Johnson's PAH portfolio, addressing all three foundational pathways recommended by guidelines – nitric oxide, endothelin, and prostacyclin. The medication's approval underscores the company's commitment to advancing treatment options and improving outcomes for PAH patients.