FDA Approves J&J’s Opsynvi as Once-Daily Single-Tablet Combination Therapy for PAH

Rare Daily Staff

The U.S. Food and Drug Administration has approved Johnson & Johnson’s Opsynvi for the chronic treatment of adults with pulmonary arterial hypertension.

Pulmonary arterial hypertension (PAH) is a rare, progressive, and life-threatening blood vessel disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation that eventually leads to right heart failure. An estimated 500 to 1,000 new cases of PAH are diagnosed each year in the U.S.

Opsynvi is the first and only treatment that combines two proven treatments (macitentan, an endothelin receptor antagonist (ERA), and tadalafil, a phosphodiesterase 5 (PDE5) inhibitor) with established efficacy and safety profiles into one tablet to be taken once daily, offering an option that helps to support the implementation of clinical guideline recommendations for early use of combination therapy.

Opsynvi may be used in patients with PAH who are treatment-naïve or who are already on an ERA, PDE5 inhibitor or both. Opsynvi may be used in patients who are currently treated concomitantly with stable doses of macitentan 10 mg and tadalafil 40 mg (20 mg x 2) as separate tablets.

The FDA’s approval of Opsynvi is based on the results from the pivotal phase 3 A DUE study, in which Opsynvi demonstrated greater reduction in Pulmonary Vascular Resistance after 16 weeks versus tadalafil or macitentan monotherapy. 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.

“Clinical guidelines recommend treating patients with initial and sequential dual-combination therapy, regardless of risk at initial diagnosis and follow-up. Historically, this required patients to take multiple pills because no single-tablet combination therapy targeting two or more pathways was available,” said Kelly Chin, professor of Internal Medicine and director of the Pulmonary Hypertension Program at UT Southwestern Medical Center, and an investigator in the A DUE study. “As administration of macitentan and tadalafil together are commonly prescribed for initial therapy for PAH, the introduction of a single tablet offering a patient-friendly approach to support initial combination therapy and rapid escalation for the appropriate patients.”

Photo: Kelly Chin, professor and director of Pulmonary Hyertension Program at UT Southwestern Medical Center