The Janssen Pharmaceutical Companies of Johnson & Johnson announced today (31st May) that a submission of a new drug application (NDA) has been submitted to the US Food and Drug Administration (FDA).
It is seeking approval for a single tablet combination therapy of macitentan 10mgs and tadafil 40mgs, called MT/STCT for the long-term treatment of PAH. This is the first and only single tablet combination therapy application to be submitted for review in the US for the rare, progressive disease.
M/T STCT is the first investigational, single tablet combination therapy that combines the endothelin receptor antagonist (ERA) macitentan and the phosphodiesterase type 5 inhibitor (PDE5i) tadalafil for once daily administration.
The application is based on positive data from the Phase 3 A DUE study, which met its primary endpoint and demonstrated that M/T STCT significantly improved blood flow through pulmonary blood vessels known as pulmonary hemodynamics versus macitentan and tadalafil monotherapies in this PAH patient population.
James List, global therapeutic area head at Janssen, said: “People with PAH are often prescribed numerous medicines to manage their condition, so the potential to offer a single tablet combination of two guideline-recommended therapies could not only decrease pill burden but may also help improve the patient treatment experience and clinical outcomes.
“Today's submission builds on our decades-long commitment to address patient needs and bring forward medicines with the potential to offer an improved standard of PAH care.”
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.
Currently it is recommended patients take multiple pills as there is no single pill that combines two or more PAH-specific pathways. The most recent European Society of Cardiolgy/European Respiratory guidelines recommend initial double combination therapy with macitentan and tadalafil for PAH without cardiopulmonary comorbidities.
The study met its co-primary endpoints, demonstrating marked pulmonary hemodynamic improvement. The safety profile of M/T STCT was consistent with the safety profile of the individual components, macitentan and tadafil. The open label arm of the A DUE study is ongoing.
If approved, Janssen's comprehensive PAH portfolio has the potential to cover all guidelines-recommended treatment pathways.