FDA Expands Treprostinil Label for PAH Care

October 21, 2019
Kevin Kunzmann

The approved label update now indicates the therapy delays disease progression when used in combination with an approved oral background PAH therapy.

The US Food and Drug Administration (FDA) has approved a supplement to the New Drug Application (NDA) for treprostinil (Orentiram) extended-release tablets for the treatment of patients with pulmonary arterial hypertension (PAH).

The approved label update now indicates the United Therapeutics therapy delays disease progression when used in combination with an approved oral background PAH therapy. The indication was based on the findings from the FREEDOM-EV trial, in which investigators assessed for primary efficacy endpoint of time to first clinical worsening event, defined as morbidity or mortality.

Treprostinil resulted in a significant increase in time to first clinical worsening event compared with patients who received placebo—consistently across patient subgroups, as well—indicating an achieved primary endpoint and a proven reduction in patient event risk.

The phase 3, international, multicenter, randomized, double-blind, event-driven assessment compared oral treprostinil to placebo among patients with PAH receiving background oral monotherapy—either a phosphodiesterase type 5 inhibitor, an endothelin receptor antagonist, or a soluble guanylate cyclase stimulator.

The therapy decreased adjudicated clinical-worsening event risk by 25% versus placebo (P = .039). Overall, treprostinil decrease disease risk progression by 61% compared with placebo (P = .0002).

Treprostinil was originally approved by the FDA in 2013 for indication in improving PAH patient exercise capacity as a monotherapy. The therapy is a prostacyclin mimetic proven in trials to predominately benefit patients with idiopathic or heritable PAH, or PAH associated with connective tissue disease.

Leigh Peterson, PhD, United Therapeutics vice president of Product Development, said the therapy’s new label indication should bolster the competitive position of treprostinil in PAH care.

"We believe this improved label will provide physicians and patients with even more confidence in the efficacy and benefit of Orenitram,” Peterson said in a statement.


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