FDA advisors recommend approval of novel agent for pulmonary hypertension

Troy Brown

August 08, 2013

Silver Spring, MD - An advisory committee to the US Food and Drug Administration (FDA) has voted unanimously to recommend that the agency approve riociguat (Adempas, Bayer Healthcare) for the treatment of two forms of pulmonary hypertension[1,2]. The recommended indications are to improve exercise capacity and World Health Organization (WHO) functional class in chronic thromboembolic pulmonary hypertension (CTEPH, WHO group 4) and to improve exercise capacity and WHO functional class and delay clinical worsening in pulmonary arterial hypertension (PAH, WHO group 1).

The data "were convincing and showed efficacy and acceptable safety," according to panelist Dr Vasilios Papademetriou (Veterans Affairs Medical Center, Washington, DC).

Riociguat is an oral soluble guanylate cyclase stimulator that can potentiate nitric-oxide pathways. The panel's decisions were based on two randomized, controlled trials, recently published and reported by heartwire , the Pulmonary Arterial Hypertension Soluble Guanylate Cyclase-Stimulator Trial 1 (PATENT-1) and Chronic Thromboembolic Pulmonary Hypertension Soluble Guanylate Cyclase-Stimulator Trial 1 (CHEST-1). In both studies, in their respective pulmonary-hypertension populations, the drug significantly improved both pulmonary-artery (PA) hemodynamics and exercise capacity over 12 to 16 weeks.

"The benefit/risk [profile] was really positive," according to panelist Dr Philip Sager (Stanford University School of Medicine, CA). Although, the panelists said, the concomitant use of riociguat with nitrates should be contraindicated.

In patients with inoperable CTEPH, riociguat "is probably the only medical therapy that has been shown to have a benefit," according to panelist Dr Linda F Fried (University of Pittsburgh, PA). She echoed the opinion of several others that riociguat should not be limited to patients with inoperable CTEPH and that all patients with CTEPH should have the option of using the drug.

The committee members have disclosed no relevant financial relationships.




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