FDA Advisory Committee Recommends Approval of Bayer's Riociguat for Pulmonary Hypertension Indications

August 6, 2013—Bayer HealthCare (Whippany, NJ) announced that the Cardiovascular and Renal Drugs Advisory Committee of the US Food and Drug Administration (FDA) has recommended approval for the company's investigational riociguat (proposed tradename: Adempas) for two forms of pulmonary hypertension.

According to Bayer HealthCare, the committee voted 11 to 0 that riociguat should be approved for the treatment of World Health Organization (WHO) group 1 pulmonary arterial hypertension [PAH]. The committee also voted 11 to 0 that riociguat should be approved for the treatment of WHO group 4 chronic thromboembolic pulmonary hypertension (CTEPH). The company stated that the advisory committee's vote will be taken into consideration by the FDA when making its decision on the approvability of Bayer's new drug application (NDA) for riociguat.

In February 2013, Bayer submitted the NDA for riociguat in two indications: (i) the treatment of PAH (WHO group 1) to improve exercise capacity, improve WHO functional class, and delay clinical worsening; and (ii) the treatment of persistent/recurrent CTEPH (WHO group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class. In April, Bayer announced that it would receive priority review, which is given to drugs that have the potential to offer significant improvement in treatment or provide a treatment option where no adequate therapy exists.

Bayer advised that riociguat is an investigational, oral medication for the treatment of adult patients with PAH or inoperable or persistent/recurrent CTEPH; if approved by the FDA later this year, riociguat would create a new class of therapy available in the United States. The company noted that pulmonary hypertension is associated with endothelial dysfunction, impaired synthesis of nitric oxide (NO), and insufficient stimulation of soluble guanylate cyclase (sGC). Riociguat stimulates sGC independent of NO and increases the sensitivity of sGC to NO.

According to Bayer HealthCare, data presented at the FDA advisory committee meeting included results from the global phase 3 clinical program, which enrolled 704 patients across two phase 3 studies. Both studies met their primary endpoint by demonstrating a statistically significant improvement in the 6-minute walk test (6MWT), after 16 and 12 weeks, respectively. Riociguat was also associated with improvements across multiple, relevant, secondary endpoints in the studies. The most common treatment-emergent adverse events with riociguat were headache, dizziness, dyspepsia, peripheral edema, nausea, diarrhea, and vomiting, noted the company.