The U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development has granted two separate orphan drug designations for Bayer HealthCare’s investigational, oral medication riociguat, proposed trade name Adempas, for the treatment of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH), according to the company.
The Orphan Drug Designation program provides orphan status to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the United States, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
Bayer submitted a New Drug Application (NDA) for riociguat in February 2013 for two indications: 1) the treatment of PAH (WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening; and 2) the treatment of persistent/recurrent CTEPH (WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class.
PAH and CTEPH are both life-threatening forms of pulmonary hypertension that cause significantly increased pressure in the pulmonary arteries. Riociguat is an investigational, oral medication for the treatment of adult patients with PAH or persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH. If approved by the FDA, it would create a new class of drugs, sGC stimulators, available in the United States.
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.
Date: September 25, 2013