Bayer Drug Granted Orphan Status

Bayer HealthCare has received from the FDA two separate orphan drug designations for its investigational, oral medication riociguat (proposed trade name Adempas), for the treatment of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). 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 U.S., 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 an NDA for riociguat in February 2013 for two indications: (i) the treatment of PAH to improve exercise capacity, improve WHO functional class and delay clinical worsening; and (ii) the treatment of persistent/recurrent CTEPH after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class.