A committee within the U.S. Food and Drug Administration (FDA) unanimously recommended approval of the drug riociguat to treat two kinds of pulmonary hypertension. The Cardiovascular and Renal Drugs Advisory Committee voted 11 to 0 that the FDA should approve riociguat. Bayer HealthCare developed the drug, which if approved, would be sold under the trade name Adempas.
The proposed drug, taken in pill form, would be used to treat pulmonary arterial hypertension (PAH). It would also be used to treat patients with chronic thromboembolic pulmonary hypertension (CTEPH) who cannot undergo a pulmonary endarterectomy surgery to remove clots in their lungs, or for those who still have complications after having the procedure.
PHA president Rino Aldrighetti wrote a letter to the FDA in advance of the committee’s hearing urging the agency to approve riociguat if they found it to be safe, effective and a great need for it. “While there have been great advances in research and expanded treatment options for pulmonary arterial hypertension in recent years, I want to assure you that it is not enough,” he writes. “We continue to lose too many patients with this disease.” He added: “The need is there for PH where it will be a valuable addition in the arsenal of therapies.”
In an op-ed piece for the New England Journal of Medicine, Stephen Archer, MD, notes that the study results were somewhat modest. CTEPH patients still had better results undergoing surgery than they did taking riociguat. “Patients who are suitable candidates for surgery should continue to undergo surgery and not be relegated to an inferior treatment,” writes Dr. Archer.
In the study that focuses on PAH patients, Archer observes that only 21 percent of patients who took the drug in the trial saw a functional improvement, which is not too different from other pills that treat PAH.
“However, I view the glass as half full, because riociguat appears to be safe and is a promising addition to the pharmacopeia for [PAH and CTEPH patients],” Dr. Archer says.