The Food and Drug Administration granted accelerated approval to Voceryq, made by Inovio Pharmaceuticals, for adults with recurrent respiratory papillomatosis, a rare chronic disease caused by HPV types 6 and 11 in which growths form in the airways, most often around the vocal cords. The approval is the first in the United States for a plasmid DNA-based immunotherapy: the treatment delivers two plasmids into the body’s cells, one carrying genetic instructions for HPV proteins and one carrying an immune-enhancing molecule, IL-12, given by intramuscular injection with an electroporation device.
The agency based the accelerated approval on a trial in 32 adults who had needed at least two surgeries in the year before treatment: of the 26 patients who had at least three baseline surgeries, seven, or 27 percent, needed no surgery at all in the first year after treatment. Because the approval rests on the reduced frequency of surgery, an intermediate endpoint, Inovio must run post-approval trials to confirm the clinical benefit. The therapy holds Breakthrough Therapy and Orphan Drug designations, and the most common adverse reactions were injection site reactions, headache, fatigue, hypertension, nausea and dizziness.