The Voceryq FDA approval on Oct. 30 makes Inovio Pharmaceuticals’ treatment for adults with recurrent respiratory papillomatosis the first plasmid DNA-based immunotherapy approved in the United States, the agency said. The approval is accelerated, which means it rests on an early marker of benefit.
That marker is a reduced need for surgery, and the trial behind it enrolled 32 adults in an open-label design. Of 26 patients with at least three surgeries before treatment, 7 needed no surgical intervention in the first year. That is 27%, which leaves 19 of 26 patients, or 73%, who still had at least one operation, a calculation from the FDA’s figures. For a disease that sends patients back to the operating room repeatedly, each avoided procedure counts, and the FDA is requiring more trials to show the benefit holds.
What Recurrent Respiratory Papillomatosis Is
Recurrent respiratory papillomatosis is a rare, chronic disease caused by human papillomavirus types 6 and 11. It produces wart-like growths in the airways, most often around the larynx. The patients in the trial were adults who had two or more laryngotracheal papilloma surgeries in the prior 12 months, including a surgery scheduled when they entered the study.
How Voceryq Works and How Doctors Give It
Voceryq combines two plasmids, which are small circular pieces of DNA. One carries instructions for specific HPV proteins. The other carries instructions for IL-12, a molecule that enhances the immune response. Doctors give the drug by intramuscular injection with the CELLECTRA 5PSP device, which uses brief electrical pulses to help move the drug into cells.
Karim Mikhail, director of the FDA’s Center for Biologics Evaluation and Research, said the approval “represents a meaningful scientific breakthrough”. He said it gives patients a new option that targets the cause of the disease and could mean fewer surgeries.
Side Effects and Required Follow-Up Trials
The most common side effects were injection site reactions, headache, fatigue, high blood pressure, nausea and dizziness. The electrical delivery adds its own list: brief muscle contractions, temporary bleeding at the injection site, anxiety, vasovagal reactions such as lightheadedness, short-lived changes in creatine phosphokinase, and redness and pain where the device touches the skin. The FDA granted Voceryq Breakthrough Therapy and Orphan Drug designations. As a condition of accelerated approval, Inovio must run post-approval clinical trials to confirm and describe the treatment’s clinical benefit.
