FDA grants accelerated approval to a plasmid DNA therapy for a rare airway disease

The Food and Drug Administration on October 30 granted accelerated approval to Voceryq, made by Inovio Pharmaceuticals, for adults with recurrent respiratory papillomatosis. It is the first plasmid DNA-based immunotherapy approved in the United States. The disease, tied to certain human papillomavirus types, causes growths in the airway that often have to be removed again and again. The FDA said surgery does not clear the virus, and it based this approval on fewer surgeries, an intermediate result it considers reasonably likely to predict benefit.

That evidence comes from an open-label trial of 32 adults who had already needed at least two surgeries in the prior year. Among 26 patients who had at least three surgeries in that year, 7 needed no surgery during the first year after treatment. Accelerated approval still requires the company to run post-approval trials to confirm clinical benefit. The result is not a cure, and it does not say how the drug will perform for every adult with the disease.