FDA Grants Accelerated Approval to Novel Immunotherapy for Rare Airway Disease

The FDA has granted accelerated approval to a novel plasmid DNA-based immunotherapy, marketed under the name Voceryq and consisting of doruxapapogene ralaplasmid and lalikinogene sifuplasmid, designed to reduce the frequency of surgical excisions in adults with recurrent respiratory papillomatosis. The condition is characterized by wart-like growths in the airways, particularly around the larynx, that are caused by human papillomavirus. While there is no cure for the rare disease, severe cases can obstruct breathing and require repeated surgeries.

Clinical Trial Findings and Response Rates

The regulatory decision was supported by efficacy data from the phase I and phase II open-label clinical trial known as RRP-001. The study included 32 patients who had undergone a minimum of two surgeries in the prior year to remove the growths. Among a subset of 26 patients who required at least three surgeries in the 12 months before initiating treatment, 27% achieved a complete response. This outcome was defined as needing no surgical excisions during 12 months of follow-up. Investigators noted that the therapy aims to transition care away from frequent surgeries, which carry inherent risks of laryngeal injury.

Mechanism and Treatment Regimen

The newly approved treatment utilizes two plasmids containing genetic coding for human papillomavirus proteins and interleukin 12. Healthy patient cells transcribe this genetic code to stimulate the immune system to target cells infected with human papillomavirus types 6 and 11. The treatment regimen involves an initial surgical debulking of visible papilloma, followed by an intramuscular dose administered every three weeks for a total of four doses, delivered in combination with a proprietary DNA medicine delivery device called CELLECTRA 5PSP.

Safety and Regulatory Requirements

Label warnings highlight potential adverse events, including muscle contractions, transient bleeding, and vasovagal reactions. The most frequently reported adverse events in clinical trials were injection site reactions, headache, fatigue, hypertension, nausea, and dizziness. Because the therapy received accelerated approval based on initial clinical response data, the manufacturer will be required to conduct postmarketing trials to verify and describe its clinical benefit further.