The US Food and Drug Administration (FDA) has granted accelerated approval to Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd), previously referred to as INO-3107, to reduce the frequency of surgery in adults with recurrent respiratory papillomatosis (RRP).
RRP is a rare viral disease where tumors grow in the respiratory tract. The tumors can cause a hoarse voice, chronic cough, and breathing difficulties. These tumors rarely become cancerous, but can cause long-term airway and voice complications. RRP is caused by two types of human papillomaviruses (HPV), HPV 6 and HPV 11.
Voceryq is a plasmid DNA-based immunotherapy designed to generate an immune response using DNA plasmids that encode for HPV-6 and HPV-11 antigens. The immunotherapy also contains another plasmid that encodes for an interleukin-12 (IL012) immune adjuvant to stimulate an immune response to HPV-6 and HPV-11 infected cells.
The approval is based on data from the RRP-001 clinical trial (NCT04398433), a phase 1/2 open-label, multicenter trial evaluating Voceryq in 32 adult patients with RRP who had two or more surgeries in the year prior to treatment.
During the Voceryq dosing window, no additional papilloma surgical excisions were required in the protocol. Of the 32 patients, 26 patients had very severe RRP and required 3 or more surgeries for papilloma excision in the 12 months prior to treatment. Seven of those 26 patients demonstrated a complete response, defined as requiring no surgical excision of papillomas, after 12 months of follow up. For all 32 patients, 23 of them had a complete or partial response to therapy, with a partial response defined as a 50% or more reduction in surgical interventions in the 12 month period.
Additionally, Voceryq was well tolerated, with the most common adverse reactions including injection site reactions, headache, fatigue, hypertension, nausea and dizziness. Most treatment-related adverse reactions were grade 1 and 2 and transient. There were no grade 4 or 5 common adverse events and all 32 patients completed the study.
Continued approval for this indication may be contingent on verification and description of clinical benefit in a confirmatory study(ies).
To view the complete clinical data, visit Nature Communications and The Laryngoscope.
To learn more about RPP and other rare lung conditions, visit https://checkrare.com/diseases/lung-diseases/
