The FDA in the US has recently approved a first-of-its-kind immunotherapy to treat a HPV-induced disease based on positive animal and clinical trials.
Human papilloma viruses (HPV) are sexually transmitted pathogens known to cause cervical cancers in women. There are more than 150 different HPVs and not all induce the same symptoms. They can also lead to other types of cancers, including anal, penile, and oropharyngeal cancers.
A small number of people who are exposed to HPV 6 and HPV 11 can develop non-cancerous tumours in their air passages, a disease called recurrent respiratory papillomatosis (RRP). Although these respiratory tract papillomas are benign, they can grow very quickly eventually blocking the airway passage, resulting in discomfort and pain in roughly 20,000 adults and children in the US alone. Symptoms tend to be more severe in children than in adults, and once RRP develops, there is no cure. Surgery can be considered but tumours often return chronically meaning patients require dozens or even hundreds of surgeries over their lifetimes.
However, a new treatment called Papzimeos, recently approved by the FDA, could transforms the therapeutic landscape for RRP as the first and so far only therapy for this rare disease.
Papzimeos is an immunotherapy designed to stimulate the patient's immune system to recognise and attack cells infected with HPV types 6 and 11. It targets the underlying viral cause of RRP rather than merely managing symptoms. In preclinical studies, injection of the drug in mice elicited an immune response directed at the respiratory tract papillomas without toxicity. It led to the activation and migration of papilloma-specific T cells into the benign tumours. Although tumours were not eradicated, the drug significantly delayed tumour growth in mice.
After successful tests in animals, clinical trials were run in adults with RRP. Among the 35 patients evaluated, 51% achieved a complete response, defined as no need for surgical intervention in the 12 months following treatment, and 86% requiring fewer surgical interventions. Follow-up data demonstrated that these responses were durable for two years. Administered as four subcutaneous injections over 12 weeks, Papzimeos offers a novel mechanism of action with the potential for long-term disease control through a single course of treatment.
These positive outcomes led to the approval of Papzimeos by the FDA earlier this year for the treatment of RRP. This approval is representative of a growing number of drug approvals with lower clinical data ceilings (backed by solid pre-clinical data in animal models) when intended for rare disease populations. With this validation, Papzimeos becomes the only medical therapy for RRP. For the RRP community, this step offers long-awaited hope as the drug has the potential to offer lasting relief for patients who were previously faced with repeated surgeries to control symptoms of their disease.
Last edited: 4 December 2025 10:56
