Papzimeos is the first immunotherapy approved for recurrent respiratory papillomatosis, a rare lung disorder involving the development of benign tumors in the airways.
The FDA on Thursday signed off on Precigen’s immunotherapy zopapogene imadenovec-drba for the treatment of adults with recurrent respiratory papillomatosis. The product will carry the brand name Papzimeos.
According to the FDA’s announcement of the approval, Papzimeos is a “first-of-its-kind non-replicating” immunotherapy for the disease. Patients with recurrent respiratory papillomatosis (RRP), a rare neoplastic lung disease, suffer from benign tumors in the airways, leading to difficulties in swallowing and breathing. There are currently no cures for RRP and patients have to undergo repeated surgeries to remove the tumors. RRP can lead to death if left unchecked.
The Maryland-based biotech’s stock rose 44% in pre-market trading Friday.
Delivered using adeno-associated virus vectors, Papzimeos works by triggering an immune response against cells infected with HPV 6 or HPV 11—viruses that are known to result in RRP. Pivotal Phase I/II data, which supported Papzimeos’ approval, showed that the biologic elicited a complete response rate of more than 50% in treated patients, while over 85% needed fewer surgical interventions In the year after treatment.
According to the FDA, Papzimeos also has a “favorable” safety profile. The majority of treatment-emergent adverse events were mild or moderate, with no dose-limiting toxicities detected. The regulator in 2023 gave Papzimeos—then called PRGN-2012—its breakthrough therapy designation for RRP.
Papzimeos’ approval is a bright spot for what has been a cloudy few months for the rare disease space. In July, Capricor Therapeutics failed to secure the FDA’s blessing for its cell therapy deramiocel in Duchenne muscular dystrophy (DMD). In the complete response letter, the regulator said that Capricor’s supporting data in its application did not meet the “statutory requirement for substantial evidence of effectiveness.”
A few days later, the FDA rejected another rare disease drug, this time for Ultragenyx’s Sanfilippo syndrome gene therapy UX111. The regulatory rebuff was due to manufacturing issues, the biotech announced at the time, and was “not directly related to the quality of the product.”
