Precigen Wins First FDA Immunotherapy Nod for Recurrent Respiratory Papillomatosis

The FDA on Thursday signed off on Precigen’s immunotherapy zopapogene imadenovec-drba for the therapy of adults with recurrent respiratory papillomatosis. The product will carry the model identify Papzimeos.

In response to the FDA’s announcement of the approval, Papzimeos is a “first-of-its-kind non-replicating” immunotherapy for the illness. Sufferers with recurrent respiratory papillomatosis (RRP), a uncommon neoplastic lung illness, undergo from benign tumors within the airways, resulting in difficulties in swallowing and respiration. There are at present no cures for RRP and sufferers should endure repeated surgical procedures to take away the tumors. RRP can result in loss of life if left unchecked.

The Maryland-based biotech’s inventory rose 44% in pre-market buying and selling Friday.

Delivered utilizing adeno-associated virus vectors, Papzimeos works by triggering an immune response towards cells contaminated with HPV 6 or HPV 11—viruses which might be identified to lead to RRP. Pivotal Part I/II knowledge, which supported Papzimeos’ approval, confirmed that the biologic elicited a whole response fee of greater than 50% in handled sufferers, whereas over 85% wanted fewer surgical interventions Within the yr after therapy.

In response to the FDA, Papzimeos additionally has a “favorable” security profile. Nearly all of treatment-emergent hostile occasions have been delicate or reasonable, with no dose-limiting toxicities detected. The regulator in 2023 gave Papzimeos—then referred to as PRGN-2012—its breakthrough remedy designation for RRP.

Papzimeos’ approval is a brilliant spot for what has been a cloudy few months for the uncommon illness area. In July, Capricor Therapeutics didn’t safe the FDA’s blessing for its cell remedy deramiocel in Duchenne muscular dystrophy (DMD). Within the full response letter, the regulator stated that Capricor’s supporting knowledge in its utility didn’t meet the “statutory requirement for substantial proof of effectiveness.”

A couple of days later, the FDA rejected one other uncommon illness drug, this time for Ultragenyx’s Sanfilippo syndrome gene remedy UX111. The regulatory rebuff was resulting from manufacturing points, the biotech introduced on the time, and was “indirectly associated to the standard of the product.”