Ferring Pharmaceuticals announced that the U.S. FDA has approved its novel adenovirus vector-based gene therapy, Adstiladrin — marking the first gene therapy approval for bladder cancer.
Specifically, Adstiladrin was approved for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
Adstiladrin, an intravesical therapy administered every three months, targets the patient’s own bladder wall cells to enhance the body’s natural defenses to fight cancer. The FDA approval was based on results of the 157-patient phase 3 clinical trial, which met its primary endpoint with more than half of patients with carcinoma in situ with or without concomitant high-grade Ta or T1 disease, achieving a complete response by three months.
The application was originally submitted by the now-defunct Blackstone-backed Ferring subsidiary FerGene, but was rejected by the FDA in May 2020 after the agency raised questions around CMC and manufacturing.
The resubmitted application was granted Priority Review, Breakthrough Therapy, and Fast Track designations.
It's been a good month for Switzerland-based Ferring, who also grabbed the first FDA approval for a fecal microbiota therapy for the prevention of the recurrence of Clostridioides difficile infection in adults just a few weeks ago when it got the nod for Rebyota.