US FDA green lights first gene therapy for bladder cancer
Ferring Pharmaceuticals secured the approval for Adstiladrin (nadofaragene firadenovec-vncg), an adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC).
Adstiladrin provides NMIBC patients a valuable alternative compared to an invasive bladder removal surgery.
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said the approval addresses an area of critical need. “The FDA remains committed to facilitating the development and approval of safe and effective cancer treatments.”
Bladder cancer is the sixth most common cancer in the US.
Ferring's application had been granted priority review, breakthrough therapy, and fast-track designations from the FDA.
The FDA approval was based on results of the company’s Phase 3 clinical trial of Adstiladrin, which met its primary endpoint. The results demonstrated that more than half of patients achieved a complete response at three months and, of these, 46% continued to remain free of high-grade recurrence at 12 months.
Adstiladrin is administered once every three months into the bladder via a urinary catheter. The most common adverse reactions associated with it include bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria, chills, fever, and painful urination.
Ferring expects that the treatment will be commercially available in the US in the second half of 2023, following its expansion of manufacturing capacity, with the company looking to pioneer commercial scale vector production for oncology.
There are now 27 FDA approved cell and gene therapies - five getting the green light this year alone – November saw the US regulatory body approve the first gene therapy for hemophilia B.
And there are huge number of others in development, supporting the FDA’s forecast that it would be approving 10-20 new therapies annually by 2025.