FDA approves Ferring’s Adstiladrin as first gene therapy for bladder cancer
The US Food and Drug Administration (FDA) has approved Ferring Pharma’s Adstiladrin (nadofaragene firadenovec-vncg) as the first gene therapy for the treatment of adult patients with non-muscle-invasive bladder cancer (NMIBC). The novel adenovirus vector-based gene therapy is specifically indicated for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours and is to be administered into the patient's bladder once every three months. The FDA based its decision on a phase 3 multicentre clinical study in 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumours and could be evaluated for response.
The study met its primary endpoint, with 51% of Adstiladrin-treated patients achieving a complete response by three months, and 46% continuing to remain free of high-grade recurrence at 12 months. Bladder cancer is the sixth most common cancer in the US, with NMIBC – a type of cancer that has grown through the lining of the bladder but has not yet invaded the muscle layer – representing approximately 75% of all new bladder cancer cases.
Treatment of patients with high-risk NMIBC, including those with carcinoma in situ, or CIS, often involves removing the tumour and the use of BCG to reduce the risk that the cancer will recur. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease, for which very few effective treatment options exist.
“Patients with BCG-unresponsive NMIBC have historically had limited treatment options other than bladder removal surgery,” said Steven Boorjian, Carl Rosen professor and chair of the Department of Urology at Mayo Clinic, and lead investigator on the recent clinical trial of Adstiladrin. The company expects that Adstiladrin will be commercially available in the US in the second half of 2023, following manufacturing capacity expansion.
Armin Metzger, executive vice president and chief science officer, Ferring Pharma, said: “We are proud to have achieved this critical milestone towards fulfilling the potential of Adstiladrin, a first-of-its-kind therapy, for bladder cancer patients.
“Adstiladrin is the culmination of a complex research, development and production process, and we are grateful to the teams, physicians and patients who have helped us reach this approval.”
Read more »The US Food and Drug Administration (FDA) has approved Ferring Pharma’s Adstiladrin (nadofaragene firadenovec-vncg) as the first gene therapy for the treatment of adult patients with non-muscle-invasive bladder cancer (NMIBC). The novel adenovirus vector-based gene therapy is specifically indicated for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours and is to be administered into the patient's bladder once every three months. The FDA based its decision on a phase 3 multicentre clinical study in 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumours and could be evaluated for response.