After securing approval at the end of last year for a potential alternative to bladder removal in certain bladder cancer patients, Ferring Pharmaceuticals has given the gene therapy Adstiladrin to its first commercial patient.
The patient, a 78-year-old man in the Midwest, is the first of many to receive treatment through Ferring’s early experience program. The program is meant to ensure patients can access the drug during a major manufacturing scale-up.
Because the adenovirus vector-based therapy is complicated to make, the company knew it had to be “very, very responsible” to ensure it was getting the med to patients while ramping up manufacturing, Ferring’s chief scientific officer Elizabeth Garner, M.D., MPH, said in an interview.
Two new sites were cleared to specifically produce Adstiladrin earlier this summer, one in Finland and the other in New Jersey. Manufacturing should hit full capacity by 2025 when Ferring expects to receive $200 million in milestone payments from Royalty Pharma through a royalty deal worth up to $500 million.
For now, investigators who studied Adstiladrin in its phase 3 trial and community clinics with the highest number of eligible patients can participate in the early experience program.
More clinics will be invited to join the program as manufacturing output increases. The program will continue to expand in the next year or so, Garner said.
Importantly, the framework makes sure that any patient dosed during the first round can come back for their entire treatment regimen of every three months stretched out over a minimum of one year, despite the tight supply. The recently treated patient scheduled his next appointment for December.
Adstiladrin was approved last December to treat high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (CIS) with or without papillary tumors. It’s the first gene therapy to target high-risk and non-muscle invasive bladder cancer in patients who no longer respond to standard therapy and is administered directly into the bladder from a catheter.
The patient was also enrolled in Ferring’s real-world evidence study, called ABLE-41. The study looks to enroll up to 800 patients.
Through the study, the company will analyze data evaluating not only the drug’s effectiveness through measures such as durability, survival and complete response rates but also patient, caregiver and physician experience. Those outcomes will be determined through several questionnaires that can give the company a better picture of what the quality of life looks like for patients treated and the impact on their caregivers, Garner said.
ABLE-41 is expected to last up to two years.