After prior rejection, Ferring finally nabs FDA nod for bladder cancer gene therapy

With its second approval in as many months, Ferring Pharmaceuticals is celebrating a fruitful end to 2022. 

On Friday, the FDA approved Ferring’s gene therapy Adstiladrin to treat certain patients with bladder cancer. Specifically, the drug is cleared to treat high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors. 

In granting the approval, the FDA cited data showing more than half of patients who received the drug achieved a complete response. The drug is given every three months into the bladder via a urinary catheter.

Adstiladrin offers patients a new treatment in a cancer setting where few effective options exist. Historically, patients who developed BCG-unresponsive disease would undergo bladder removal surgery, Ferring Chief Science Officer Armin Metzger, Ph.D., said in an interview.  

Meanwhile, the drug will have some Big Pharma competition. Merck & Co.’s Keytruda won an approval in this setting back in early 2020. 

Despite the Ferring drug’s solid data, it wasn't exactly a smooth path to the FDA nod. In May 2020, the FDA rejected the drug, citing outstanding questions for the company’s manufacturing partner.  

Since then, Ferring has been working with its partner and the FDA to bring the production process in line with the agency’s expectations, Metzger said. As with other gene therapies, Adstiladrin has a “very complicated, very long, very product-tailored" production process, he noted. 

The company is working with a Finnish manufacturing partner called FinVector. At the same time, Ferring is also working to grow its own capacity, Metzger said. 

Ferring previously spun off the drug into a separate company called FerGene. But Ferring later reassumed control of the gene therapy when FerGene wound down its operations.

Beyond this initial bladder cancer use, Ferring will investigate its gene therapy platform in other indications, the CSO said. 

The approval follows another recent FDA nod for Ferring. Late last month, the agency signed off on the first FDA-approved fecal microbiota product, Rebyota, to treat Clostridium difficile infections. Ferring picked up that treatment in the 2018 acquisition of Minnesota-based Rebiotix.   

As Metzger sees it, gaining two approvals in the span of several weeks is “outstanding" for a company of Ferring’s size and “gives us a good outlook into the future.”