- After years of testing and an initial rejection from the Food and Drug Administration, Ferring Pharmaceuticals on Friday won U.S. approval of its gene therapy for a form of high-risk bladder cancer.
- Bladder cancer is one of the most common forms of cancer. Most newly diagnosed cases are classified as non-muscle invasive bladder cancer, or NIMBC. Ferring’s treatment, called Adstiladrin, is for high-risk NIMBC that is not responsive to Bacillus Calmette-Guérin therapy.
- Adstiladrin’s approval was based on the results of a Phase 3 trial of 157 patients. While it’s now cleared for use, Ferring doesn’t expect to make it available on the U.S. market until the second half of next year as it works to expand its manufacturing capacity.
Also known as nadofaragene firadenovec, Adstiladrin has been in development since 2011, originating at a research institute in Finland. Its original owner, FKD Therapies Oy, licensed the treatment to Ferring in 2018.
One year later, Ferring partnered with Blackstone Life Sciences to invest $570 million in development of the therapy for high-risk BCG-unresponsive NMIBC. At the time, the two companies set up a subsidiary called FerGene to commercialize the drug in the U.S.
In December 2019, Ferring revealed positive results from a Phase 3 trial, showing that just over half achieved a complete response, or disappearance of all signs of cancer, following treatment. According to the FDA, the median duration of response was 9.7 months and 46% of those who responded remained in remission for at least one year.
The company’s first attempt to secure approval ended with a complete response letter from the FDA over manufacturing issues, however. FerGene later restructured.
Adstiladrin is an adenovirus vector-based gene therapy that contains the gene interferon alfa-2b. Administered via a catheter into the bladder once every three months, the therapy delivers the gene to bladder cells, turning “the patient’s own bladder wall cells into interferon microfactories,” according to Ferring.
The approval follows several other recent gene therapy OKs from the FDA. Bluebird bio recently received clearances for its drugs Zynteglo and Skysona, while CSL recently won approval of its hemophilia B treatment Hemgenix.
Ferring, meanwhile, had a microbiota-based treatment cleared by the FDA for a recurrent type of gut infection earlier this month.