Bladder cancer can be treated with several types of medication. Gene therapy is now part of the mix. The FDA approves Ferring Pharmaceuticals’ gene therapy as a treatment for patients whose cancer is at high risk of progressing by invading muscle tissue and spreading throughout the body.
The regulatory decision announced Friday covers the treatment of adults whose bladder cancer does not respond to Bacillus Calmette-Guérin (BCG), an immunotherapy usually used as first-line treatment. Ferring’s therapy, known in development as nadofaragene firadenovec, is designed to make the body produce a certain therapeutic protein that fights cancer.
Engineered versions of the Interferon-alfa 2b protein are already being used for indications that include hepatitis B and C infections, hairy cell leukemia and melanoma. Ferring’s gene therapy, which will be marketed under the brand name Adstiladrin, turns cells lining a patient’s bladder into tiny factories that produce large amounts of human interferon-alpha 2b. Delivered via an adenovirus, the therapy delivers a gene that codes for interferon-alpha 2b to the cells of the bladder walls. The cells’ protein-making machinery translates the DNA sequence and then expresses the therapeutic protein that is designed to provide antitumor effects.
The FDA’s decision on Ferring’s gene therapy was based on the results of an open-label, single-arm, phase 3 study that enrolled 157 patients. The gene therapy, which is dosed through a catheter, is administered once every three months for up to 12 months. The results showed that 51% of the patients who received Adstiladrin achieved a complete response, meaning the disappearance of all signs of cancer, after three months. The median duration of this response was 9.7 months; 46% of these respondents maintained this response for at least one year. Bladder discharge, fatigue, bladder spasm, urinary urgency, and blood in the urine were among the most common side effects reported from the clinical trial. The results were published from The Lancet Oncology in 2020.
Adstiladrin was originally developed by FKD Therapies. Ferring, a privately held company based in Saint-Prex, Switzerland, licensed global rights to the gene therapy in 2019; next year, Ferring and Blackstone Life Sciences have formed a company called FerGene to further develop and commercialize treatments for bladder cancer. The partners backed FerGene with a total of $570 million.
An application for FDA approval of Adstiladrin was rejected in 2020 due to manufacturing issues. Two months ago Ferring and Blackstone restructured their alliance to give Ferring full control of FerGene and its gene therapy for bladder cancer. IN news release announcing regarding the FDA approval, Ferring said it expects Adstiladrin to become commercially available in the second half of 2023, once the company expands its manufacturing capacity.
Adstiladrin’s approval is the second positive FDA decision for Ferring in the past month. At the end of November, the agency approved Ferring’s Rebyota, the first microbiota-based living biotherapeutic to prevent relapse of Clostridioides difficile infection in adults.
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