FDA Nod Makes Ferring Pharma Gene Therapy the First for Bladder Cancer

2022-12-19

基因疗法临床3期临床结果上市批准免疫疗法

Bladder cancer can be treated with several types of drugs. Gene therapy is now part of the mix. The FDA has approved a 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 that’s commonly used as a first-line treatment. The Ferring therapy, known in development as nadofaragene firadenovec, is designed to get the body to produce a particular therapeutic protein that fights the cancer. Engineered versions of the protein Interferon-alfa 2b are already 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 the cells lining a patient’s bladder into tiny factories that churn out high quantities of human interferon-alfa 2b. Delivered via an adenovirus, the therapy brings to bladder wall cells a gene that encodes interferon-alfa 2b. The protein-making machinery of the cells translate the DNA sequence and then express the therapeutic protein, which is intended to provide anti-tumor effects. The FDA decision for the Ferring 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 via a catheter, was administered once every three months for up to 12 months. Results showed that 51% of patients who received Adstiladrin achieved a complete response, meaning the disappearance of all signs of the cancer, at three months. The median duration of that response was 9.7 months; 46% of those responders maintained that response for at least one year. Bladder discharge, fatigue, bladder spasm, urinary urgency, and blood in the urine were among the most common adverse reactions reported from the clinical trial. The results were published by The Lancet Oncology in 2020. Adstiladrin was initially developed by FKD Therapies. Ferring, a privately held company based in Saint-Prex, Switzerland, licensed global rights to the gene therapy in 2019; the following year, Ferring and Blackstone Life Sciences formed a company called FerGene to continue development and commercialization of the bladder cancer treatment. The partners backed FerGene FerGene with a combined $570 million. An application seeking FDA approval of Adstiladrin was turned down in 2020 due to manufacturing issues. Two months ago, Ferring and Blackstone restructured their alliance to give Ferring full control of FerGene FerGene and its bladder cancer gene therapy. In a news release announcing regarding the FDA approval, Ferring said it expects Adstiladrin will become commercially available in the second half of 2023 after the company expands its manufacturing capacity. Approval of Adstiladrin is the second affirmative FDA decision for Ferring in the past month. In late November, the agency approved Ferring’s Rebyota, the first microbiota-based live biotherapeutic for preventing recurrence of Clostridioides difficile infection in adults. Image: Getty Images, magicmine

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