Ferring Pharmaceuticals today announced the opening of a state-of-the-art global manufacturing hub in Finland for the drug substance of its intravesical non-replicating gene therapy Adstiladrin® (nadofaragene firadenovec-vncg), an adenovirus vector-based gene therapy drug.
READ MORE: New, modified CRISPR protein can fit inside virus used for gene therapy
READ MORE: Novel nanosensing technique offers quality control of viral vectors in gene therapy
Adstiladrin® (nadofaragene firadenovec-vncg) is approved in the U.S. for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours.
Clinical research
“In January this year, we announced full commercial availability of Adstiladrin® in the U.S. We have now embarked on a broad programme of clinical research to enlarge the body of evidence for Adstiladrin® and plan for regulatory submissions in other global markets. Today’s announcement marks an important milestone in ensuring stable and sustainable global supply of Adstiladrin® to meet the anticipated growth in demand,” said Bipin Dalmia, Global Head, Uro-Oncology and Urology Franchise, Ferring Pharmaceuticals.
Armin Metzger, Executive Vice President and Chief Technical Operations Officer, Ferring Pharmaceuticals added, “The continued investment and expansion of our gene therapy manufacturing infrastructure reflects Ferring’s strong commitment to deliver end-to-end solutions for even the most highly complex manufacturing challenges as part of our mission to fulfil the unmet clinical needs for people with bladder cancer.”
Bladder cancer
According to the International Agency for Research on Cancer, bladder cancer is now the ninth most diagnosed cancer worldwide, previously ranked tenth. This rising global incidence underscores the necessity to expand the supply of innovative treatments for this disease.
In addition to this European investment in drug substance, Ferring has further strengthened and diversified its nadofaragene firadenovec-vncg supply chain and is also nearing completion of a new manufacturing facility for drug product at Ferring’s U.S. campus in Parsippany, New Jersey. The bolstering of Ferring’s gene therapy manufacturing capabilities and footprint reinforces its commitment to ensuring the future supply of this therapy for patients.
Located in Kuopio, Finland, the new European manufacturing site features a cutting-edge manufacturing suite, fully integrated with modern technology to produce adenovirus vector-based gene therapy drug substance in large quantities. The 25,000 square metre facility features renewable energy solutions such as waste heat recovery with heat pumps and solar energy, complementing Ferring’s commitment to protect the environment by reducing its negative impact on the planet.
About the drug
Adstiladrin® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.
It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defences against the cancer.
Adstiladrin® (nadofaragene firadenovec-vncg) has been studied in a clinical trial programme that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).
Gene therapy
Gene therapies aim to treat, prevent, or cure diseases by modifying or manipulating gene expression or altering the biological properties of living cells. The concept involves techniques like adding new copies of a gene or replacing faulty genes with healthy ones.
Over the decades, gene therapies have been applied to inherited genetic diseases, cancers, and other disorders. Viral vectors, bacterial vectors, plasmid DNA, human gene editing, and patient-derived cellular gene therapies are various forms of gene therapy.
Viral vectors are particularly significant due to their high transduction efficiency and stable expression, offering potentially curative treatments with fewer applications. Successful applications include treatments for bladder cancer, leukaemia, lymphoma, haemophilia, certain neurological disorders, retinal diseases, and metabolic disorders.
No comments yet