The gene therapy, nadofaragene firadenovec-vncg, is now fully available across the United States for patients with this aggressive bladder cancer.
Nadofaragene firadenovec-vncg is now fully available for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with CIS with or without papillary tumors across the United States.1
In December 2022, nadofaragene firadenovec was approved by the FDA based on findings from Study CS-003 (NCT02773849), an open-label, multicenter, single-arm, phase 3 trial in which nadofaragene firadenovec-vncg was effective and demonstrated a favorable risk profile.2,3
In the study of 157 patients with high-risk BCG-unresponsive NMIBC with CIS with or without papillary tumors, complete responses (CRs) to the single intravesical 75 mL dose of nadofaragene firadenovec (3 × 1011 viral particles per mL) were reached in 51% (95% CI, 41%-61%) of patients with carcinoma in situCIS with or without concomitant high-grade Ta or T1 disease within 3 months. With this result, the primary end point of the study was met. Further, the median duration of response (DOR) was 9.7 months with nadofaragene firadenovec, and 46% of the patients who had a CR were high-grade recurrence-free at 12 months.4
Safety results showed that the most common grade 3/4 drug-related adverse event, which was seen in 2 patients, was micturition. Both of these events were grade 3, and no treatment-related deaths were seen during the study.
“From the day we received FDA approval, Ferring has been committed to making [nadofaragene firadenovec] available to every appropriate high-risk NMIBC patient quickly and responsibly, working collaboratively with the bladder cancer community to inform our approach,” said Bipin Dalmia, senior vice president, global head, uro-oncology franchise at Ferring Pharmaceuticals, in a press release.1 “With our significant manufacturing investments, we have achieved full product supply ahead of schedule. We are therefore ending our [nadofaragene firadenovec] Early Experience Program and look forward to bringing this novel therapy to every patient who needs it.”
The ADSTILADRIN Early Experience Program was initiated by Ferring Pharmaceuticals in September 2023. The program included a mix of trial sites that participated in the phase 3 study of nadofaragene firadenovec, as well as community clinics with the highest number of appropriate patients with NMIBC. The temporary program can now be ended as full supply of the product is available ahead of schedule. This will dramatically increase patient access to the agent.
Further, the non-interventional ADSTILADRIN in BLadder CancEr” (ABLE-41) U.S. Real World Evidence (RWE) Study is evaluating early utilization, experiences, and outcomes of nadofaragene firadenovec in the routine care setting. The multicenter, prospective trial is ongoing and has an estimated enrollment of 800 patients aged 18 years and older.5
To be eligible for the study, patients must have been prescribed and scheduled treatment with nadofaragene firadenovec per physician discretion or have received the first instillation of nadofaragene firadenovecof the agent per physician discretion after September 5, 2023, but prior to site activation.
The primary end points of the trial include whether a patient with CIS achieves CR at the first evaluation from first nadofaragene firadenovec instillation or not, and whether or not they achieve a CR any time within 1 year of their first nadofaragene firadenovec instillation.
“[Nadofaragene firadenovec] represents an effective alternative therapy for patients with NMIBC who, historically, had very few options once they no longer responded to standard BCG therapy,” said Andrea Maddox-Smith, chief executive officer of the Bladder Cancer Advocacy Network, BCAN, in a press release.1 “Increasing access to this innovative therapy offers the potential of what patients need most – safe, effective treatment options that bring hope.”