Nadofaragene firadenovec-vncg

On Friday, the EMA's drug advisory panel issued its latest raft of recommendations and regulatory updates, unveiling positive opinions for five new medicines.Heading the list was a recommendation to approve Amgen's Imdylltra (tarlatamab) to treat adults with extensive-stage small-cell lung cancer (ES-SCLC) whose disease relapsed during or after an initial treatment with platinum-based chemotherapy.The T-cell engager, which targets both DLL3 and CD3, was authorised in the same indication in the US in 2024, where it is marketed as Imdelltra. While the FDA's accelerated approval was based on the Phase II DeLLphi-301 study, the EMA's decision was supported by findings from the Phase III DeLLphi-304 trial. Data reviewed by the agency's Committee for Medicinal Products for Human Use (CHMP) showed that Imdylltra was associated with a significant improvement in median overall survival of 13.6 months, versus 8.3 months for standard care, corresponding to a 40% reduction in the risk of death. Meanwhile, median progression-free survival in the two groups was 4.2 months and 3.2 months, respectively.Similar to the US — where Amgen's drug carries a boxed warning for cytokine release syndrome (CRS) and neurologic toxicities — the EMA said that the product information for Amgen's drug will describe these risks and guidance on their management. Staying in ES-SCLC, the CHMP also recommended approval of PharmaMar's Zepzelca (lurbinectedin) for the maintenance treatment of patients whose disease has not progressed after first-line induction therapy. The alkylating agent, which was recently cleared for this use in the US, is indicated in combination with Roche's anti–PD-L1 Tecentriq (atezolizumab).Thumbs up for Sanofi's SC SarclisaAlso earning itself a CHMP nod was Sanofi and its on-body injector subcutaneous (SC) formulation of Sarclisa (isatuximab). The recommendation covers all four of the multiple myeloma indications currently held by the intravenous (IV) version of the anti-CD38 mAb in the EU.The automated hands-free device uses a 30-gauge hidden and retractable needle that is thinner than commonly used SC injection needles.At the American Society of Clinical Oncology (ASCO) meeting last year, Sanofi shared data from the Phase III IRAKLIA study showing that injectable Sarclisa achieved a 71.1% objective response rate after a median follow-up of 12 months, compared to 70.5% for the IV formulation, meeting the non-inferiority primary endpoint. Progression-free survival rates at 12 months were similar between the two formulations at 66.1% and 65.1%, respectively.Decisions go the way of Ferring, PharmingIn other decisions, the CHMP recommended conditional approval of Ferring Pharmaceuticals' gene therapy Adstiladrin (nadofaragene firadenovec) to treat adults with Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumours.Adstiladrin has been on the market in the US for several years after winning FDA approval at the end of 2022.The CHMP also backed authorisation of Pharming's Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older weighing 45 kg or more. While the oral, selective PI3Kδ inhibitor is approved for this patient group in the US, efforts to expand use into children aged four to 11 years with the rare and progressive primary immunodeficiency were recently rejected by the FDA. Although Pharming is hopeful of addressing the US regulator's issues — which relate to clinical pharmacology and batch testing methodology — the paediatric approval had been expected to drive a Joenja growth inflection this year.Elsewhere, the CHMP issued a positive opinion recommending paediatric use of Proveca Pharma's Bopediat (furosemide) to treat oedema of cardiac or renal origin, oedema of hepatic origin and hypertension in children from birth to under 18 years of age with chronic kidney disease. Bopediat is a hybrid medicine of Lasilix Faible (furosemide), which has been authorised in France since 1977. The EMA noted that Proveca's drug contains the same active substance as Lasilix Faible, but it is available as orodispersible tablets and at a lower strength so that it can be given to young children.Re-examinations end badlyThe EMA on Friday also confirmed the recent decision by Anavex Life Sciences to withdraw an application for its Alzheimer's drug candidate blarcamesine. Having recommended refusing approval at the end of last year, the CHMP was in the process of re-examining the application — as requested by the drugmaker — when the company opted to pull the filing.Another re-examination resulting in a negative outcome went to Hetlioz (tasimelteon) and its developer Vanda Pharmaceuticals, who was seeking to expand use into people with a rare hereditary disorder, Smith-Magenis syndrome. The melatonin‑receptor agonist is already approved in the EU to treat totally blind adults with non-24-hour sleep-wake disorder.Having initially rejected use of Hetlioz in Smith-Magenis syndrome last November, the CHMP reaffirmed its decision at an extraordinary meeting on March 16. The committee expressed concerns about the study used to support the application, including the statistical analysis of the results and the way the trial had been conducted, which led to uncertainties about the drug's effects.Tecovirimat use restrictedThe EMA also said Friday that SIGA Technologies' antiviral tecovirimat should no longer be used for mpox. The drug, also known as TPOXX, was approved in the EU in 2022 to treat smallpox, mpox and cowpox based on data from animal studies.Subsequent trials conducted in humans — including the STOMP study sponsored by the US National Institutes of Health (NIH) — showed that tecovirimat did not heal lesions faster compared with placebo in people presenting with active mpox lesions, while it also failed to improve other outcomes, such as relieving pain or helping to clear the virus from the body faster.

Accelerated water-bath thawing, completed in about 25 minutes, may help streamline clinical workflows and support efficient patient management Label update includes data showing that product stability is maintained with the 25-minute, 25° C water-bath method Label update reinforces Ferring’s commitment to improving patient and provider experience through continued innovation in the delivery and handling of ADSTILADRIN PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has approved a label update for ADSTILADRIN ® (nadofaragene firadenovec-vncg), enabling an accelerated water-bath thawing method, completed in about 25 minutes, to enhance efficient clinical preparation for healthcare teams. ADSTILADRIN is the first and only FDA-approved non-replicating intravesical gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). In the United States, bladder cancer is the sixth most common cancer, with NMIBC comprising about 75% of all new cases. 1,2 "The reduction in thawing time for ADSTILADRIN streamlines preparation and handling for healthcare providers treating patients with high-risk non-muscle invasive bladder cancer," said Vikram M. Narayan, MD, FACS, Associate Professor at Emory University Department of Urology, Chief of Urology at Grady Memorial Hospital. “This updated preparation process has the potential to save valuable time and enable healthcare providers and practices to deliver this treatment option to more patients efficiently.” ADSTILADRIN is shipped and stored as a sterile frozen suspension and must be brought to room temperature (20° C to 25° C [68° F to 77° F]) prior to use. The FDA’s approval is supported by a thawing and handling study demonstrating that frozen vials remained stable when thawed for about 25 minutes in a water bath maintained at 25°C (77°F). The storage conditions for ADSTILADRIN remain unchanged once thawing begins and permit storage for up to 24 hours at room temperature or up to 7 days refrigerated (2-8°C), including thaw time. "At Ferring, we remain deeply committed to evolving our therapies in ways that address the real-world needs of patients and the healthcare providers who care for them," said Denise D’Andrea, MD, FACP, Senior Director, Medical Affairs Uro-Oncology, Ferring Pharmaceuticals. "ADSTILADRIN already offers convenient, once-every-three-months dosing that helps reduce visit frequency, travel burden and clinic workflow demands. The new 25 minutes at 25°C thaw option provides additional flexibility and represents another important step in our ongoing efforts to streamline operational workflow across care settings.” About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall. 3 In the United States, bladder cancer is the sixth most common cancer, 1 fourth among men, 4 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025. 4 Historically, 75% of bladder cancer presents as NMIBC. 2 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care; however, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease. 5 Current treatment options for BCG-unresponsive patients are very limited and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder). 6 About ADSTILADRIN 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 tumors (±Ta/T1). 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 defenses against the cancer. ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, 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 and final five-year follow-up analysis published in The Journal of Urology ). 7-8 INDICATION ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product. WARNINGS AND PRECAUTIONS: Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy. Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals. DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use or oral administration. USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose. ADVERSE REACTIONS: The most common (>10%) adverse reactions; including, laboratory abnormalities (>15%), were increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition (urination urgency), increased creatinine, hematuria (blood in urine), decreased phosphate, chills, pyrexia (fever) and dysuria (painful urination). You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING. Please click to see the full Prescribing Information . About Ferring Pharmaceuticals Ferring Pharmaceuticals is a privately-owned, specialty, biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Our company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries. Ferring USA is based in Parsippany, New Jersey, and employs more than 900 employees. For more information, please visit , call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn , and X . References: National Cancer Institute. Cancer Statistics. . Accessed February 2026. Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer. Eur Urol. 2022 Jan;81(1):75-94. . Accessed February 2026. Urology Care Foundation. Non-muscle Invasive Bladder Cancer. . Accessed February 2026. American Cancer Society. Cancer Facts & Figures 2025. . Accessed February 2026. Lidagoster S, et al. BCG and Alternative Therapies to BCG Therapy for Non-Muscle-Invasive Bladder Cancer. Curr Oncol. 2024 Feb 16;31(2):1063-1078. . Accessed February 2026. National Comprehensive Cancer Network. Bladder Cancer (Version 1.2026). . Accessed March 2026. ADSTILADRIN in Patients With High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. . Accessed February 2026. Narayan VM, Boorjian SA, Alemozaffar M, et al. Efficacy of intravesical nadofaragene firadenovec for patients with Bacillus Calmette-Guérin-unresponsive nonmuscle-invasive bladder cancer: 5-year follow-up from a phase 3 trial. J Urol.2024;212(1):1-12. . Accessed February 2026. Contacts For more information, please contact: Carol Ready Director, Brand Communications (973) 765-7307 carol.ready@ferring.com