2024 Urology Times pipeline report

In 2024, the FDA authorized many new treatments and devices for urology practice. Here are the highlights.

January

Erdafitinib for FGFR3-positive urothelial carcinoma

FDA

The FDA granted full approval to erdafitinib (Balversa) for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least 1 line of prior systemic therapy, according to the FDA. The treatment is not recommended for patients who have not received treatment with PD-1/PD-L1 checkpoint inhibitors.¹

Erdafitinib previously received accelerated approval for the treatment of adult patients with FGFR2/3-positive locally advanced or metastatic urothelial carcinoma that has progressed on or after at least 1 line of platinum-containing chemotherapy, including 12 or fewer months of neoadjuvant or adjuvant platinum-containing chemotherapy.²

Transitioning an accelerated FDA approval into a full approval requires verification of the treatment’s clinical benefit in a confirmatory trial. The confirmatory trial supporting the full approval for erdafitinib was the phase 3 THOR trial (NCT03390504), which showed that erdafitinib significantly improved overall survival compared with investigator’s choice of chemotherapy in patients with FGFR2/3-altered metastatic urothelial cancer who had previously received an anti–PD-(L)1 therapy.³

February

Cefepime/enmetazobactam for complicated UTI

The FDA approved cefepime/enmetazobactam (Exblifep) for treating complicated urinary tract infections (cUTIs), including pyelonephritis, according to a news release from Allecra Therapeutics, which maintains sole responsibility for the therapy.⁴

The FDA has also granted Allecra a 5-year marketing exclusivity extension for cefepime/enmetazobactam as part of the Generating Antibiotic Incentives Now (GAIN) Act. The GAIN Act provides benefits to manufacturers of qualified infectious disease products to encourage the creation of anti-infective therapeutics.

The FDA approval is supported by data from the phase 3 ALLIUM trial (NCT03687255), which demonstrated the noninferiority and superiority of cefepime/enmetazobactam vs piperacillin/tazobactam regarding the primary composite outcome measure of clinical cure and microbiological eradication in patients with cUTIs.^5,6

Nivolumab plus cisplatin/gemcitabine in urothelial carcinoma

The FDA approved nivolumab (Opdivo) in combination with frontline cisplatin and gemcitabine for the treatment of adult patients with unresectable or metastatic urothelial carcinoma.⁷ The approval is based on data from the phase 3 CheckMate901 trial (NCT03036098), which demonstrated improved overall survival (OS) and progression-free survival with the nivolumab-combination therapy vs cisplatin/gemcitabine alone in this patient population.⁸

At a median follow-up of 33 months, nivolumab in combination with cisplatin and gemcitabine, followed by nivolumab maintenance therapy, led to a 22% reduction in the risk of death, demonstrating a median OS of 21.7 months (95% CI, 18.6-26.4) vs 18.9 months (95% CI, 14.7-22.4) with cisplatin and gemcitabine alone (HR, 0.78; 95% CI, 0.63, 0.96; P =.0171).

March

EdgeFlow UH10 Bladder Scanner

The FDA granted 510(k) clearance to the ultrasound bladder scanner EdgeFlow UH10, announced Edgecare, the technology developer, in a news release.⁹ According to Edgecare, the EdgeFlow UH10 bladder scanner utilizes high-resolution ultrasound imaging to measure postvoid residual urine in the bladder noninvasively. The scanner offers a volume measurement range of 0 to 999 mL, with accuracy plus or minus 7.5% (over 100 mL).¹⁰

The device is equipped with DualActive probe technology, which allows simultaneous scanning of the bladder’s traverse and sagittal planes with 1 scan. Additionally, the tool incorporates deep learning artificial intelligence (AI)–powered LiveContour and MaxContour technologies to quickly identify the bladder and provide fast calculations of accurate measurement values of maximum bladder volume.

The device also offers a lightweight and compact design that facilitates portability. The total weight of the console and probe is 419 g.

April

RenaFlex ureteroscope system

The FDA granted 510(k) clearance to the RenaFlex single-use flexible ureteroscope system for treating patients with urinary diseases such as kidney stones, announced Olympus, the system developer, in a news release.¹¹ The RenaFlex ureteroscope system is intended for accessing and visualizing the urinary tract (the urethra, bladder, ureter, calyces, and renal papillae) via transurethral or percutaneous access routes. The ureteroscope is designed to work with the compatible CV-S1 video system center for flexible single-use endoscopes.

Nogapendekin alfa inbakicept plus BCG for NMIBC

The FDA approved N-803 (Anktiva, nogapendekin alfa inbakicept-pmln), a first-in-class IL-15 receptor agonist immunotherapy, for use in combination with BCG for the treatment of patients with BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors, announced ImmunityBio, the developer of the therapy, in a news release.¹²

The approval is supported by findings from cohort B of the ongoing phase 2/3 QUILT-3.032 trial (NCT03022825), assessing BCG in combination with N-803 in patients with BCG-unresponsive, high-grade Ta/T1 papillary NMIBC. Patients in cohort B received N-803 in combination with BCG maintenance therapy for up to 37 months.

Data from the trial have shown a complete response (CR) of 62% to 73% among the 77 evaluable patients included in the analysis. At the time of the data cutoff, the duration of CR was longer than 47 months, exceeding the threshold for meaningful clinical results per a panel of experts at the International Bladder Cancer Group.

Pivmecillinam tablets for uncomplicated UTIs

The FDA approved oral pivmecillinam (Pivya) tablets for the treatment of adult female patients with uncomplicated UTIs (uUTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis, and Staphylococcus saprophyticus.¹³ The antibiotic’s approval is based on data from 3 clinical trials evaluating the efficacy of different pivmecillinam dosing regimens compared with placebo, another oral antibacterial drug, or ibuprofen. These studies all enrolled adult female patients with uUTIs.

May

AI companion tool for TULSA-PRO system

The FDA granted 510(k) clearance to the Contouring Assistant, a machine-learning–based prostate segmentation tool designed for use in conjunction with the TULSA-PRO system to assist in delineating the prostate and target ablation volume for transurethral ultrasound ablation (TULSA) procedure treatment planning.¹⁴ The Contouring Assistant utilizes a large database of successful physician-created TULSA treatment designs to recommend a treatment plan based on learned patterns. Specifically, the tool uses 24,611,072 parameters extracted from 7466 training images.

Yōni.Fit Bladder Support for SUI

The FDA granted 510(k) clearance to Yōni.Fit Bladder Support, a vaginal insert for adult patients with urine leakage due to stress urinary incontinence (SUI), announced Watkins-Conti Products, the developer of the device, in a news release.¹⁵

Yōni.Fit is a soft vaginal insert intended to reduce urine leakage due to SUI without interfering with voluntary urination. The device is self-administered and can be used during activity or for up to 12 hours for general symptom control.

The FDA clearance of the device was supported by findings from a randomized, controlled trial (NCT03978741) that evaluated the efficacy, user acceptance, safety, and tolerability of the Yōni.Fit device vs a comparator device for the temporary management of SUI.

Overall, data from the study showed that a significantly higher percentage of patients experienced a clinically meaningful reduction in 12-hour pad weights with the Yōni.Fit device than with the comparator device (53.0% vs 23.3%; P =.013; absolute difference, 29.6%; 95% CI, 7.4%-51.8%).¹⁶

July

FoundationOneLiquid CDx for niraparib/abiraterone in mCRPC

The FDA approved the liquid biopsy test FoundationOneLiquid CDx for use as a companion diagnostic in identifying patients with BRCA-positive metastatic castration-resistant prostate cancer (mCRPC) who may benefit from treatment with niraparib and abiraterone acetate dual action tablets (Akeega), Foundation Medicine announced in a news release.¹⁷

This announcement follows an earlier FDA decision to approve the company’s tissue-based comprehensive genomic profiling test FoundationOne CDx as a companion diagnostic for the same therapy and indication in August 2023.¹⁸ Niraparib and abiraterone acetate is a dual-action tablet approved for the treatment of adult patients with deleterious or suspected deleterious BRCA-mutated mCRPC.

Senhance Surgical System in urology

The FDA granted 510(k) clearance to an expanded indication of the Senhance Surgical System for use in adult and pediatric patients undergoing urologic procedures, announced Asensus Surgical, the device developer, in a news release.¹⁹ According to Asensus, the Senhance Surgical System is a digital laparoscopy system “intended to assist in the accurate control of laparoscopic instruments for visualization and endoscopic manipulation of tissue including grasping, cutting, blunt and sharp dissection, approximation, ligation, electrocautery, suturing, mobilization, and retraction.”²⁰

The device utilizes augmented intelligence and deep learning technologies to increase surgeon control and reduce variability, making it the first intraoperative augmented intelligence technology approved for robotic surgery, the company noted.

August

AI-powered robotic system for Aquablation therapy

The FDA granted 510(k) clearance to the HYDROS robotic system, an AI-powered platform designed to enhance the delivery of Aquablation therapy for patients with benign prostatic hyperplasia, PROCEPT BioRobotics, the developer of the platform, announced in a news release.²¹ According to the company, key features of the HYDROS robotic system include FirstAssist AI treatment planning, advanced image guidance, robotic resection, and a streamlined workflow. FirstAssist AI is built on data from over 50,000 real-world Aquablation procedures and uses image recognition software to provide an optimal treatment plan for each patient. The system integrates ultrasound and cystoscopy images to offer a multidimensional view of the entire prostate, enabling individualized treatment planning based on patients’ anatomy.

September

Companion diagnostic for olaparib/abiraterone in BRCA+ mCRPC

The FDA approved the tissue-based FoundationOne CDx and blood-based FoundationOneLiquid CDx tests for use as companion diagnostics in identifying patients with deleterious or suspected deleterious BRCA-mutated mCRPC who may benefit from treatment with olaparib (Lynparza) in combination with abiraterone acetate (Zytiga) and prednisone or prednisolone, Foundation Medicine announced in a news release.²²

October

Ambu’s HD cystoscopy solution

The FDA granted 510(k) clearance to Ambu’s HD cystoscopy solution, consisting of the company’s HD single-use cystoscope, the Ambu aScope 5 Cysto HD, and its 2 full-HD endoscopy systems, Ambu aView 2 Advance and Ambu aBox 2, the company announced in a news release.²³

In October 2023, the cystoscopy solution was granted a CE mark for regulatory clearance in Europe. The company is planning a commercial launch of the solution in the US, designed to support urologists performing diagnostic and therapeutic ureteroscopy procedures.

Oral sulopenem for uUTI

The FDA has granted approval to oral sulopenem (sulopenem etzadroxil and probenecid; Orlynvah) for the treatment of uUTIs caused by the designated microorganisms Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options, Iterum Therapeutics announced in a news release.²⁴ According to the company, oral sulopenem is the first oral penem approved for use in the US.

The approval is supported by data from the phase 3 SURE 1 (NCT03366207) and REASSURE (NCT05584657) trials. In SURE 1, oral sulopenem demonstrated superiority over ciprofloxacin in the treatment of patients with fluoroquinolone-resistant infections and noninferiority to ciprofloxacin in the treatment of patients with a quinolone-susceptible uropathogen. In REASSURE, the oral penem demonstrated noninferiority to oral amoxicillin/clavulanate in treating patients with uUTI.

November

Stone Clear device for kidney stones

The FDA has granted de novo clearance to the Stone Clear device, which delivers non-invasive ultrasound pulses to patients to facilitate passage of residual kidney stone fragments post-lithotripsy, SonoMotion announced in a news release.²⁵

Stone Clear is designed to manage kidney stones in the clinic setting without requiring anesthesia.

Data on the safety and effectiveness of ultrasonic propulsion were recently published in The Journal of Urology.²⁶ Overall, the prospective, open-label, randomized, controlled study (NCT02028559) showed that ultrasonic propulsion reduced relapse (defined as stone growth, a stone-related urgent medical visit, or surgery) and added minimal risk in facilitating kidney stone fragment clearance compared with observation.

December

NanoKnife System for prostate tissue ablation

The FDA has granted 510(k) clearance to the NanoKnife System for use in prostate tissue ablation, AngioDynamics announced in a news release.²⁵ The NanoKnife System, which utilizes irreversible electroporation (IRE) technology, was previously approved in the US for use in soft tissue ablation. The current 510(k) clearance grants a more specific indication for ablation of prostate tumors.

The clearance is supported by data from the PRESERVE trial (NCT04972097), which evaluated the safety and effectiveness of the NanoKnife System in ablating prostate tissue in patients with intermediate-risk prostate cancer. Overall, the PRESERVE trial met its primary efficacy end point by demonstrating a negative in-field biopsy rate of 84% at 12 months following treatment with the NanoKnife System.

Vibegron for OAB symptoms in pharmacologically treated BPH

The FDA has approved vibegron (Gemtesa) for the treatment of patients with overactive bladder (OAB) symptoms who are receiving pharmacological treatment for benign prostatic hyperplasia (BPH), Sumitomo Pharma America (SMPA) announced in a news release.²⁶ According to the company, this approval marks the first and only beta-3 (β₃) agonist approved for this indication. Vibegron was previously approved in the US for the treatment of adult patients with OAB with symptoms of urge urinary incontinence, urgency, and urinary frequency.

Subcutaneous nivolumab for solid tumors, including RCC

The FDA has approved nivolumab (Opdivo) plus hyaluronidase-nvhy (Qvantig) for subcutaneous injection across most previously approved solid tumor indications for nivolumab as a monotherapy, monotherapy maintenance following completion of combination therapy with nivolumab plus ipilimumab (Yervoy), or in combination with chemotherapy or cabozantinib (Cabometyx).²⁷

Nivolumab was previously approved under several indications in renal cell carcinoma and urothelial carcinoma.

The approval is supported by findings from the phase 3 CheckMate-67T trial (NCT04810078), in which subcutaneous nivolumab plus hyaluronidase-nvhy met the study’s co-primary end points by demonstrating noninferiority to IV nivolumab regarding time average nivolumab serum concentration over 28 days (C_avgd28; geometric mean ratio [GMR}, 2.098; 90% CI, 2.001-2.200) and minimum serum concentration at a steady state (C_minss; GMR, 1.774; 90% CI, 1.633-1.927).² For both pharmacokinetic end points, the predefined acceptance margin was met, with the lower boundary of the 90% confidence interval of geometric mean ratios not reaching less than 0.8.

REFERENCES

1. FDA approves erdafitinib for locally advanced or metastatic urothelial carcinoma. FDA. January 19, 2024. Accessed January 19, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-erdafitinib-locally-advanced-or-metastatic-urothelial-carcinoma

2. Janssen submits supplemental new drug application to the U.S. Food and Drug Administration seeking full approval of BALVERSA (erdafitinib) for the treatment of patients with locally advanced or metastatic urothelial carcinoma and selected fibroblast growth factor receptor gene alterations. Accessed August 29, 2023. https://finance.yahoo.com/news/janssen-submits-supplemental-drug-application-201500611.html

3. Loriot Y, Matsubara N, Park SH, et al. Phase 3 THOR study: results of erdafitinib (erda) versus chemotherapy (chemo) in patients (pts) with advanced or metastatic urothelial cancer (mUC) with select fibroblast growth factor receptor alterations (FGFRalt). J Clin Oncol. 2023;41(suppl 17):LBA4619. doi:10.1200/JCO.2023.41.17_suppl.LBA4619

4. Allecra Therapeutics announces U.S. FDA approval for EXBLIFEP for the treatment of complicated urinary tract infections. News release. Allecra Therapeutics. February 27, 2024. Accessed February 27, 2024. https://www.businesswire.com/news/home/20240227132611/en/Allecra-Therapeutics-Announces-U.S.-FDA-Approval-for-EXBLIFEP%C2%AE-for-the-Treatment-of-Complicated-Urinary-Tract-Infections

5. Kaye KS, Belley A, Barth P, et al. Effect of cefepime/enmetazobactam vs piperacillin/tazobactam on clinical cure and microbiological eradication in patients with complicated urinary tract infection or acute pyelonephritis: a randomized clinical trial. JAMA. 2022;328(13):1304-1314. doi:10.1001/jama.2022.17034

6. Allecra Therapeutics publishes final phase 3 ALLIUM data in JAMA: cefepime/enmetazobactam met criteria for superiority. News release. Allecra Therapeutics. May 10, 2022. Accessed February 27, 2024. https://www.allecra.com/

7. FDA approves nivolumab in combination with cisplatin and gemcitabine for unresectable or metastatic urothelial carcinoma. FDA. Updated March 7, 2024. Accessed March 7, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-combination-cisplatin-and-gemcitabine-unresectable-or-metastatic-urothelial

8. van der Heijden MS, Sonpavde G, Powles T, et al; CheckMate 901 Trial Investigators. Nivolumab plus gemcitabine-cisplatin in advanced urothelial carcinoma. N Engl J Med. 2023;389(19):1778-1789. doi:10.1056/NEJMoa2309863

9. Edgecare Inc. obtains U.S. FDA clearance for ultrasound bladder scanner EdgeFlow UH10. News release. Edgecare Inc. March 7, 2024. Accessed March 20, 2024. https://www.edgecare.co.kr/bbs/board.php?bo_table=gallery&wr_id=29

10. EdgeFlow UH10. Product brochure. Edgecare. https://www.edgecare.co.kr/bbs/board.php?bo_table=bladder

11. Olympus announces FDA clearance of RenaFlex, its first single-use flexible ureteroscope. News release. Olympus. April 2, 2024. Accessed April 2, 2024. https://www.olympusamerica.com/press-release/2024-04-02/olympus-announces-fda-clearance-renaflextm-its-first-single-use-flexible

12. ImmunityBio announces FDA approval of ANKTIVA, first-in-class IL-15 receptor agonist for BCG-unresponsive non-muscle invasive bladder cancer. News release. ImmunityBio. April 22, 2024. Accessed April 23, 2024. https://immunitybio.com/immunitybio-announces-fda-approval-of-anktiva-first-in-class-il-15-receptor-agonist-for-bcg-unresponsive-non-muscle-invasive-bladder-cancer/

13. FDA approves new treatment for uncomplicated urinary tract infections. News release. FDA. April 29, 2024. Accessed April 24, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-uncomplicated-urinary-tract-infections

14. Profound Medical receives U.S. FDA 510(k) clearance for ‘Contouring Assistant’ AI module that enables creation of an automated TULSA treatment plan. News release. Profound Medical Corp. May 14, 2024. Accessed May 14, 2024. https://www.biospace.com/article/releases/profound-medical-receives-u-s-fda-510-k-clearance-for-contouring-assistant-ai-module-that-enables-creation-of-an-automated-tulsa-treatment-plan/

15. Women’s healthcare company Watkins-Conti receives FDA 510(k) clearance for new stress urinary incontinence device Yōni.Fit. News release. Watkins-Conti Products, Inc. May 17, 2024. Accessed May 20, 2024. https://www.prnewswire.com/news-releases/womens-healthcare-company-watkins-conti-receives-fda-510k-clearance-for-new-stress-urinary-incontinence-device-ynifit-302149332.html

16. Randomized controlled trial evaluating efficacy and safety of a novel stress incontinence device. Presented at: PFD Week 2023; October 4-6, 2023; Portland, OR.

17. U.S. Food and Drug Administration (FDA) approves FoundationOneLiquid CDx as a companion diagnostic for AKEEGA (niraparib and abiraterone acetate) for patients with BRCA-positive metastatic castration-resistant prostate cancer. News release. Foundation Medicine, Inc. July 1, 2024. Accessed July 1, 2024. https://www.foundationmedicine.com/press-release/fda-approval-foundationone-liquid-cdx-akeega

18. U.S. Food and Drug Administration (FDA) approves FoundationOneCDx as a companion diagnostic for Janssen’s AKEEGA (niraparib and abiraterone acetate dual action tablet) for patients with BRCA-positive metastatic castration-resistant prostate cancer. News release. Foundation Medicine, Inc. August 15, 2023. Accessed July 1, 2024. https://www.foundationmedicine.com/press-releases/u.s.-food-and-drug-administration-%28fda%29-approves-foundationone%C2%AEcdx-as-a-companion-diagnostic-for-janssen%E2%80%99s-akeega%E2%84%A2-%28niraparib-and-abiraterone-acetate-dual-action-tablet%29-for-patients-with-brca-po

19. Asensus Surgical receives FDA 510(k) clearance for Senhance Surgical System in urology. News release. Asensus Surgical, Inc. July 23, 2024. Accessed July 23, 2024. https://ir.asensus.com/news-releases/news-release-details/asensus-surgical-receives-fda-510k-clearance-senhance-surgical

20. Indications for use. Asensus Surgical. Accessed July 23, 2024. https://www.asensus.com/indications-for-use

21. PROCEPT BioRobotics announces FDA clearance of the HYDROS robotic system, the next-generation, AI-powered platform for Aquablation therapy. News release. PROCEPT BioRobotics. August 21, 2024. Accessed August 21, 2024. https://ir.procept-biorobotics.com/news-releases/news-release-details/procept-biorobotics-announces-fda-clearance-hydrostm-robotic

22. U.S. Food and Drug Administration approves FoundationOne CDx and FoundationOneLiquid CDx as companion diagnostics for Lynparza (olaparib) in combination with abiraterone for patients with BRCA-mutated metastatic castration-resistant prostate cancer. News release. Foundation Medicine, Inc. September 3, 2024. Accessed September 3, 2024. https://www.foundationmedicine.com/press-release/fda-approval-cdxs-lynparza-brca-mcrpc

23. Ambu launches HD cystoscopy solution in the U.S. News release. Ambu. October 17, 2024. Accessed October 23, 2024. https://www.ambu.com/Files/Files/Ambu/Investor/News/English/2024/Ambu-launches-HD-cystoscopy-solution-in-the-US.pdf

24. Iterum Therapeutics receives U.S. FDA approval of ORLYNVAH (oral sulopenem) for the treatment of uncomplicated urinary tract infections. News release. Iterum Therapeutics. October 25, 2024. Accessed October 25, 2024. https://www.iterumtx.com/news/press-releases/detail/136/iterum-therapeutics-receives-u-s-fda-approval-of

25. AngioDynamics receives FDA clearance for the NanoKnife System for prostate tissue ablation. News release. AngioDynamics, Inc. Published online and accessed December 9, 2024. https://www.businesswire.com/news/home/20241209958174/en/AngioDynamics-Receives-FDA-Clearance-for-The-NanoKnife%C2%AE-System-for-Prostate-Tissue-Ablation

26. Sumitomo Pharma America announces U.S. FDA approval of GEMTESA (vibegron) for men with overactive bladder symptoms receiving pharmacological therapy for benign prostatic hyperplasia. News release. Sumitomo Pharma America. Published online and accessed December 23, 2024. https://news.us.sumitomo-pharma.com/2024-12-23-Sumitomo-Pharma-America-Announces-U-S-FDA-Approval-of-GEMTESA-R-vibegron-for-Men-with-Overactive-Bladder-Symptoms-Receiving-Pharmacological-Therapy-for-Benign-Prostatic-Hyperplasia

27. FDA approves nivolumab and hyaluronidase-nvhy for subcutaneous injection. News release. Published online and accessed December 27, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-and-hyaluronidase-nvhy-subcutaneous-injection