UroGen Initiates Submission of a Rolling NDA to the FDA for UGN-102
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Insights
The submission of the Chemistry, Manufacturing and Controls (CMC) section for UGN-102 represents a crucial phase in UroGen Pharma's regulatory pathway. The CMC is a comprehensive documentation that details the drug's production process, quality control measures and consistency in manufacturing. The thoroughness of this section is paramount as it assures the FDA of the drug's safety and efficacy from a production standpoint.
From a research perspective, the acceptance of the CMC by the FDA can be seen as a testament to UroGen's rigorous scientific approach and its adherence to stringent regulatory standards. The successful review of this section could potentially smooth the path for subsequent clinical data submissions. For stakeholders, this progress signifies a step closer to market entry, which could translate into revenue generation and a positive impact on UroGen's financials in the long-term, especially considering the current lack of innovative therapies for low-grade, intermediate-risk non-muscle invasive bladder cancer.
UGN-102's advancement in the regulatory process is a promising development within the field of uro-oncology. Non-muscle invasive bladder cancer (NMIBC), particularly the low-grade, intermediate-risk category, has limited treatment options beyond surgery and traditional chemotherapy. Intravesical therapy, which involves the direct instillation of the therapeutic agent into the bladder, could offer a less invasive and potentially more effective treatment modality for patients. Mitomycin, the active ingredient in UGN-102, has been used in various formulations for bladder cancer, but UGN-102's specific formulation could offer improvements in terms of efficacy or safety profiles.
For patients, the potential approval of UGN-102 could mean access to a novel treatment that may reduce the need for more invasive procedures like cystectomy, thus improving quality of life. For the healthcare system, it could mean a shift in the standard of care for NMIBC, which could lead to changes in treatment protocols and healthcare spending in this domain.
In evaluating the impact of UroGen Pharma's submission on the stock market, it's important to consider the competitive landscape of bladder cancer treatments. Currently, the market is characterized by a high demand for innovative therapies and the introduction of UGN-102 could disrupt current treatment paradigms. The anticipation of a new drug application can often lead to positive investor sentiment, potentially driving up UroGen's stock in the short-term.
However, the full impact on the company's valuation will depend on the eventual approval and commercial success of UGN-102. Factors such as market size, adoption rate and reimbursement policies will be critical in determining the long-term financial implications. For investors, the rolling submission strategy also suggests a proactive engagement with the FDA, which might be perceived as reducing regulatory risk and could be factored into investment decisions. It's crucial to monitor UroGen's progress and any feedback from the FDA as these will be key indicators of the drug's future prospects.
"The submission of the CMC portion of the NDA for UGN-102 marks a significant milestone for UroGen and underscores our dedication to advancing innovative therapies for the benefit of individuals grappling with low-grade, intermediate-risk non-muscle invasive bladder cancer,” said Liz Barrett, President and CEO, UroGen. “We look forward to working closely with the FDA throughout the review process and remain steadfast in our commitment to address unmet medical needs in the uro-oncology space and advance patient care."
The objective of the rolling NDA for UGN-102 is to facilitate early engagement with the FDA, and a more efficient and timely review of the NDA. Based on its agreement with the FDA, UroGen will complete the submission of the rolling NDA for UGN-102 in 2024 with a potential FDA decision as early as the first quarter of 2025.
The CMC section of a regulatory submission typically includes information about the drug product such as its physicochemical properties, formulation, methods of manufacture, specifications, stability data, and analytical methods used to test the product.
About UGN-102
UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of low-grade, intermediate-risk, non-muscle invasive bladder cancer (LG-IR-NMIBC). Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting. Assuming positive findings from the durability of response endpoint from the ENVISION Phase 3 study, UroGen anticipates completing its NDA submission for UGN-102 in 2024 with a potential FDA decision as early as the first quarter of 2025.
About UroGen Pharma Ltd.
UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with low-grade non-muscle invasive bladder cancer are designed to ablate tumors by non-surgical means. UroGen is headquartered in
Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: the planned completion of the NDA submission for UGN-102 and the timing thereof; the timing for an FDA decision on UGN-102; the potential for UGN-102 to introduce a new non-surgical treatment paradigm for LG-IR-NMIBC and the potential of UroGen’s proprietary RTGel technology to improve therapeutic profiles of existing drugs and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as “anticipate,” “assume,” “potential,” “will,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: preliminary results may not be indicative of results that may be observed in the future; the timing and success of clinical trials and potential safety and other complications thereof; unforeseen delays that may impact the timing of progressing clinical trials and reporting data; the ability to obtain regulatory approval within the timeframe expected, or at all; the findings from the durability of response endpoint from the ENVISION Phase 3 study may not be positive, and in such event, our NDA pathway could be negatively impacted; even if the durability of response endpoint data from the ENVISION Phase 3 study are positive there is no guarantee that the current clinical development plan for UGN-102 will ultimately support submission of an NDA, notwithstanding the current agreement with the FDA; even if an NDA for UGN-102 is accepted by the FDA, there is no guarantee that such NDA will be sufficient to support approval of UGN-102 on the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) for our product and product candidates and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen’s ability to attract or retain key management, members of the board of directors and personnel; UroGen’s RTGel technology may not perform as expected; UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology; and UroGen’s financial condition and need for additional capital in the future. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, filed with the SEC on November 14, 2023 (which is available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.
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INVESTOR CONTACT:
Vincent Perrone
Senior Director, Investor Relations
vincent.perrone@urogen.com
609-460-3588 ext. 1093
MEDIA CONTACT:
Cindy Romano
Director, Corporate Communications
cindy.romano@urogen.com
609-460-3583 ext. 1083
Source: UroGen Pharma Ltd.
FAQ
What is the New Drug Application (NDA) submitted by UroGen Pharma Ltd. (URGN)?
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