Latest news with #ODAC
Medscape
23-05-2025
- Health
- Medscape
FDA Questions Single-Arm Studies for Cancer Approvals
The US Food and Drug Administration's (FDA's) approval decision about a new treatment for non–muscle invasive bladder cancer (NMIBC) hinges on whether a single-armed trial can be trusted to adequately demonstrate risks and benefits. The issue came to a head May 21 when the agency asked its Oncologic Drugs Advisory Committee (ODAC) to weigh in on UGN-102 (UroGen Pharma), an intravesical mitomycin formulation meant to be an alternative to transurethral resection of bladder tumor (TURBT) for recurrent low-grade intermediate-risk NMIBC. UroGen presented results from its ENVISION trial in 223 patients with recurrent lesions on day 2 of a meeting of the FDA advisory committee. After 6 weekly 75 mg instillations, the complete response rate at the 3-month checkup was 77.6%. Twelve months after a complete response, almost 80% of subjects remained recurrence-free; at 18 months, just over half were still recurrence-free. UroGen highlighted the convenience of UGN-102, which can be instilled in a urologist's office, over TURBT, which is a surgical procedure usually done under general anesthesia. The company said UGN-102 'can reduce the burden of repeated TURBTs' in elderly, comorbid patients. However, there was no control arm in ENVISION, so outcomes vs TURBT, the current standard of care, are unclear. 'The lack of a concurrent control in the single-arm ENVISION trial makes interpretation of efficacy challenging,' the FDA said in a meeting document. Although the complete response rate indicates drug activity, the agency said the durations of response found in the study could simply be due to the fact that some patients with NMBIC recur, while others do not. The same holds true for safety. Most of the adverse events in the trial were genitourinary and low grade, but it's unclear if there are fewer than with TURBT. For one, patients were at risk for adverse events throughout the entire 6-week treatment window, whereas the duration of side effects with TURBT, as a single procedure, is generally shorter. 'The applicant has not demonstrated that treatment with UGN-102 is safer or more tolerable than TURBT,' the FDA said in the document. The FDA wanted UroGen to run a head-to-head randomized trial against TURBT. UroGen started one, but there was disagreement with the agency about how it should be conducted, and the trial ended early. How Committee Members Voted on Benefits vs Risks for UGN-102 On day 2 of the ODAC meeting, the FDA said the preliminary findings can't be used to compare the two approaches due to methodological issues. Richard Pazdur, MD, head of FDA's Oncology Center of Excellence, said there are also larger issues at play. If the agency approves UGN-102 for such a condition as common as NMIBC based on a single-arm study, 'guess what? Nobody's going to do a randomized trial. It goes to the lowest denominator for companies,' Pazdur said. The FDA didn't ask ODAC's opinion on approving UGN-102. Instead, it simply wanted committee members to vote on whether the overall benefit-risk of UGN-102 is favorable for recurrent, low-grade, intermediate-risk NMIBC based on the data. Five panelists voted 'no,' and four voted 'yes.' Radiation oncologist Daniel Spratt, MD, a prostate cancer specialist at Case Western Reserve University, Cleveland, was one of the 'no' votes. Without the randomized trial, he said, 'It's very hard to determine the true benefit of this, and there is toxicity.' Plus, 'this is a $140,000 treatment that might not change how many follow-up TURBTs you need after 3 months.' Another 'no' vote, Heidi McKean, MD, a community oncologist in Sioux Falls, South Dakota, said that 'in this population, a randomized control trial is feasible and would have really helped us understand the clinical meaningfulness of the intervention.' Surgical oncologist Mark Ball, MD, a kidney cancer specialist at the National Cancer Institute in Bethesda, Maryland, voted 'yes' in favor of UGN-102. The complete response rate at 12 months and beyond, 'even though there are differences in the interpretation of the data, is quite encouraging. I don't find the toxicity profile alarming, and therefore,' Ball said, 'the benefit-risk ratio is favorable.' Isla Garraway, MD, PhD, a urologic surgical oncologist at the University of California, Los Angeles, also voted 'yes,' saying it would be ideal to have a less invasive, in-office alternative to TURBT for the older patients most often affected by NMIBC. In the end, Pazdur said, 'This a relatively split vote, so we will be meeting with the sponsor to look at future directions.' ODAC was also asked for its thoughts on whether the FDA should require randomized trials for future low-grade, intermediate-risk NMIBC approval submissions. Committee members generally agreed, as long as the requirement doesn't delay the approval of promising treatments.
Yahoo
21-05-2025
- Business
- Yahoo
UroGen Announces Outcome of Oncologic Drugs Advisory Committee for UGN-102 for the Treatment of Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC)
PRINCETON, N.J., May 21, 2025--(BUSINESS WIRE)--UroGen Pharma Ltd. (Nasdaq: URGN), a leading biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, announced the outcome of today's meeting of the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA), which discussed the new drug application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution. By a narrow margin, the ODAC voted 4 to 5 that the benefit/risk of UGN-102 (mitomycin) for intravesical solution was favorable for the treatment of recurrent LG-IR-NMIBC. "While we are disappointed by today's outcome, we continue to believe our clinical data support UGN-102 for the treatment of recurrent LG-IR-NMIBC, a disease with no FDA-approved therapies," said Liz Barrett, President and CEO of UroGen. "The FDA carefully considers the independent advice from ODAC, and we look forward to working with the FDA as they complete their review of the application for UGN-102." The ODAC reviewed the body of clinical data supporting the efficacy and safety of UGN-102, including the results from the Phase 3 ENVISION study. "Low-grade intermediate-risk non-muscle invasive bladder cancer is a highly recurrent disease and often requires patients – many of whom are elderly – to undergo repeat surgeries under general anesthesia. This is a disease with high unmet needs, and we believe patients deserve more options," said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. "UroGen remains committed to developing innovative treatment options to people living with recurrent LG-IR-NIMBC." The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention, which are typically manageable in routine urologic practice. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102. The NDA for UGN-102 is currently under review by the FDA with a Prescription Drug User Fee Act (PDUFA) date of June 13, 2025. 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 recurrent 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 by a trained healthcare professional. UroGen completed the submission of the rolling NDA for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA target action date of June 13, 2025. About Non-Muscle Invasive Bladder Cancer (NMIBC) LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include trans-urethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at About ENVISION The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at three months after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at (NCT05243550). 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. UroGen's first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with recurrent LG-IR-NMIBC are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit to learn more or follow us on X (Twitter), @UroGenPharma. 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 potential for UGN-102 as the first and only FDA-approved treatment for recurrent LG-IR-NMIBC; statements related to UroGen's UGN-102 NDA and expected PDUFA target action date; the estimated annual U.S. patient population and demographics for LG-IR-NMIBC; the potential patient benefits and opportunities for UGN-102, if approved; 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 "expect," "if," "potential," 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 ability to obtain regulatory approval within the timeframe expected, or at all; although the FDA is not bound by the ODAC's recommendation, the recommendation may adversely impact the FDA's decision on the NDA for UGN-102; the PDUFA target action date may be delayed due to various factors outside UroGen's control; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; the ability to obtain and maintain regulatory approval within the timeframe expected, or at all; complications associated with commercialization activities; labeling limitations; competition in UroGen's industry; the scope, progress and expansion of developing and commercializing UroGen's product and product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies or procedures, such as surgery; UroGen's ability to attract or retain key management, members of the board of directors and other personnel; UroGen's RTGel technology may not perform as expected; and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology. 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 March 31, 2025, filed with the SEC on May 12, 2025. 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. View source version on Contacts INVESTOR CONTACT: Vincent PerroneSenior Director, Investor 609-460-3588 ext. 1093 MEDIA CONTACT: Cindy RomanoDirector, 908-963-7827 Sign in to access your portfolio
Business Wire
21-05-2025
- Business
- Business Wire
UroGen Announces Outcome of Oncologic Drugs Advisory Committee for UGN-102 for the Treatment of Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC)
PRINCETON, N.J.--(BUSINESS WIRE)--UroGen Pharma Ltd. (Nasdaq: URGN), a leading biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, announced the outcome of today's meeting of the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA), which discussed the new drug application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution. By a narrow margin, the ODAC voted 4 to 5 that the benefit/risk of UGN-102 (mitomycin) for intravesical solution was favorable for the treatment of recurrent LG-IR-NMIBC. 'While we are disappointed by today's outcome, we continue to believe our clinical data support UGN-102 for the treatment of recurrent LG-IR-NMIBC, a disease with no FDA-approved therapies,' said Liz Barrett, President and CEO of UroGen. 'The FDA carefully considers the independent advice from ODAC, and we look forward to working with the FDA as they complete their review of the application for UGN-102.' The ODAC reviewed the body of clinical data supporting the efficacy and safety of UGN-102, including the results from the Phase 3 ENVISION study. 'Low-grade intermediate-risk non-muscle invasive bladder cancer is a highly recurrent disease and often requires patients – many of whom are elderly – to undergo repeat surgeries under general anesthesia. This is a disease with high unmet needs, and we believe patients deserve more options,' said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. 'UroGen remains committed to developing innovative treatment options to people living with recurrent LG-IR-NIMBC.' The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention, which are typically manageable in routine urologic practice. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102. The NDA for UGN-102 is currently under review by the FDA with a Prescription Drug User Fee Act (PDUFA) date of June 13, 2025. 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 recurrent 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 by a trained healthcare professional. UroGen completed the submission of the rolling NDA for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA target action date of June 13, 2025. About Non-Muscle Invasive Bladder Cancer (NMIBC) LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include trans-urethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at About ENVISION The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at three months after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at (NCT05243550). 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. UroGen's first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with recurrent LG-IR-NMIBC are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit to learn more or follow us on X (Twitter), @UroGenPharma. 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 potential for UGN-102 as the first and only FDA-approved treatment for recurrent LG-IR-NMIBC; statements related to UroGen's UGN-102 NDA and expected PDUFA target action date; the estimated annual U.S. patient population and demographics for LG-IR-NMIBC; the potential patient benefits and opportunities for UGN-102, if approved; 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 'expect,' 'if,' 'potential,' 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 ability to obtain regulatory approval within the timeframe expected, or at all; although the FDA is not bound by the ODAC's recommendation, the recommendation may adversely impact the FDA's decision on the NDA for UGN-102; the PDUFA target action date may be delayed due to various factors outside UroGen's control; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; the ability to obtain and maintain regulatory approval within the timeframe expected, or at all; complications associated with commercialization activities; labeling limitations; competition in UroGen's industry; the scope, progress and expansion of developing and commercializing UroGen's product and product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies or procedures, such as surgery; UroGen's ability to attract or retain key management, members of the board of directors and other personnel; UroGen's RTGel technology may not perform as expected; and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology. 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 March 31, 2025, filed with the SEC on May 12, 2025. 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.
Yahoo
20-05-2025
- Business
- Yahoo
U.S. FDA Oncologic Drugs Advisory Committee votes in favor of the benefit-risk profile of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) for high-risk smoldering multiple myeloma
ODAC recommendation based on the positive progression-free survival and clinical benefit in the Phase 3 AQUILA study If approved, DARZALEX FASPRO® would be the first treatment to potentially delay or prevent progression to multiple myeloma RARITAN, N.J., May 20, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) announced today the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) voted (6-2) in favor of the benefit-risk profile of single-agent DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM). An application for the approval of DARZALEX FASPRO® for adult patients with HR-SMM was submitted to the FDA in November 2024. The vote highlights a pivotal moment in the care of patients most likely to develop active multiple myeloma (MM), potentially altering the course of disease and treatment. DARZALEX FASPRO® is a foundational therapy in MM, and if approved in this indication, would provide a potential path for earlier intervention. No treatments are approved specifically to treat HR-SMM. In 2024, it was estimated that more than 35,000 people would be diagnosed with MM in the U.S., and approximately 15 percent of newly diagnosed MM cases are classified as smoldering. While patients diagnosed with HR-SMM are asymptomatic, approximately 50 percent are likely to develop active disease within two to three years. The current standard of care (SOC) for smoldering multiple myeloma (SMM), even those considered high-risk, is active monitoring ("Watch and Wait") until progression, which may lead to therapeutic intervention only after the detection of end-organ damage. "Early intervention in high-risk smoldering multiple myeloma demonstrated a reduction in the risk of progression or death," said Sen Zhuang, M.D., Vice President, Oncology Clinical Research, Johnson & Johnson Innovative Medicine. "The proactive approach demonstrated in the AQUILA study is an example of Johnson & Johnson's aspiration to get in front of cancer by providing a platform to treat disease before progression to active disease." The committee reviewed data from the AQUILA study, a Phase 3, randomized, open-label trial which evaluated the efficacy and safety of DARZALEX FASPRO® versus SOC active monitoring in patients with HR-SMM.1 Results were initially presented at the 2024 American Society of Hematology (ASH) Annual Meeting and simultaneously published in The New England Journal of Medicine.2 "High-risk smoldering multiple myeloma remains a challenging clinical conundrum with no approved therapies, and earlier intervention may delay or even prevent progression to active multiple myeloma," said Peter Voorhees, M.D., Atrium Health / Levine Cancer Institute, Charlotte, N.C.‡ "We appreciate the balance the committee provided when assessing the risks and benefits of finite treatment at this stage and its recognition of the promise of DARZALEX FASPRO." The recommendation reinforces Johnson & Johnson's vision for the future of oncology – one where early diagnosis and treatments become standard, and where science moves us closer to a world without cancer. With bold choices over time, J&J is dedicated to our mission of evolving the treatment paradigm of patients with multiple myeloma. The ODAC is convened upon request of the FDA to review and evaluate safety and efficacy data of human drug products for use in the treatment of oncologic diseases. The committee provides non-binding recommendations based on its evaluation; however, final decisions on approval of the drug are made by the FDA. About the AQUILA StudyAQUILA (NCT03301220) is a randomized, multicenter Phase 3 study comparing treatment with DARZALEX FASPRO® to active monitoring in patients with smoldering multiple myeloma (SMM). The primary endpoint is progression-free survival (PFS), defined as progression to active multiple myeloma (MM) as assessed by an independent review committee, according to IMWG diagnostic criteria for MM (SLiM-CRAB), or death. Major secondary endpoints included overall response rate, PFS on first-line MM treatment (PFS2), and overall survival. About Multiple Myeloma Multiple myeloma is a blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.3 In multiple myeloma, these malignant plasma cells proliferate and replace normal cells in the bone marrow.4 Multiple myeloma is the second most common blood cancer worldwide and remains an incurable disease.5 In 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma in the U.S. and more than 12,000 will die from the disease.6 People with multiple myeloma have a 5-year survival rate of 59.8 percent. While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.7,8 About Smoldering Multiple MyelomaSmoldering multiple myeloma (SMM) is an asymptomatic intermediate disease state of multiple myeloma characterized by abnormal monoclonal bone marrow plasma cell (BMPC) proliferation and abnormally high levels of circulating M proteins with absence of myeloma-defining events. SMM is associated with a 10 percent annual risk of progressing to multiple myeloma (MM) or a related disorder, but half of patients with high-risk SMM progress to MM and are at risk of developing severe symptoms and organ damage within just two years of diagnosis. About DARZALEX FASPRO® and DARZALEX® DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) received U.S. FDA approval in May 2020 and is approved for nine indications in MM, four of which are for frontline treatment in newly diagnosed patients who are transplant eligible or ineligible.3,6 It is the only subcutaneous CD38-directed antibody approved to treat patients with MM. DARZALEX FASPRO® is co-formulated with recombinant human hyaluronidase PH20, Halozyme's ENHANZE® drug delivery technology. DARZALEX® (daratumumab) received U.S. FDA approval in November 2015 and is approved in eight indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant eligible and ineligible.9 DARZALEX® is the first CD38-directed antibody approved to treat MM.9 DARZALEX®-based regimens have been used in the treatment of more than 618,000 patients worldwide. In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab. For more information, visit DARZALEX FASPRO® INDICATIONS AND IMPORTANT SAFETY INFORMATION INDICATIONS DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) is indicated for the treatment of adult patients with MM: In combination with bortezomib, lenalidomide, and dexamethasone for induction and consolidation in newly diagnosed patients who are eligible for autologous stem cell transplant In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory MM who have received at least one prior therapy In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor (PI) In combination with carfilzomib and dexamethasone in patients with relapsed or refractory MM who have received one to three prior lines of therapy In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy As monotherapy in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double refractory to a PI and an immunomodulatory agent IMPORTANT SAFETY INFORMATION CONTRAINDICATIONSDARZALEX FASPRO® is contraindicated in patients with a history of severe hypersensitivity to daratumumab, hyaluronidase or any of the components of the formulation. WARNINGS AND PRECAUTIONS Hypersensitivity and Other Administration ReactionsBoth systemic administration-related reactions, including severe or life-threatening reactions, and local injection-site reactions can occur with DARZALEX FASPRO®. Fatal reactions have been reported with daratumumab-containing products, including DARZALEX FASPRO®. Systemic ReactionsIn a pooled safety population of 1249 patients with MM (N=1056) or light chain (AL) amyloidosis (N=193) who received DARZALEX FASPRO® as monotherapy or in combination, 7 percent of patients experienced a systemic administration-related reaction (Grade 2: 3.2 percent, Grade 3: 0.7 percent, Grade 4: 0.1 percent). Systemic administration-related reactions occurred in 7 of patients with the first injection, 0.2 percent with the second injection, and cumulatively 1 percent with subsequent injections. The median time to onset was 2.9 hours (range: 5 minutes to 3.5 days). Of the 165 systemic administration-related reactions that occurred in 93 patients, 144 (87 percent) occurred on the day of DARZALEX FASPRO® administration. Delayed systemic administration-related reactions have occurred in 1 percent of the patients. Severe reactions included hypoxia, dyspnea, hypertension, tachycardia, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Other signs and symptoms of systemic administration-related reactions may include respiratory symptoms, such as bronchospasm, nasal congestion, cough, throat irritation, allergic rhinitis, and wheezing, as well as anaphylactic reaction, pyrexia, chest pain, pruritus, chills, vomiting, nausea, hypotension, and blurred vision. Pre-medicate patients with histamine-1 receptor antagonist, acetaminophen, and corticosteroids. Monitor patients for systemic administration-related reactions, especially following the first and second injections. For anaphylactic reaction or life-threatening (Grade 4) administration-related reactions, immediately and permanently discontinue DARZALEX FASPRO®. Consider administering corticosteroids and other medications after the administration of DARZALEX FASPRO® depending on dosing regimen and medical history to minimize the risk of delayed (defined as occurring the day after administration) systemic administration-related reactions. Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with daratumumab-containing products. If ocular symptoms occur, interrupt DARZALEX FASPRO® and seek immediate ophthalmologic evaluation prior to restarting DARZALEX FASPRO®. Local ReactionsIn this pooled safety population, injection-site reactions occurred in 7 percent of patients, including Grade 2 reactions in 0.8 percent. The most frequent (>1 percent) injection-site reaction was injection-site erythema. These local reactions occurred a median of 5 minutes (range: 0 minutes to 6.5 days) after starting administration of DARZALEX FASPRO®. Monitor for local reactions and consider symptomatic management. NeutropeniaDaratumumab may increase neutropenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX FASPRO® until recovery of neutrophils. In lower body weight patients receiving DARZALEX FASPRO®, higher rates of Grade 3-4 neutropenia were observed. ThrombocytopeniaDaratumumab may increase thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Consider withholding DARZALEX FASPRO® until recovery of platelets. Embryo-Fetal ToxicityBased on the mechanism of action, DARZALEX FASPRO® can cause fetal harm when administered to a pregnant woman. DARZALEX FASPRO® may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX FASPRO® and for 3 months after the last dose. The combination of DARZALEX FASPRO® with lenalidomide, thalidomide, or pomalidomide is contraindicated in pregnant women because lenalidomide, thalidomide, and pomalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, thalidomide, or pomalidomide prescribing information on use during pregnancy. Interference With Serological TestingDaratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab administration. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient's serum. The determination of a patient's ABO and Rh blood type are not impacted. Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX FASPRO®. Type and screen patients prior to starting DARZALEX FASPRO®. Interference With Determination of Complete ResponseDaratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some DARZALEX FASPRO®-treated patients with IgG kappa myeloma protein. ADVERSE REACTIONS In MM, the most common adverse reaction (≥20 percent) with DARZALEX FASPRO® monotherapy is upper respiratory tract infection. The most common adverse reactions with combination therapy (≥20 percent for any combination) include fatigue, nausea, diarrhea, dyspnea, insomnia, headache, pyrexia, cough, muscle spasms, back pain, vomiting, hypertension, upper respiratory tract infection, peripheral sensory neuropathy, constipation, pneumonia, and peripheral edema. The most common hematology laboratory abnormalities (≥40 percent) with DARZALEX FASPRO® are decreased leukocytes, decreased lymphocytes, decreased neutrophils, decreased platelets, and decreased hemoglobin. Please click here to see the full Prescribing Information for DARZALEX FASPRO®. DARZALEX® INDICATIONS AND IMPORTANT SAFETY INFORMATION INDICATIONS DARZALEX® (daratumumab) is indicated for the treatment of adult patients with MM: In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory MM who have received at least one prior therapy In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor In combination with carfilzomib and dexamethasone in patients with relapsed or refractory MM who have received one to three prior lines of therapy In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy As monotherapy in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent CONTRAINDICATIONS DARZALEX® is contraindicated in patients with a history of severe hypersensitivity (eg, anaphylactic reactions) to daratumumab or any of the components of the formulation. WARNINGS AND PRECAUTIONS Infusion-Related Reactions DARZALEX® can cause severe and/or serious infusion-related reactions including anaphylactic reactions. These reactions can be life-threatening, and fatal outcomes have been reported. In clinical trials (monotherapy and combination: N=2066), infusion-related reactions occurred in 37 percent of patients with the Week 1 (16 mg/kg) infusion, 2 percent with the Week 2 infusion, and cumulatively 6 percent with subsequent infusions. Less than 1 percent of patients had a Grade 3/4 infusion-related reaction at Week 2 or subsequent infusions. The median time to onset was 1.5 hours (range: 0 to 73 hours). Nearly all reactions occurred during infusion or within 4 hours of completing DARZALEX®. Severe reactions have occurred, including bronchospasm, hypoxia, dyspnea, hypertension, tachycardia, headache, laryngeal edema, pulmonary edema, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Signs and symptoms may include respiratory symptoms, such as nasal congestion, cough, throat irritation, as well as chills, vomiting, and nausea. Less common signs and symptoms were wheezing, allergic rhinitis, pyrexia, chest discomfort, pruritus, hypotension and blurred vision. When DARZALEX® dosing was interrupted in the setting of ASCT (CASSIOPEIA) for a median of 3.75 months (range: 2.4 to 6.9 months), upon re-initiation of DARZALEX®, the incidence of infusion-related reactions was 11 percent for the first infusion following ASCT. Infusion-related reactions occurring at re-initiation of DARZALEX® following ASCT were consistent in terms of symptoms and severity (Grade 3 or 4: <1 percent) with those reported in previous studies at Week 2 or subsequent infusions. In EQUULEUS, patients receiving combination treatment (n=97) were administered the first 16 mg/kg dose at Week 1 split over two days, ie, 8 mg/kg on Day 1 and Day 2, respectively. The incidence of any grade infusion-related reactions was 42 percent, with 36 percent of patients experiencing infusion-related reactions on Day 1 of Week 1, 4 percent on Day 2 of Week 1, and 8 percent with subsequent infusions. Pre-medicate patients with antihistamines, antipyretics, and corticosteroids. Frequently monitor patients during the entire infusion. Interrupt DARZALEX® infusion for reactions of any severity and institute medical management as needed. Permanently discontinue DARZALEX® therapy if an anaphylactic reaction or life-threatening (Grade 4) reaction occurs and institute appropriate emergency care. For patients with Grade 1, 2, or 3 reactions, reduce the infusion rate when re-starting the infusion. To reduce the risk of delayed infusion-related reactions, administer oral corticosteroids to all patients following DARZALEX® infusions. Patients with a history of chronic obstructive pulmonary disease may require additional post-infusion medications to manage respiratory complications. Consider prescribing short- and long-acting bronchodilators and inhaled corticosteroids for patients with chronic obstructive pulmonary disease. Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with DARZALEX® infusion. If ocular symptoms occur, interrupt DARZALEX® infusion and seek immediate ophthalmologic evaluation prior to restarting DARZALEX®. Interference With Serological Testing Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab infusion. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient's serum. The determination of a patient's ABO and Rh blood type is not impacted. Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX®. Type and screen patients prior to starting DARZALEX®. Neutropenia and Thrombocytopenia DARZALEX® may increase neutropenia and thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer's prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX® until recovery of neutrophils or for recovery of platelets. Interference With Determination of Complete Response Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some patients with IgG kappa myeloma protein. Embryo-Fetal Toxicity Based on the mechanism of action, DARZALEX® can cause fetal harm when administered to a pregnant woman. DARZALEX® may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX® and for 3 months after the last dose. The combination of DARZALEX® with lenalidomide, pomalidomide, or thalidomide is contraindicated in pregnant women because lenalidomide, pomalidomide, and thalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, pomalidomide, or thalidomide prescribing information on use during pregnancy. ADVERSE REACTIONS The most frequently reported adverse reactions (incidence ≥20 percent) were: upper respiratory infection, neutropenia, infusion related reactions, thrombocytopenia, diarrhea, constipation, anemia, peripheral sensory neuropathy, fatigue, peripheral edema, nausea, cough, pyrexia, dyspnea, and asthenia. The most common hematologic laboratory abnormalities (≥40 percent) with DARZALEX® are: neutropenia, lymphopenia, thrombocytopenia, leukopenia, and anemia. Please click here to see the full Prescribing Information. About Johnson & Johnson At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at or at Follow us at @JNJInnovMed. Janssen Research & Development, LLC, Janssen Biotech, Inc. and Janssen Global Services, LLC are Johnson & Johnson companies. Cautions Concerning Forward-Looking StatementsThis press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of DARZALEX FASPRO®. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen-Cilag International NV, Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC, Janssen-Cilag, S.A., Janssen Scientific Affairs, LLC and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at or on request from Johnson & Johnson. None of Janssen-Cilag International NV, Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC, Janssen-Cilag, S.A., Janssen Scientific Affairs, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments. ‡ Peter Voorhees, M.D., Levine Cancer Institute, Charlotte, N.C., has provided consulting, advisory, and speaking services to Johnson & Johnson; he has not been paid for any media work. Media contact: Oncology Media Relations Oncology_media_relations@ Investor contact:Lauren Johnson investor-relations@ U.S. medical inquiries:+1 800 526-7736 1 Dimopoulos, M.-A. Phase 3 randomized study of daratumumab monotherapy versus active monitoring in patients with high-risk smoldering multiple myeloma: primary results of the AQUILA study. Abstract #773 [Oral Presentation]. Presented at the 2024 American Society of Hematology Annual Meeting.2 Dimopoulos, M.-A., et al. Daratumumab or Active Monitoring for High-Risk Smoldering Multiple Myeloma. New England Journal of Medicine. Accessed March 25, 2025.3 Rajkumar SV. Multiple Myeloma: 2020 Update on Diagnosis, Risk-Stratification and Management. Am J Hematol. 2020;95(5):548-5672020;95(5):548-567. 4 National Cancer Institute. Plasma Cell Neoplasms. Accessed August 2024. Available at: 5 Multiple Myeloma. City of Hope, 2022. Multiple Myeloma: Causes, Symptoms & Treatments. Accessed August 2024. Available at: 6 American Cancer Society. Myeloma Cancer Statistics. Accessed August 2024. Available at: 7 American Cancer Society. What is Multiple Myeloma? Accessed August 2024. Available at: 8 American Cancer Society. Multiple Myeloma Early Detection, Diagnosis, and Staging. Accessed August 2024. Available at: View original content to download multimedia: SOURCE Johnson & Johnson Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
