Aponermin-Based Bridging Therapy Prior to CAR-T Infusion in Relapsed/Refractory Multiple Myeloma Patients With Extramedullary Disease: A Prospective, Single-Arm, Multicenter, Open-Label Study

This is a prospective, single-arm, multicenter, open-label study to evaluate the efficacy and safety of aponermin-based bridging therapy prior to CAR-T infusion in relapsed/refractory multiple myeloma patients with extramedullary disease.

Start Date01 Mar 2025

Sponsor / Collaborator-

NCT06772480

/ Not yet recruitingNot ApplicableIIT

Thalidomide Versus Argon Plasma Coagulation in Gastric Antral Vascular Ectasia(GAVE)-Related Anaemia in Cirrhosis (TAG Trial)- A Randomised Controlled Trial

Gastric Antral Vascular Ectasia (GAVE) is an erosive form of gastritis distinguished by veno-capillary ectasia, which manifests as tortuous columns of dilated vessels. Histologically, these vessels show dilated mucosal capillaries filled with fibrin thrombi, accompanied by fibromuscular hyperplasia and spindle cell proliferation of the lamina propria. GAVE is prevalent in about 12% of patients with cirrhosis, with 60-70% of these patients becoming transfusion-dependent due to severe anaemia caused by GAVE related bleeding. The most commonly used treatment for GAVE is endoscopic therapy using Argon Plasma Coagulation (APC), which, while effective, often requires multiple sessions due to a high recurrence rate of 30-60%. These frequent interventions increase the burden on patients and healthcare systems. As a result, alternative treatments have been sought. Thalidomide, known for its potent antiangiogenic properties, significantly lowers vascular endothelial growth factor (VEGF) levels, offering a promising non-invasive treatment option. Early studies, such as those by García-Pagán have demonstrated thalidomide's effectiveness in reducing transfusion requirements and managing bleeding in cirrhotic patients with GAVE, yet its comprehensive efficacy and safety profile remains under-studied. This project aims to rigorously evaluate the efficacy and safety of thalidomide compared to APC in managing GAVE-related anemia in cirrhotic patients. Through a controlled trial, this study will provide vital data to potentially shift treatment paradigms, enhance patient quality of life, and reduce the need for repetitive invasive procedures.

Start Date05 Jan 2025

Sponsor / Collaborator

Institute of Liver & Biliary Sciences

100 Clinical Results associated with Thalidomide

100 Translational Medicine associated with Thalidomide

100 Patents (Medical) associated with Thalidomide

12,508

Literatures (Medical) associated with Thalidomide

31 Dec 2025·Hematology

Bortezomib, thalidomide, and dexamethasone versus bortezomib, lenalidomide, and dexamethasone in transplant-eligible newly diagnosed multiple myeloma: a systemic review and meta-analysis

Review

Author: Lin, Cheng-Hsien ; Teng, Chieh-Lin Jerry ; Su, Yu-Chen ; Wang, Yin-Che

BACKGROUNDThe current study aimed to compare treatment responses, the incidence of the need for auto-HSCT, and the occurrence of specific adverse events (AEs) between VTD and velcade, VRD induction regimens in patients with transplant-eligible newly diagnosed multiple myeloma (NDMM).METHODSThis systematic review and meta-analysis included 15 studies: six evaluating the VTD regimen and nine evaluating the VRD one. The primary endpoints were response rates after induction therapy and the incidence of a need for autologous hematopoietic stem cell transplantion (auto-HSCT) between the groups. We also examined the occurrence of grade 3 or 4 hematological, infection, and thrombotic AEs in both groups.RESULTSThe VTD group showed an overall response rate (ORR) of 93%, while the VRD group had an ORR of 86%. The very good partial response (VGPR) rates were 61% in the VTD group and 60% in the VRD one. The auto-HSCT rate was higher in the VTD group, averaging 93% compared to 70% in the VRD one. The incidence of grade 3 or 4 hematological AEs was 31% for VTD and 33% for VRD. The rates of grade 3 or 4 infection-related AEs were 9% in the VTD group and 14% in the VRD one. The incidence of grade 3 or 4 thrombotic AEs was 4% for VTD and 3% for VRD.CONCLUSIONSWith comparable safety profiles, VTD and VRD induction therapies are similarly effective for transplant-eligible NDMM, showing similar ORRs and VGPR rates.

31 Dec 2025·Renal Failure

Outcomes of BTD vs. BCD as initial treatment of renal amyloid light-chain amyloidosis: a retrospective cohort study in China

Article

Author: Li, Liwen ; He, Weiting ; Zhong, Liye ; Chen, Xiaojie ; Yau, Hok-him ; Zhang, Shaogui ; Liu, Hui ; Liao, Pengjun ; Zhang, Yifan ; Li, Sheng ; Xie, Jianteng ; Wang, Wenjian ; Zhu, Yaxi

OBJECTIVESTo compare the efficacy and safety of bortezomib with thalidomide and dexamethasone (BTD) and bortezomib with cyclophosphamide and dexamethasone (BCD) as the initial treatment for renal amyloid light chain (AL) amyloidosis in Chinese cohort.METHODSA cohort of 174 patients with AL amyloidosis was studied in Guangdong Provincial People's Hospital from January 2008 to August 2023. Propensity-score matching cases were applied to assess the outcomes of patients treated with BTD and BCD regimen. Primary outcomes were patients achieving hematologic response and organ responses, and the secondary endpoints were patients progressing to end-stage renal disease or all-cause death.RESULTS44 Patients were included. The hematologic complete response rate (CR) in the BTD group was comparable between the groups of BTD group and BCD. However, the time to achieve hematologic CR was significantly shorter in the BTD group compared to the BCD group (4.97 vs. 7.71 mon, p = 0.010). Furthermore, when reaching hematologic response, the cumulative dose of bortezomib that standardized by body surface area (BSA) was lower in BTD group than in the BCD group (10.4 vs. 15.6 mg/m², p = 0.013). There was no significant difference of renal and cardiac response between groups. However, post-treatment proteinuria levels after treatment were significantly lower in the BTD group compared to those in the BCD group (747 mg/24h vs. 2928 mg/24h, p = 0.048).CONCLUSIONSCompared to BCD regimen for renal AL amyloidosis, initial treatment with BTD regimen demonstrated similar rates of hematologic CR but showed superior reduction in proteinuria, reduced cumulative dose of bortezomib and faster time-to-response.

01 Dec 2025·Blood Research

Efficacy and safety of thalidomide with hydroxyurea in sickle cell anemia: a quasi-experimental clinical trial

Article

Author: Bahirat, Harshwardhan ; Paul, Anindita ; Epari, Venkatarao ; Samal, Priyanka ; Bishoyi, Ajit Kumar

AbstractBackgroundThe clinical course of sickle cell anemia (SCA) is variable, with chronic hemolysis and end-organ damage caused by microvascular occlusion. We evaluated the efficacy and safety of thalidomide plus hydroxyurea (HU) compared with HU alone to determine whether the combination provides a superior clinical benefit and safety profile.MethodsThis was an open-label quasi-experimental clinical trial (Clinical Trials Registry of India, CTRI Registration Number 2023/04/065682). Patients with SCA aged > 12 years and postmenopausal females aged > 45 years were allocated 1:1 to receive either HU (20 mg/kg/day) and thalidomide (50 mg/day) in Group A or HU (20 mg/kg/day) only in Group B.ResultsThe frequency of vaso-occlusive crises (VOCs), transfusion requirements, variations in hematological parameters (hemoglobin [Hb], fetal hemoglobin [HbF], and sickle hemoglobin [HbS]), and side effects between the groups were assessed over 12 months. Repeated-measures analysis of variance was used to determine changes across the observation period. The mean age of the 66 patients diagnosed with SCA (homozygous HbS mutation) was 32.9 (standard deviation ± 11.5) years, and 57.6% were males. Over the 12-month observation period, Group A had significantly fewer VOCs (3.48 ± 2.81) and packed red blood cell transfusions (3.61 ± 2.19) than Group B (11.36 ± 4.20 VOCs; 13.27 ± 3.70 transfusions) (p = 0.0001). There was a significant increase in Hb (8.2 ± 1.8 to 11.8 ± 1.2 g/dL), a decrease in HbS% (72.5 ± 5.5 to 64.5 ± 5.4), and a rise in HbF% (18.9 ± 5.1 to 28.4 ± 5.6) (p < 0.0001) in Group A.ConclusionCombining thalidomide with HU significantly reduced VOCs and transfusion requirements, improved Hb and HbF%, and decreased HbS levels.

103

News (Medical) associated with Thalidomide

21 Apr 2025

·www.biospace.com

Banning Industry From FDA Adcomms Won’t ‘Move the Needle Much’ in Makary, RFK’s Fight Against COIs

iStock/ werayuth Newly confirmed FDA commissioner Marty Makary last week announced a directive that would limit industry participation in the agency’s advisory committees. But not only do company reps serve only as non-voting members, a 1997 law actually requires industry involvement. Newly confirmed FDA commissioner Marty Makary wants to “restore impeccable integrity” to the drug review process, and he’s starting by banning pharmaceutical representatives from serving as members on some of the agency’s advisory committees. Reaction to this move, however, has been relatively muted. “I don’t think [the new directive] will move the needle much,” Genevieve Kanter, an associate professor of public policy at the University of Southern California Price School of Public Policy, told BioSpace in an email. Kanter specifically noted that industry representatives are already non-voting members. “The central and perhaps more difficult conflict of interest issues relate to the industry ties of the scientific members of the committee, and that issue hasn’t been directly addressed yet,” Kanter said. Steven Grossman, policy and regulatory consultant and author of the FDA Matters blog, concurred. “My advice to stakeholders: put your energies into proposals that make more of a difference than this one,” he told BioSpace in an email. It’s unclear at this point how comprehensive the new policy will be. The new directive “limits individuals employed at companies regulated by the [FDA], such as pharmaceutical companies, from serving as official members on FDA advisory committees, where statutorily allowed,” according to the agency’s Friday announcement . This reference to “statutorily allowed” could present a bit of a quagmire for the FDA. A provision in the Food and Drug Administration Modernization Act of 1997 “has been interpreted to mean that newly created advisory committees must include an industry representative,” Kanter said. “My guess is that the FDA may remove industry reps from advisory committees that existed prior to 1997.” As of April 2024, the FDA had 33 advisory committees . The advisory panel, the Obstetrics and Gynecology Advisory Committee, was established in 1965 , largely to track the safety of thalidomide. As of 1967, the Anti-Infective Agents Advisory Committee was also up and running. As of 1997 , the Vaccines and Related Biological Products Advisory Committee and Oncologic Drugs Advisory Committee were also active. But for advisory committees established after that 1997 legislation, it seems that industry representatives cannot, by law, be excluded. Another interpretation is that the language only applies to advisory committees “regarding a clinical investigation or the approval for market for a drug,” Kanter said. Panels that review, for example, post-market safety or that discuss other regulatory issues, would not be required to have an industry rep. Makary: ‘Surrounded by Swamp Creatures’ The question of potential industry influence on the FDA has been simmering for years, largely centered around the so-called “ revolving door ,” through which employees move from industry to FDA and vice versa. In an interview with former Fox News journalist Megyn Kelly last week, Makary referenced the case of Curtis Wright , the FDA examiner who signed off on Purdue’s infamous painkiller OxyContin and later left the agency to work for the now-defunct pharma. “That’s the kind of thing that breeds distrust and that’s why people perceive that this agency, the FDA, has been captured by industry,” Makary told Kelly. Newly appointed Health and Human Services Commissioner Robert F. Kennedy Jr. has taken this up, along with other potential “conflicts of interest,” he sees within the FDA. In a tour of the FDA campus earlier this month, Kennedy blasted the agency and its staff, accusing them of being a “sock puppet” to the pharma industry. After ousting Peter Marks , head of the FDA’s Center for Biologics Evaluation and Research, the FDA appointed interim director Scott Steele , who has never worked at a biopharma company. At another point during the interview, Makary said he was “surrounded by the swamp creatures” after his nomination for FDA commissioner. “When I got his nomination for the job, I cannot tell you how many lobbyists, former members of Congress, the swamp, reached out to me,” offering to write letters on behalf of their companies to the senators on the confirmation committee. “You know what I said? Don’t talk to the senators. I don’t want your letters. They’re not for free. Those are obligations that then you feel indebted to return once you’re in office.” The Advisory Committee Debate Addressing the new policy directive regarding adcomms, Makary said, “The idea is that there should not be a cozy relationship. . . . There should be a user-friendly process for industry, but not a cozy relationship.” For his part, Grossman offered points on both sides. “Industry representatives on advisory panels do not vote, do not have a professional or monetary interest in the outcome and have little ability to sway the outcome. Yet, they do provide a useful perspective that other panelists often value,” he said. “Nonetheless, the appearance is jarring and it is understandable that questions are being raised.” Kanter added that industry reps “could present a practical perspective on the feasibility and implications of committee recommendations, as well as important constraints facing the sector—for example, issues with the supply chain or manufacturing.” The sponsor, who would also be in the meeting, “isn’t generally able to raise these concerns without being directly asked by committee members.” As per Thursday’s announcement, Makary plans to replace the ousted industry representatives with input from patients, caregivers and other community representatives. “Patient advocates and caregivers know a great deal about the health conditions that are affecting their daily lives, and some of them may be physicians or researchers themselves,” Kanter said. She acknowledged, however, that “in general, they do not go through the same screening process for scientific expertise as the other members and may not know as much about the scientific issues.” Instead, these panel members play a different role in the conversation, “introducing information about the experience of living with the health condition or caring for someone with the condition, the needs, priorities, and sentiment of the patient community at large, and adding context and perhaps helping interpret the numbers and statistics,” Kanter continued. Overall, she said, “This move is an important sign that Commissioner Makary will take a second look at the issue of industry influence on FDA activities, and I am generally supportive of that.”

Executive Change

07 Apr 2025

·pipelinereview.com

European Commission approves Johnson & Johnson’s subcutaneous DARZALEX® (daratumumab)-based quadruplet regimen for the treatment of patients with newly diagnosed multiple myeloma, regardless of transplant eligibility

Approval cements daratumumab as a foundational therapy in newly diagnosed multiple myeloma and the only anti-CD38 antibody for all patient types in this setting Phase 3 CEPHEUS study shows significant improvement in minimal residual disease (MRD)-negativity rate, progression-free survival and complete response or better versus standard of care 1 BEERSE, Belgium I April 07, 2025 I Janssen-Cilag International NV, a Johnson & Johnson company, today announced that the European Commission (EC) has approved an indication extension of DARZALEX ® (daratumumab) subcutaneous (SC) formulation in the frontline setting. The approval is for daratumumab SC in combination with bortezomib, lenalidomide, and dexamethasone (daratumumab-VRd) for the treatment of adult patients with newly diagnosed multiple myeloma (NDMM). 1 “Multiple myeloma is a complex and evolving disease. Starting with more effective regimens in the frontline setting offers patients the best chance of sustained long-term outcomes by preventing disease resistance and relapse,” said Professor Katja Weisel, University Medical Centre Hamburg-Eppendorf. “The subcutaneous daratumumab-VRd regimen delivers an effective and convenient new standard of care for patients with newly diagnosed multiple myeloma, regardless of transplant eligibility, with responses that are deep and durable, and translate into significantly reduced risk of disease progression or death.” Daratumumab is now approved in nine indications for multiple myeloma, five of which are in the frontline setting, including as part of treatment regimens for newly diagnosed patients who are eligible or ineligible for autologous stem-cell transplant (ASCT). 1 Today’s approval follows the indication extension approval for daratumumab-VRd in October 2024, for the treatment of newly diagnosed patients with multiple myeloma who are eligible for ASCT, based on the results from the Phase 3 PERSEUS study. The study evaluated this daratumumab SC-based quadruplet regimen for induction and consolidation therapy, followed by daratumumab SC and lenalidomide maintenance. 2,3 “Daratumumab has become a cornerstone of multiple myeloma treatment over the past decade and is now the only anti-CD38 antibody approved to treat all patient types in the frontline setting, regardless of transplant eligibility,” said Edmond Chan, MBChB, M.D. (Res), EMEA Therapeutic Area Lead Haematology, Johnson & Johnson Innovative Medicine. “This latest approval confirms the enhanced benefit of daratumumab SC-based quadruplet regimens and its versatility and effectiveness in addressing the diverse needs of those affected by this complex disease.” The Phase 3 CEPHEUS (NCT03652064) study evaluated the efficacy and safety of daratumumab-VRd (n=197) compared to VRd (n=198) for patients with NDMM who are transplant ineligible or for whom ASCT was not planned as initial therapy (transplant ineligible or deferred). 1 Data from the study were previously presented at the 2024 International Myeloma Society (IMS) Annual Meeting. 4 At a median follow-up of 59 months, the primary endpoint was met, with an overall minimal residual disease (MRD)-negativity rate at a sensitivity of 10 -5 (no cancer cells detected within 100,000 bone marrow cells) of 60.9 percent for patients receiving daratumumab-VRd and 39.4 percent for VRd (Odds ratio [OR], 2.37; 95 percent confidence interval [CI], 1.58-3.55; p <0.0001). 1 Similarly, the proportion of patients achieving sustained MRD-negativity of ≥ 12 months almost doubled with daratumumab-VRd vs VRd (48.7 percent vs 26.3 percent; OR, 2.63; 95 percent CI, 1.73-4.00; p <0.0001). 1 The daratumumab SC-based quadruplet regimen, compared to VRd, also significantly increased the depth of response with higher rates of complete response (CR) or better. 1 The CR or better rate was 81.2 percent with daratumumab-VRd vs 61.6 percent with VRd (OR 2.73; 95 percent CI, 1.71-4.34; p <0.0001). 1 The study also demonstrated that daratumumab-VRd significantly reduced the risk of progression or death by 43 percent (Hazard ratio [HR], 0.57; 95 percent CI, 0.41-0.79; p <0.0005) vs VRd. The median progression-free survival was not reached for daratumumab-VRd vs 52.6 months for VRd. 1 Overall survival data were not yet mature. The overall safety profile of daratumumab-VRd was consistent with the known safety profiles for daratumumab SC and VRd. 4 The most common (>10 percent) Grade 3/4 haematologic and non-haematologic adverse events with daratumumab-VRd vs VRd were neutropenia (44.2 percent vs 29.7 percent), thrombocytopenia (28.4 percent vs 20.0 percent), anaemia (13.2 percent vs 11.8 percent), peripheral neuropathies (8.1 percent vs 8.2 percent), diarrhoea (12.2 percent vs 9.2 percent), and COVID-19 (11.2 percent vs 4.6 percent). 4 “At Johnson & Johnson, our dedication to advance multiple myeloma research spans more than 20 years and our commitment to transforming outcomes for patients has never been stronger than it is today,” said Jordan Schecter, M.D., Vice President, Disease Area Leader, Multiple Myeloma, Johnson & Johnson Innovative Medicine. “This landmark approval enables us to offer all patient populations access regardless of age, fitness or risk to a daratumumab-based triplet or quadruplet regimen in the frontline setting – a critical step towards our ultimate goal of delivering a functional cure.” Johnson & Johnson also submitted a supplemental Biologics License Application to the U.S. Food and Drug Administration seeking approval of a new indication for daratumumab SC in combination with VRd for the treatment of adult patients with NDMM for whom ASCT is deferred or who are ineligible for ASCT, on 30 September 2024. About the CEPHEUS Study CEPHEUS ( NCT03652064 ) is an international, randomised, open-label, Phase 3 study comparing subcutaneous daratumumab, bortezomib, lenalidomide, and dexamethasone (daratumumab-VRd) with standard bortezomib, lenalidomide, and dexamethasone (VRd). 4,5 The trial enrolled 395 patients with newly diagnosed multiple myeloma who were either ineligible for stem cell transplantation (SCT) or for whom SCT is not planned. 4 The primary endpoint was overall minimal residual disease (MRD)-negativity rate. 4 The minimum age for participation was 18 years for patients in both the daratumumab-VRd arm and VRd arm, with a median patient age of 70 (range 31-80). 4 The study was conducted in 13 countries across North America, South America, and Europe. 4 About daratumumab and daratumumab SC Johnson & Johnson is committed to exploring the potential of daratumumab for patients with multiple myeloma across the spectrum of the disease. In August 2012 , Janssen Biotech, Inc., a Johnson & Johnson company, and Genmab A/S entered a worldwide agreement, which granted Johnson & Johnson an exclusive licence to develop, manufacture and commercialise daratumumab. Since launch, daratumumab has become a foundational therapy in the treatment of multiple myeloma, having been used in the treatment of more than 618,000 patients worldwide. 6 Daratumumab is the only CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma. 7 Daratumumab SC is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE ® drug delivery technology. 7 CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease. 7 Daratumumab binds to CD38 and inhibits tumour cell growth causing myeloma cell death. 7 Daratumumab may also have an effect on normal cells. 7 Data across ten Phase 3 clinical trials, in both the frontline and relapsed settings, have shown that daratumumab-based regimens resulted in significant improvement in progression-free survival and/or overall survival. 4,8,9,10,11,12,13,14,15 For further information on daratumumab, please see the Summary of Product Characteristics at: https://ec.europa.eu/health/documents/community-register/html/h1101.htm . About Multiple Myeloma Multiple myeloma is currently an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow. 16,17 In multiple myeloma, these malignant plasma cells continue to proliferate, accumulating in the body and crowding out normal blood cells, as well as often causing bone destruction and other serious complications. 17 In the European Union, it is estimated that more than 35,000 people were diagnosed with multiple myeloma in 2022, and more than 22,700 patients died. 18 People living with multiple myeloma experience relapses which become more frequent with each line of therapy 19,20 while remissions become progressively shorter. 19,20,21 Whilst some patients with multiple myeloma initially have no symptoms, others can have common signs and symptoms of the disease, which can include bone fracture or pain, low red blood cell counts, fatigue, high calcium levels, infections, or kidney damage. 22 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 www.innovativemedicine.jnj.com/emea . Follow us at www.linkedin.com/company/jnj-innovative-medicine-emea . Janssen-Cilag International NV, Janssen Pharmaceutica NV, Janssen-Cilag Limited, Janssen Biotech, Inc., and Janssen Research & Development, LLC are Johnson & Johnson companies. 1 European Medicines Agency. DARZALEX (daratumumab) Summary of Product Characteristics. April 2025. 2 Rodríguez-Otero P, et al. Daratumumab (DARA) + bortezomib/lenalidomide/dexamethasone (VRd) in transplant-eligible (TE) patients (pts) with newly diagnosed multiple myeloma (NDMM): Analysis of minimal residual disease (MRD) in the PERSEUS trial. 2024 American Society for Clinical Oncology Annual Meeting. June 3, 2024. 3 Johnson & Johnson Innovative Medicine EMEA. DARZALEX® (daratumumab)-SC based quadruplet regimen approved by the European Commission for patients with newly diagnosed multiple myeloma who are transplant-eligible. Available at: https://www.jnj.com/media-center/press-releases/darzalex-daratumumab-sc-based-quadruplet-regimen-approved-by-the-european-commission-for-patients-with-newly-diagnosed-multiple-myeloma-who-are-transplant-eligible . Last accessed: April 2025. 4 Usmani S Z, et al. Daratumumab + Bortezomib/Lenalidomide/Dexamethasone in Patients With Transplant-ineligible or Transplant-deferred Newly Diagnosed Multiple Myeloma: Results of the Phase 3 CEPHEUS Study. Oral presentation. 21st International Myeloma Society (IMS) Annual Meeting. September 25 – 28, 2024. 5 Clinicaltrials.gov. A Study Comparing Daratumumab, VELCADE (Bortezomib), Lenalidomide, and Dexamethasone (D-VRd) With VELCADE, Lenalidomide, and Dexamethasone (VRd) in Participants With Untreated Multiple Myeloma and for Whom Hematopoietic Stem Cell Transplant is Not Planned as Initial Therapy. NCT03652064. Available at: https://clinicaltrials.gov/study/NCT03652064?term=NCT03652064&cond=Multiple%20Myeloma&rank=1&a=63 . Last accessed: April 2025. 6 Johnson & Johnson [data on file]. RF-452129. Number of patients treated with DARZALEX worldwide as of December 2024. 7 Janssen EMEA. European Commission Grants Marketing Authorisation for DARZALEX ® (Daratumumab) Subcutaneous Formulation for All Currently Approved Daratumumab Intravenous Formulation Indications. Available at: http://www.businesswire.com/news/home/20200604005487/en/European-Commission-GrantsMarketingAuthorisation-for-DARZALEX%C2%AE%E2%96%BC-daratumumab-SubcutaneousFormulation-for-all-CurrentlyApproved-Daratumumab-Intravenous-Formulation-Indications . Last accessed: April 2025. 8 Moreau P, et al. Bortezomib, thalidomide, and dexamethasone with or without daratumumab before and after autologous stem-cell transplantation for newly diagnosed multiple myeloma (CASSIOPEIA): a randomised, openlabel, phase 3 study. Lancet 2019;394(10192):29-38. 9 Facon T, et al. MAIA Trial Investigators. Daratumumab plus Lenalidomide and Dexamethasone for Untreated Myeloma. N Engl J Med 2019;380(22):2104-2115. 10 Mateos MV, et al. Overall survival with daratumumab, bortezomib, melphalan, and prednisone in newly diagnosed multiple myeloma (ALCYONE): a randomised, open-label, phase 3 trial. The Lancet 2020;395:P132-141. 11 Dimopoulos MA, et al. APOLLO Trial Investigators. Daratumumab plus pomalidomide and dexamethasone versus pomalidomide and dexamethasone alone in previously treated multiple myeloma (APOLLO): an open-label, randomised, phase 3 trial. Lancet Oncol 2021;22(6):801-812. 12 Palladini G, et al. Daratumumab plus CyBorD for patients with newly diagnosed AL amyloidosis: safety run-in results of ANDROMEDA. Blood 2020;2;136(1):71-80. 13 Chari A, et al. Daratumumab plus pomalidomide and dexamethasone in relapsed and/or refractory multiple myeloma. Blood 2017;130(8):974-981. 14 Bahlis NJ, et al. Daratumumab plus lenalidomide and dexamethasone in relapsed/refractory multiple myeloma: extended follow-up of POLLUX, a randomized, open-label, phase 3 study. Leukemia 2020;34(7):1875-1884. 15 Mateos MV, et al. Daratumumab, Bortezomib, and Dexamethasone Versus Bortezomib and Dexamethasone in Patients With Previously Treated Multiple Myeloma: Three-year Follow-up of CASTOR. Clin Lymphoma Myeloma Leuk 2020;20(8):509-518. 16 Abdi J, et al. Drug resistance in multiple myeloma: latest findings on molecular mechanisms. Oncotarget 2013;4(12):2186-2207. 17 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.org/cancer/types/multiple-myeloma/if-you-have-multiple-myeloma . Last accessed: April 2025. 18 ECIS – European Cancer Information System. Estimates of cancer incidence and mortality in 2022, by country. Multiple myeloma. Available at: https://ecis.jrc.ec.europa.eu/explorer.php?$0-0$1-All$2-All$4-1,2$3-51$6-0,85$5-2022,2022$7-7$CEstByCountry$X0_8-3$X0_19-AE27$X0_20-No$CEstBySexByCountry$X1_8-3$X1_19-AE27$X1_-1-1$CEstByIndiByCountry$X2_8-3$X2_19-AE27$X2_20-No$CEstRelative$X3_8-3$X3_9-AE27$X3_19-AE27$CEstByCountryTable$X4_19-AE27 . Last accessed: April 2025. 19 Bhatt P, Kloock C, Comenzo R. Relapsed/Refractory Multiple Myeloma: A Review of Available Therapies and Clinical Scenarios Encountered in Myeloma Relapse. Curr Oncol. 2023;30(2):2322-2347. 20 Hernández-Rivas JÁ, et al. The changing landscape of relapsed and/or refractory multiple myeloma (MM): fundamentals and controversies. Biomark Res. 2022;10(1):1-23. 21 Gavriatopoulou M, et al. Metabolic Disorders in Multiple Myeloma. Int J Mol Sci. 2021;22(21):11430. 22 American Cancer Society. Multiple myeloma: early detection, diagnosis and staging. Available at: https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf . Last accessed: April 2025. SOURCE: Johnson & Johnson

Clinical ResultPhase 3Drug ApprovalASCOLicense out/in

29 Jan 2025

·www.fiercebiotech.com

Fewer than 1% of clinical trials in the US include pregnant patients, study finds

Researchers found only 19 trials for noninfectious chronic conditions, like depression and asthma, that included pregnant patients over the last 15 years.\n Pregnancy comes with unique health risks and considerations, but pregnant people also continue to experience the same health problems that nonpregnant people do. Despite this, a new study has found that fewer than 1% of randomized controlled trials in the U.S. include pregnant participants.The study was published in the American Journal of Obstetrics and Gynecology on Jan. 3 and was announced in a Jan. 21 press release.Out of 90,860 drug trials involving reproductive-age women from the last 15 years, only 0.8% included pregnant participants. What’s more, about 75% of the studies explicitly excluded pregnant people from participating.Excluding pregnant people from trials \"slows down the use of beneficial medications because doctors and pregnant people are hesitant to use drugs when there is limited safety data available,\" study author Alyssa Bilinski, Ph.D., a health policy researcher at Brown University, told Fierce Biotech in an email. \"Conversely, when a medication is harmful, more people likely experience adverse effects before the problem is discovered because not all individuals taking the medication are studied.\"Most of the trials that did include pregnant people were for pregnancy-specific conditions, like preventing preterm birth, preeclampsia and pregnancy-related symptoms like nausea.“We found only 19 trials for non-infectious chronic conditions like anxiety, depression and asthma that have included pregnant participants,” Bilinski said in the press release. Several tactics to increase pregnant patient participation in trials have been proposed by committees from the National Academies of Science, Engineering and Medicine and an FDA task force, Bilinski said. These include \"adjusting grant funding and timelines to account for challenges in working in this setting, improving communication to address liability concerns and changing classification of pregnant individuals as vulnerable in some review processes.\"In their paper, Bilinski and co-author Natalia Emanuel, Ph.D., an economist at the Federal Reserve Bank of New York, call for more randomized controlled trials that include pregnant patients. In the release, Bilinski pointed out that trials were only required to include women at all in 1993. “That really wasn’t that long ago,” Bilinski said. “Our hope is that 30 years from now, lacking high-quality evidence about medication safety during pregnancy will seem as strange and outdated as excluding women from trials seems to us today.”The two are now working on a paper about the health effects of excluding pregnant patients from trials, Bilinski said, using case studies like the COVID-19 vaccines and thalidomide, which was once used for treating morning sickness before it was discovered to cause serious birth defects.Editor\'s note: This story has been updated to include additional information from the study author.

Clinical StudyVaccine

100 Deals associated with Thalidomide

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KEGG	Wiki	ATC	Drug Bank
D00754	Thalidomide	L04AX02	DB01041

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
POEMS Syndrome	Japan	Fujimoto Pharmaceutical Corp.	24 Feb 2021
Multiple Myeloma	South Korea	Celgene Corp.	07 Apr 2006
Erythema Nodosum	United States	Bristol Myers Squibb Co.	16 Jul 1998
Leprosy, Lepromatous	China	Changzhou Pharmaceutical Factory Co., Ltd.	-

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Advanced Hepatocellular Carcinoma	Preclinical	Taiwan Province	TTY Biopharm Co. Ltd.	01 Feb 2003
Refractory Multiple Myeloma	Preclinical	France	Celgene Corp.	01 Dec 2001
Refractory Multiple Myeloma	Preclinical	Belgium	Celgene Corp.	01 Dec 2001
Multiple Myeloma	Discovery	Canada	Celgene Corp.	01 Feb 2003
Multiple Myeloma	Discovery	Switzerland	Celgene Corp.	01 Feb 2003
Multiple Myeloma	Discovery	Russia	Celgene Corp.	01 Feb 2003
Refractory Multiple Myeloma	Discovery	France	Celgene Corp.	01 Dec 2001
Refractory Multiple Myeloma	Discovery	Belgium	Celgene Corp.	01 Dec 2001
Anemia	Discovery	United States	Celgene Corp.	01 Aug 2001
Myelodysplastic Syndromes	Discovery	United States	Celgene Corp.	01 Aug 2001

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ChiCTR2200055486 (NEWS) Manual	Early Phase 1	Multiple Myeloma	19	塞利尼索+硼替佐米+沙利度胺+地塞米松	(soxfyzqcyq) = goymxxtrun bqbxyolymk (vymnzftpkg ) View more	Positive	11 Nov 2024
UEGW2024	Not Applicable	Hemorrhage	-	Thalidomide	fkzulwkzbr(ixurultmdy) = Mild side effects (meteorism, nausea, exanthemas) were observed in these patients bvltokbqxi (wxrcxnxhtd )	-	13 Oct 2024
ESMO2024	Not Applicable	Pancreatic Cancer \| Hyperhidrosis	45	Thalidomide 100mg	djscwurehl(zydfkvfkgy) = czneeeksby viffzsvvyd (fpvaejyqva ) View more	Positive	15 Sep 2024
NCT02507336 (CTgov)	Phase 2	Mantle-Cell Lymphoma	2	thalidomide	(uovfidlmcx) = vmvyxuukhg xthbeecbaw (elmrcghjcr, pknzyipqvh - syfhwpsooa) View more	-	01 Aug 2024
DDW2024	Not Applicable	Gastrointestinal Hemorrhage	-	Thalidomide	raazylazal(nrftvnercs) = Pooled rates of adverse events were 46.35% (95% CI 21.23-72.31, p=0.04, I2: 59%). Peripheral neuropathy was the most common adverse event. ybqmzlsreb (jxnxzgsiui )	Positive	21 May 2024
AGMT MM02 (EHA2024) Manual	Not Applicable	Multiple Myeloma Maintenance \| First line \| Induction	123	CarfilzomibCarfilzomib +( thalidomidethalidomide or lenalidomidelenalidomide )+ dexamethasone	(xuzbegznej) = were mostly hematologic in nature. ngwyivhadq (iwvevqpvje ) View more	Positive	14 May 2024
AGMT MM02 (EHA2024) Manual	Not Applicable	Multiple Myeloma Maintenance \| First line \| Induction	123	Carfilzomibthalidomidedexamethasone		Positive	14 May 2024
NCT02541383 (CASSIOPEIA, EHA2024) Manual	Phase 3	Multiple Myeloma First line \| Maintenance	-	BortezomibThalidomideDexamethasonedaratumumab	(iikbpimiiq) = yrwcnboxiz gvkyzzqoik (uazlemsieg ) View more	Positive	14 May 2024
NCT02541383 (CASSIOPEIA, EHA2024) Manual	Phase 3	Multiple Myeloma First line \| Maintenance	-	BortezomibThalidomideDexamethasone	(iikbpimiiq) = uvyhklzlad gvkyzzqoik (uazlemsieg ) View more	Positive	14 May 2024
ChiCTR1800015702 (EHA2024) Manual	Not Applicable	Beta-Thalassemia	100	Thalidomide	sxkozucjdt(lwandgxmjv) = njywbtyioh rzdnaipocw (ijrecquduy, 6.42) View more	Positive	14 May 2024
ChiCTR1800015702 (EHA2024) Manual	Not Applicable	Beta-Thalassemia	100	Placebo	sxkozucjdt(lwandgxmjv) = xqgtaceeoa rzdnaipocw (ijrecquduy, 4.98) View more	Positive	14 May 2024
GBRAM (EHA2024)	Not Applicable	Multiple Myeloma	-	Daratumumab + Bortezomib + Thalidomide + Dexamethasone	oslvvimjus(riswzvbjno) = spruhywndf kcuofqjcah (jyemtxwrio ) View more	Positive	14 May 2024
EHA2024	Not Applicable	POEMS Syndrome VEGF	50	Bortezomib + Dexamethasone	vnyqsanosl(akbjtcrdkm) = qhmpmavsim onlpytvzsv (cxiivkdxcm ) View more	Negative	14 May 2024
EHA2024	Not Applicable	POEMS Syndrome VEGF	50	Lenalidomide or Thalidomide	vnyqsanosl(akbjtcrdkm) = mcnnwgymrk onlpytvzsv (cxiivkdxcm ) View more	Negative	14 May 2024

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