Latest news with #multiplemyeloma
New York Times
3 days ago
- Business
- New York Times
From No Hope to a Potential Cure for a Deadly Blood Cancer
A group of 97 patients had longstanding multiple myeloma, a common blood cancer that doctors consider incurable, and faced a certain, and extremely painful, death within about a year. They had gone through a series of treatments, each of which controlled their disease for a while. But then it came back, as it always does. They reached the stage where they had no more options and were facing hospice. They all got immunotherapy, in a study that was a last-ditch effort. A third responded so well that they got what seems to be an astonishing reprieve. The immunotherapy developed by Legend Biotech, a company founded in China, seems to have made their cancer disappear. And after five years, it still has not returned in those patients — a result never before seen in this disease. These results, in patients whose situation had seemed hopeless, has led some battle-worn American oncologists to dare to say the words 'potential cure.' 'In my 30 years in oncology, we haven't talked about curing myeloma,' said Dr. Norman Sharpless, a former director of the National Cancer Institute who is now a professor of cancer policy and innovation at the University of North Carolina School of Medicine. 'This is the first time we are really talking seriously about cure in one of the worst malignancies imaginable.' The new study, reported Tuesday at the annual conference of the American Society of Clinical Oncology and published in The Journal of Clinical Oncology, was funded by Johnson & Johnson, which bought Legend Biotech. Want all of The Times? Subscribe.
Associated Press
4 days ago
- Business
- Associated Press
Press Release: ASCO: new Sarclisa data support subcutaneous administration with on-body injector
ASCO: new Sarclisa data support subcutaneous administration with on-body injector Paris, June 3, 2025. New data from two clinical studies of the investigational use of Sarclisa administered subcutaneously (SC) via an on-body injector (OBI) (also referred to as an on-body delivery system) in relapsed or refractory multiple myeloma (R/R MM) support the potential use of this innovative delivery method to advance patient care, while upholding Sarclisa's efficacy and safety profile. The results were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting and include full data from the IRAKLIA phase 3 study, the first to incorporate the use of an OBI in the treatment of MM, and demonstrate non-inferior efficacy and pharmacokinetics compared to Sarclisa intravenous (IV) infusion. Alyssa Johnsen, MD, PhD Global Therapeutic Area Head, Immunology and Oncology Development 'Our subcutaneous clinical program is rooted in our mission to address patient needs and reduce treatment burden in multiple myeloma. We believe the novel on-body injector represents a significant innovation thatcouldimprove and streamline the treatment process for both patients and providers. We are pleased to share thesedata, the first to evaluate an on-body injector with a multiple myeloma treatment, and look forwardtopotentiallybringing this formulation and administrationoptionto the multiple myeloma community.' The OBI offers the potential to improve the overall patient experience in MM treatment. Recent studies and surveys suggest the use of an OBI may be associated with greater convenience, flexibility, and patient satisfaction compared to IV or manual SC administration methods.1 In addition, an OBI may also streamline the administration process for providers, potentially reducing the physical burden on nurses and enabling them to possibly move freely through the use of a hands-free device while monitoring the patient during injection. The IRAKLIA phase 3 study and the IZALCO phase 2 study presented at ASCO were conducted using Enable Injections' enFuse® hands-free OBI, an automated injector designed to subcutaneously administer high-volume medicines beginning with the click of a button, to administer the hyaluronidase-free SC formulation of Sarclisa. The enFuse device uses a 30 gauge, hidden, and retractable needle that is smaller compared to some of the commonly used large-volume SC injection needles, which may support patient comfort. The safety and efficacy of Sarclisa SC administered with the OBI or manual administration are investigational and have not been approved for use by any regulatory authority. IRAKLIA phase 3 study IRAKLIA is a global, randomized, open-label, pivotal phase 3 non-inferiority study comparing Sarclisa SC administered via an OBI and Sarclisa IV, both in combination with pomalidomide and dexamethasone (Pd) in adult patients with R/R MM who have received at least one prior line of treatment. At the data cut-off of November 6, 2024, and a median follow-up of 12 months, the study demonstrated: Primary endpoints Secondary endpoints The overall safety profile of Sarclisa SC-Pd observed in this study was consistent with the established safety profile of Sarclisa IV-Pd, but with a notably lower rate of systemic IRs. No new safety concerns were observed, except for low-grade local injection site reactions (ISRs) associated with SC administration that occurred with a low incidence (0.4%, n=19/5,145 injections). Nearly all ISRs were grade 1, except for one episode of grade 2. Xavier Leleu, MD, PhD Head of the Department of Hematology and Myeloma Clinic at the Hôpital La Mileterie and study investigator 'Results from the IRAKLIA phase 3 study represent a potentially transformational advancement in the administration of multiple myeloma treatment. These data not only establish non-inferiority between Sarclisa administered both subcutaneously and intravenously across several key endpoints but reinforce the positive impact that this on-body injector could have on the patient treatment experience, as demonstrated by patient satisfaction scores.' In addition to the oral presentation at ASCO, the full data were simultaneously published in the Journal of Clinical Oncology. IZALCO phase 2 study In addition to the IRAKLIA phase 3 study, Sanofi also presented new data from the randomized, sequential, open-label, IZALCO phase 2 study evaluating the efficacy and safety of Sarclisa SC administered via manual push or an OBI, in combination with carfilzomib and dexamethasone (Kd) in adult patients with R/R MM who have received one to three prior lines of therapy. At a median follow-up of 10.1 months, the study demonstrated: The overall safety profile of Sarclisa SC-Kd observed in this study was consistent with the established safety profile of Sarclisa IV-Kd, with no new safety concerns observed. Advancing patient and provider-centric innovation in MM While SC administration is currently available for certain MM treatment regimens through a manual injection, administering large-volume medicines manually can present significant challenges, including a labor-intensive process for nurses, risk of strain and needlestick injuries, and potential need for larger needles that may compromise patient comfort and increase anxiety. Mehul Desai, PharmD, MBA Vice President, Medical Affairs, Enable Injections 'We believe multiple myeloma patients deserve a more convenient and comfortable treatment experience and recognize the crucial role providers play in delivering that care. Through our collaboration with Sanofi, we've aspired to advance an on-body injector that could transform the treatment experience for patients and providers alike. The results from the IRAKLIA and IZALCO studies represent a significant step toward our ambition and validate the potential of the on-body injector to deliver the same high standard of efficacy established with intravenous Sarclisa.' In addition to IRAKLIA and IZALCO, Sanofi is also evaluating Sarclisa SC administration via an OBI in the front-line treatment setting. The ISASOCUT phase 2 study conducted by the University of Poitiers, is evaluating Sarclisa in combination with bortezomib, lenalidomide and dexamethasone (VRd) in adult patients with newly diagnosed MM (NDMM) not eligible for autologous stem-cell transplant (ASCT), while the German-speaking Myeloma Multicenter Group (GMMG)-HD8 phase 3 study, conducted in collaboration with the GMMG and the German Multiple Myeloma Study Group Consortium (DSMM), is evaluating Sarclisa SC-VRd induction in NDMM patients who are eligible for ASCT. In addition, results from the IZALCO, IRAKLIA and ISASOCUT studies will be presented at the European Hematology Association Congress later this month. The IRAKLIA abstract was also hand-selected to be included in the 2025 Best of ASCO program, held later in the summer of 2025, following the ASCO Annual Meeting. The data from these studies, collectively, will form the basis for global regulatory submissions. Sarclisa administered subcutaneously via the on-body injector or manual administration is investigational and has not been approved for any use by any regulatory authority. The safety and efficacy of this formulation and delivery method have not been established. About the IRAKLIA and IZALCO studies IRAKLIA is a randomized, open-label, pivotal phase 3 study evaluating the non-inferiority of Sarclisa SC formulation administered at a fixed dose SC via an OBI versus weight-based dosed Sarclisa IV in combination with Pd in adult patients with R/R MM who have received at least one prior line of therapy. The co-primary outcomes being assessed are ORR, defined as the proportion of patients with stringent CR, CR, VGPR, and partial response (PR) according to the 2016 IMWG criteria assessed by Independent Review Committee (IRC), and observed C trough at steady state (pre-dose at C6D1), defined as observed Sarclisa plasma concentrations. IZALCO is a two-part, randomized, sequential, open-label, phase 2 study evaluating the efficacy and safety of Sarclisa SC formulation administered SC via manual push or an OBI in adult patients with R/R MM who have received one to three prior lines of therapy. The primary objective is ORR, as assessed by IRC. The secondary objective is patient preference for the OBI versus manual administration of Sarclisa SC. About Enable Injections Based in the US (Cincinnati, OH), Enable Injections is a global healthcare innovation company committed to improving the patient treatment experience through the development and manufacturing of enFuse. enFuse is an innovative wearable drug delivery platform that is designed to deliver large volumes of pharmaceutical and biologic therapeutics via subcutaneous administration, with the aim of improving convenience, supporting superior outcomes, and advancing healthcare system economics. For more information, visit About Sarclisa Sarclisa (isatuximab) is approved in more than 50 countries, including in the US, EU, Japan, and China, across multiple treatment lines for MM. Based on the ICARIA-MM phase 3 study, Sarclisa is approved in the US, EU and Japan in combination with Pd for the treatment of patients with R/R MM who have received ≥two prior therapies, including lenalidomide and a proteasome inhibitor and have relapsed on the last therapy; this combination is also approved in China for patients who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor. Based on the IKEMA phase 3 study, Sarclisa is also approved in more than 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with R/R MM who have received one to three prior lines of therapy and in the EU for patients with MM who have received at least one prior therapy. In the US, EU, UK, and China, Sarclisa is approved in combination with VRd as a front-line treatment option in transplant-ineligible NDMM patients, based on the IMROZ phase 3 study. In Japan, Sarclisa is approved in combination with VRd as a front-line treatment option regardless of transplant eligibility. At Sanofi, we are building on a long-standing commitment to oncology as we continue to chase the miracles of science to improve the lives of those living with cancer. We are committed to transforming cancer care by developing innovative, first and best-in-class immunological and targeted therapies for rare and difficult-to-treat cancers with high unmet need. For more information on Sarclisa clinical studies, please visit About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and creating compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people's lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media Relations Sandrine Guendoul | +33 6 25 09 14 25 | [email protected] Evan Berland | +1 215 432 0234 | [email protected] Léo Le Bourhis | +33 6 75 06 43 81 | [email protected] Victor Rouault | +33 6 70 93 71 40 | [email protected] Timothy Gilbert | +1 516 521 2929 | [email protected] Investor Relations Thomas Kudsk Larsen |+44 7545 513 693 | [email protected] Alizé Kaisserian | +33 6 47 04 12 11 | [email protected] Felix Lauscher | +1 908 612 7239 | [email protected] Keita Browne | +1 781 249 1766 | [email protected] Nathalie Pham | +33 7 85 93 30 17 | [email protected] Tarik Elgoutni | +1 617 710 3587 | [email protected] Thibaud Châtelet | +33 6 80 80 89 90 | [email protected] Yun Li | +33 6 84 00 90 72 | [email protected] Sanofi forward-looking statements All trademarks mentioned in this press release are the property of the Sanofi group with the exception of enFuse. Attachment
Yahoo
25-05-2025
- Business
- Yahoo
US FDA Votes in Favor of Benefit-Risk Profile of Johnson & Johnson's DARZALEX FASPRO
On May 20, Johnson & Johnson (NYSE:JNJ) announced that the 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 treating adult patients with high-risk smoldering multiple myeloma (HR-SMM). This recommendation is a step towards potentially making DARZALEX FASPRO the first treatment to delay or prevent the progression of this condition to active multiple myeloma. A closeup of pills in a pharmacy, representing the high quality medications of the company. An application for this approval was submitted to the FDA in November 2024. Currently, there are no approved treatments specifically for HR-SMM. The ODAC's decision was based on data from the Phase 3 AQUILA study. This was a randomized and open-label trial that evaluated the efficacy and safety of DARZALEX FASPRO versus active monitoring in HR-SMM patients. Smoldering multiple myeloma is an asymptomatic intermediate stage of multiple myeloma. However, ~50% of patients with HR-SMM are likely to develop active disease within 2 to 3 years. The current standard of care for SMM is active monitoring, which can lead to therapeutic intervention only after the detection of end-organ damage. Multiple myeloma is a blood cancer affecting plasma cells in the bone marrow, and it remains incurable. DARZALEX FASPRO received US FDA approval in May 2020 and is currently approved for 9 indications in multiple myeloma, including 4 for frontline treatment. Johnson & Johnson (NYSE:JNJ) engages in the R&D, manufacture, and sale of various products in the healthcare field worldwide. In August 2012, Janssen Biotech Inc. (a subsidiary of Johnson & Johnson) and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture, and commercialize daratumumab. This is the active ingredient in DARZALEX FASPRO. While we acknowledge the potential of JNJ to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than JNJ and that has 100x upside potential, check out our report about the cheapest AI stock. READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
Medscape
23-05-2025
- Health
- Medscape
Blenrep Gets European Nod to Treat Multiple Myeloma
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion for Blenrep (belantamab mafodotin) for the treatment of relapsed or refractory multiple myeloma. The drug is an antibody drug conjugate comprising a humanized IgG1κ monoclonal antibody targeting the B-cell maturation antigen (BCMA), conjugated with a cytotoxic agent, maleimidocaproyl monomethylauristatin F (mcMMAF). Belantamab mafodotin binds to BCMA on the surface of myeloma cells, causing cell cycle arrest and inducing antibody-dependent cellular cytotoxicity. According to the manufacturer, GSK, it is the first such conjugate to be approved and represents a new mechanism of action compared with existing agents. Generally Poor Prognosis Multiple myeloma is a rare and incurable disease of the plasma cells, typically affecting adults older than 60 years of age. First-line therapy is usually with targeted cancer drugs such as lenalidomide or bortezomib in combination with a steroid, sometimes with chemotherapy and a stem cell transplant. Relapses are treated similarly. The disease has a generally poor prognosis and there is a need for novel effective therapies. Blenrep had been designated as an orphan medicine, and the EMA will now review the information to determine whether this can be maintained. CHMP said that two phase 3, randomized, open-label studies had shown that adding Blenrep to standard therapy of either bortezomib and dexamethasone in patients who have received at least one prior therapy, or pomalidomide and dexamethasone in patients who have received at least one prior therapy including lenalidomide, prolonged progression-free survival in patients with relapsed or refractory multiple myeloma. Ocular Side Effects Common The safety and tolerability profiles of the Blenrep combinations were broadly consistent with those of the individual agents. The most common side effects with Blenrep include reduced visual acuity, corneal examination abnormalities (including keratopathy), thrombocytopenia, blurred vision, dry eye, foreign-body sensation in eyes, eye pain, photophobia, eye irritation, neutropenia, anemia, and diarrhea. Patients should have an ophthalmic examination, including visual acuity and slit-lamp examination, by an eye care professional before each of the first four doses of Blenrep, and as clinically indicated thereafter. Patients should be encouraged to inform their physicians of any ocular symptoms. The drug will be available as 70 mg or 100 mg powder for concentrate for solution for infusion. It is administered as a 30-minute infusion every 3 or 4 weeks. Treatment should be prescribed by physicians experienced in the treatment of multiple myeloma. Detailed recommendations will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorization has been granted by the European Commission.
Medscape
22-05-2025
- Business
- Medscape
FDA Advisory: Daratumumab Wins, Glofitamab Loses
Daratumumab and hyaluronidase (Darzalex Faspro, Johnson & Johnson) came a step closer to being approved for high-risk smoldering multiple myeloma (SMM) on Tuesday after the Oncologic Drugs Advisory Committee (ODAC) of the US Food and Drug Administration (FDA) voted that the benefits outweigh the risks. However, the prospects of glofitamab (Columvi, Roche) for relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) dimmed after the committee agreed with the FDA that the results of a trial, which was conducted largely in Asia, are not really applicable to patients in the United States. Daratumumab and hyaluronidase (DARA SC) is already on the US market as a standard first-line option for MM, but there's currently no approved medication for SMM, an MM precursor. Standard treatment for SMM is either watchful waiting or referral to a clinical trial. Glofitamab, meanwhile, has accelerated approval as monotherapy in the third or later lines for R/R DLBCL. Roche was hoping to move it to an earlier line of treatment in combination with gemcitabine and oxaliplatin for transplant-ineligible disease. The FDA called the hearing because it had concerns about the trials supporting the two applications, AQUILA in the case of DARA SC and STARGLO for glofitamab. STARGLO in the United States STARGLO evaluated substituting glofitamab for rituximab on a background of gemcitabine and oxaliplatin for transplant-ineligible R/R DLBCL, not otherwise specified, following at least one line of systemic therapy. There was a statistically significant improvement in overall survival (OS), progression-free survival (PFS), and complete response (CR) with glofitamab across 274 patients. The main concern the FDA had with the trial is that almost half of the patients were from Korea, Taiwan, and China, and there were only 25 US patients. Others came from Europe and Australia. When the FDA compared outcomes of Asian vs non-Asian patients, it found significant differences. Despite a strong hazard ratio (HR) for OS benefit in Asia (HR, 0.39), there was a trend toward worse OS in Europe and the United States and in White patients, with similar trends for worse PFS and CR rates. The reasons aren't clear. 'FDA is concerned by the lack of internal consistency observed in the STARGLO trial and how the results of the Asian region appear to be driving the overall trial results,' the agency said in meeting documents. 'The low enrollment of patients in the US limits the agency's ability to assess the applicability of the study results to a US patient population,' the FDA said in meeting documents. 'Furthermore, the FDA has identified multiple differences in patient-related, disease-related, and healthcare system–related factors between the non-Asian and Asian regional subgroup populations. Taken together, these issues raise uncertainty as to whether the results…are generalizable and applicable to a US patient population,' the agency said. Among other concerns, the FDA also noted that rituximab/gemcitabine/oxaliplatin wasn't a good comparator arm for US patients because the regimen is not commonly used in the United States, which might have contributed to low enrollment at US study sites. Trial sponsor Roche highlighted the overall outcomes and that there's an unmet need for additional DLBCL treatment options. Company representatives also said that outside of Asia, patients on glofitamab had a higher risk for disease than those on rituximab, and rituximab patients were more likely to subsequently receive new anti-lymphoma therapy like CAR T cells. It pinned the low US enrollment on COVID disruptions during the pandemic. In the end, ODAC sided with the agency, voting 8 to 1 that the trial results are not applicable to US patients. Echoing many committee members, panelist Heidi McKean, MD, community oncologist in Sioux Falls, South Dakota, said she voted that the trials wasn't applicable 'due to the inconsistencies in the results…and quite frankly, more patients in the US need to be looked at to prove efficacy and safety.' Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence, said these issues in STARGLO aren't uncommon. 'Unfortunately, if you take a look at all the oncology trials that come to us, only about 20% of the population is derived from the United States. We'd like to understand the reasons why sites are not enrolling in the United States. Potentially, that could be lack of interest because many times the control arms are not appropriate for a US population,' he said. 'This is going to be an area that the Oncology Center of Excellence is looking at quite closely. People are developing drugs for marketing in the United States, so it should address our interest here in the United States,' he said. A Win for DARA SC ODAC also considered the AQUILA trial, which randomized 390 patients with SMM at a high risk for progression to MM to either DARA SC or active monitoring for up to 3 years. At a median follow-up of 65.2 months, the risk for disease progression or death was 51% lower with DARA SC than with active monitoring. At 5 years, PFS was 63.1% with DARA SC and 40.8% with active monitoring, and OS was 93.0% with DARA SC and 86.9% with active monitoring, although the trial was not adequately powered to demonstrate a significant improvement in OS. The positive results were countered by a higher incidence of grade 3/4 treatment emergent adverse events with DARA SC, 40% vs 30%. The FDA's primary concern was that the trial, which was designed almost 10 years ago, used an outdated model to select patients at high risk for progression. With current risk models, only 41% of participants would be categorized as high risk, with 39% considered intermediate risk and 20% as low risk. 'This raises concerns regarding the applicability of the trial results to a population with high-risk SMM, as currently defined,' the FDA said in meeting materials. Also, 'while the trial met its primary PFS endpoint, there is uncertainty in the benefit of delaying progression to [multiple myeloma] in the absence of a significant improvement in OS. Additionally, the observed difference in progression was primarily due to differences observed in the biochemical or lab parameters,' not the onset of symptomatic disease, the FDA said. Not all high-risk patients progress to MM, so the agency also had concerns about unnecessary treatment — particularly with the elevated risk for serious and high-grade adverse events with daratumumab. 'Given the limitations of the clinical meaningfulness of the efficacy findings and the toxicity observed with 3 years of treatment with Dara SC, there is uncertainty regarding the benefit-risk profile of Dara SC for patients with high-risk SMM,' the agency said. Johnson and Johnson countered by emphasizing that all of the trials endpoints are positive, and that without an approved medication for SMM, patients are left powerless as they wait for a MM to emerge, something a commenter likened to 'sitting on a ticking time bomb.' Vincent Rajkumar, MD, myeloma specialist at the Mayo Clinic in Rochester, Minnesota, presenting on behalf of the company, also caught the attention of panelists when he said that high-risk SMM isn't simply a benign precursor to MM, but rather cancer in itself, raising the stakes for early intervention. 'It is asymptomatic, but not premalignant. It is cancer. Genomically, [it is] indistinguishable from multiple myeloma,' he said. In the end, the company's arguments won the day. ODAC voted 6 to 2 that AQUILA provide sufficient evidence to support a favorable risk-benefit profile for DARA SC for SMM. 'The shift for me was thinking of smoldering multiple myeloma as a malignancy and allowing the physician and patient to look at this data and intervene earlier if they so choose,' McKean said. Another committee member, Christopher Lieu, MD, gastrointestinal medical oncologist at the University of Colorado Cancer Center, Aurora, Colorado, agreed. 'I really want patients and providers to have the option to discuss this, to have the benefit-risk discussion. The conversation includes the fact that there are toxicities from this drug; that there's a chance that you can prevent a life-altering fracture; that you might be able to prevent or delay at least the onset of treatment; that you might be able to delay or prevent an organ damage. I think that that is a conversation that I want patients and providers to have the option to have,' Lieu said. However, this is going to lead to overtreatment. There has to be a predictive biomarker or some type of risk stratification to refine this high-risk group,' he said. The FDA usually follows the advice of its advisory committees.
