EMA Greenlights Aucatzyl for Adult ALL
At its May 2025 meeting, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) gave a conditional marketing authorization in the European Union for obecabtagene autoleucel (Aucatzyl) to treat adults from 26 years of age with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (B ALL).
A conditional marketing authorization is granted to a medicinal product that fulfils an unmet medical need when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required.
B ALL is a fast-growing and life-threatening cancer. Despite multiple available therapeutic options, it is associated with significant mortality and a poor survival rate.
Aucatzyl, whose active substance is obecabtagene autoleucel, is an antineoplastic cell and gene therapy. The autologous immunotherapy consists of the patient's own T cells engineered to express a chimeric antigen receptor that recognizes and binds to CD19 on target cells. This results in activation of the immunological effect of the T-cell releasing inflammatory cytokines and chemokines, killing CD19-expressing cells.
The CHMP said that the benefits of obecabtagene autoleucel were its ability to induce remission with a relevant duration in adults with relapsed or refractory acute lymphoblastic leukemia.
Treatment Achieved Durable Response
The positive decision by the CHMP was based on the results of the FELIX study, a single-arm, open-label phase 1b-2 multicenter study of obecabtagene autoleucel in adults aged 18 years and over with relapsed or refractory B ALL.
In the study, around 64% of patients had a durable response — a period without disease signs or symptoms after treatment — with a median duration of 14 months. Around 49% showed a complete response, meaning the signs of cancer disappeared.
Aucatzyl will be available as a 410 x 106 cells dispersion for infusion.
The drug's most common side effects include cytokine release syndrome, infections, musculoskeletal pain, pyrexia, pain, nausea, diarrhea, headache, fatigue, and hemorrhage.
To confirm the safety and efficacy of Aucatzyl, the manufacturer has been requested to submit long-term follow-up results of the FELIX study and to conduct a non-interventional study based on a patient registry.
In the meantime, in its overall assessment of the available data, the Committee for Advanced Therapies — the EMA's expert committee for cell- and gene-based medicines — found that the benefits of Aucatzyl outweighed the possible risks in patients with ALL, and thus the marketing authorization was granted.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Medscape
8 hours ago
- Medscape
Novo's Ozempic Linked to Rare Cases Of Serious Eye Disorder, EU Regulator Says
(Reuters) -Novo Nordisk's popular weight-loss and diabetes drugs Wegovy and Ozempic may in very rare cases cause a serious eye condition that can lead to vision loss, the European Medicines Agency's safety committee said on Friday. In the past, studies in type-2 diabetes patients have linked Ozempic to the condition called non-arteritic anterior ischemic optic neuropathy (NAION). But this is the first time a regulator has confirmed the side effect. The condition may affect up to 1 in 10,000 people taking semaglutide, the active ingredient in Wegovy and Ozempic as well as in Novo's other diabetes drug Rybelsus, for at least one year, the regulator said. NAION is the second-most common cause of blindness due to optic nerve damage, after glaucoma. "This has been reported as a potential risk for some time, so I think the clinical community is relatively aware of it. I don't see this as making any major difference to prescribing patterns," said Barclays analyst Emily Field. U.S.-listed shares of the Danish drugmaker were up nearly 2.5% in early trading. The EMA, which started its review in December, has asked Novo to add NAION as a side effect of very rare frequency in the product information accompanying drugs that contain semaglutide. Novo said it would work with the EMA to update the labels, adding clinical trials and after-market studies did not suggest a reasonable possibility that the drugs caused the condition. The "benefit-risk profile of semaglutide remains favorable," the company said in a statement. Novo has recently faced investor concerns that it is losing its first-mover advantage in the highly competitive obesity treatment market, leading the company to oust CEO Lars Fruergaard Jorgensen in May. Wegovy and Eli Lilly's Zepbound currently dominate the weight-loss drug market, potentially worth about $150 billion by the next decade. The EMA said several large studies in type-2 diabetes patients have suggested that use of Novo's drugs could raise the risk of developing NAION by twofold. A study of nearly 350,000 diabetes patients published in March showed that the risk of developing NAION more than doubled after two years of treatment with Ozempic, compared to patients taking medicines from other classes. The U.S. Department of Health and Human Services did not immediately respond to a Reuters request for comment on whether the Food and Drug Administration was conducting a probe into the side effect. (Reporting by Manas Mishra and Mariam Sunny in Bengaluru; Editing by Shilpi Majumdar)
Yahoo
12 hours ago
- Yahoo
Novo Nordisk's Wegovy Tied To Increased Risk Of Rare Vision-Loss Disorder, European Regulators Say
The European Medicines Agency's (EMA) safety committee (PRAC) has concluded its review of medicines containing semaglutide following concerns regarding a possible increased risk of developing non-arteritic anterior ischemic optic neuropathy (NAION), an eye condition that may cause vision loss. Novo Nordisk A/S's (NYSE:NVO) semaglutide, a GLP-1 receptor agonist, is the active substance in certain medicines used for diabetes and obesity (Ozempic, Rybelsus, and Wegovy). After reviewing all available data on NAION with semaglutide, including data from non-clinical studies, clinical trials, post-marketing surveillance and the medical literature, the committee on Friday concluded that NAION is a very rare side effect of semaglutide (meaning it may affect up to 1 in 10,000 people taking semaglutide).Results from several large epidemiological studies suggest that exposure to semaglutide in adults with type 2 diabetes is associated with an approximately two-fold increase in the risk of developing NAION compared with people not taking the medicine. This corresponds to approximately one additional case of NAION per 10,000 person-years of treatment; one person-year corresponds to one person taking semaglutide for one year. Data from clinical trials also point to a slightly higher risk of developing the condition in people taking semaglutide compared with people taking a placebo (a dummy treatment). Therefore, EMA has recommended that the product information for semaglutide medicines be updated to include NAION as a side effect with a frequency of 'very rare.' In December, a study conducted in Denmark and Norway revealed a potential link between the use of semaglutide for type 2 diabetes and an increased risk of non-arteritic anterior ischemic optic neuropathy (NAION). Additionally, New research suggests that GLP-1 receptor agonists, widely prescribed for diabetes and weight loss, may significantly raise the risk of age-related macular degeneration in older diabetic patients. A study published on Thursday in JAMA Ophthalmology by the University of Toronto researchers warns of a potential downside: increased risk of neovascular age-related macular degeneration (nAMD) in diabetic users. nAMD is a severe form of age-related macular degeneration characterized by the growth of abnormal blood vessels in the back of the eye. Analyzing medical data from over 1 million Ontario residents with diabetes, researchers identified 46,334 patients prescribed GLP-1 RAs, most of whom were taking semaglutide—likely Ozempic, since Wegovy was only recently approved in Canada. Patients on these medications were matched with participants not on GLP-1 RAs, sharing similar demographics and health profiles, and tracked for three years. The study found that diabetic patients taking GLP-1 RAs for at least six months had double the risk of developing neovascular AMD compared to non-users. Over a mean follow-up period of 2.4 years, 0.2% of the GLP-1 RA cohort progressed to nAMD compared with only 0.1% of control participants, resulting in an increased hazard for nAMD of 2.21 for the GLP-1 RA cohort. The risk was also highest in those with the longest duration of exposure. The Guardian report added that those on the medications for more than 30 months had over three times the risk. The risk was even greater in older patients and those with a prior stroke. Recent findings from the 2025 American Clinical Society of Oncology annual conference suggested that patients using weight loss drugs, including Ozempic, were about 33% more likely to be diagnosed with kidney cancer compared to matched individuals not using the medication. Price Action: NVO stock is up 2.45% at $74.43 at the last check Friday. Read Next:Photo by Tobias Arhelger via Shutterstock Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market. Get the latest stock analysis from Benzinga? NOVO NORDISK (NVO): Free Stock Analysis Report This article Novo Nordisk's Wegovy Tied To Increased Risk Of Rare Vision-Loss Disorder, European Regulators Say originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
Yahoo
12 hours ago
- Yahoo
Apellis and Sobi Announce EMPAVELI® (pegcetacoplan) Showed Sustained Efficacy at One Year in Phase 3 Study for C3G and Primary IC-MPGN
Robust proteinuria reduction and stable kidney function were maintained across a broad population of patients No new safety signals were observed New data presented at late-breaking session at the European Renal Association Congress Marketing applications for EMPAVELI are under review with the FDA and EMA WALTHAM, Mass. and STOCKHOLM, June 06, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® (STO:SOBI) today presented new data from the open-label period of the Phase 3 VALIANT study, investigating EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The data were presented as part of a late-breaking session at the European Renal Association (ERA) Congress. In the VALIANT study, EMPAVELI demonstrated a statistically significant 68% proteinuria reduction versus placebo at Week 26, which was sustained at one year. Additionally, patients treated with EMPAVELI continued to achieve stabilization of kidney function as measured by estimated glomerular filtration rate (eGFR). 'The one-year Phase 3 results are very compelling, confirming EMPAVELI's sustained benefits across key markers of disease,' said Fadi Fakhouri, M.D., Ph.D., presenting author, co-lead principal investigator for the VALIANT study, and professor of nephrology at CHUV Lausanne, Switzerland. 'Given the high risk of kidney failure, treatment efficacy is incredibly important to C3G and primary IC-MPGN patients, many of whom are in the prime of their lives. These data further underscore the potential of EMPAVELI to make a meaningful difference for patients.' In patients who switched from placebo to EMPAVELI at the start of the open-label period, EMPAVELI demonstrated a similar magnitude of benefit in proteinuria reduction and stabilization of kidney function. 'These data reinforce the strength of the EMPAVELI efficacy and safety profile across a broad population of patients with C3G and primary IC-MPGN, including adults and adolescents with native and post-transplant kidney disease,' said Peter Hillmen, M.B., Ch.B., Ph.D., chief medical advisor, rare disease, Apellis. 'With an FDA decision this summer, we look forward to bringing EMPAVELI to patients living with these rare and severe kidney diseases as quickly as possible.' EMPAVELI showed favorable safety and tolerability, consistent with its established profile. There were no new safety signals. 'The results from the Phase 3 VALIANT study underscore the potential of EMPAVELI in addressing the urgent needs of patients living with the kidney diseases C3G and primary IC-MPGN,' said Nils Kinnman, M.D., Ph.D., head of medical affairs and clinical development, Sobi. 'These studies are an example of Sobi's commitment to advance innovative therapies that make a meaningful difference in patients' lives.' Eight presentations highlight substantial clinical advances in rare kidney disease A total of eight presentations, including six on podium, will be highlighted at the meeting. The presentations will showcase clinically meaningful results from the Phase 3 VALIANT study, among other data. Additionally, two abstracts were selected by congress organizers as the Top 10 best ERA abstracts. The 'Top 10' are deemed significant studies underlining the growing field of clinical research in kidney disease. About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis.1 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence.2 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.3 About the VALIANT StudyThe VALIANT Phase 3 study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients with native and post-transplant kidneys. Study participants were randomized to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline. About Pegcetacoplan in Rare DiseasesPegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across hematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally. About the Apellis and Sobi CollaborationApellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit or follow us on X (Twitter) and LinkedIn. About Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at and LinkedIn. Apellis Forward-Looking Statement Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements' within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding plans to submit applications for regulatory approval for the treatment of patients with C3G and IC-MPGN. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether systemic pegcetacoplan will receive approval for those indications from the FDA or equivalent foreign regulatory agencies when expected or at all; and any other factors discussed in the 'Risk Factors' section of Apellis' Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Contacts: Apellis MediaTracy Vineismedia@ Investors Neil Carnahan, Sobi For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. References1. C3 glomerulopathy. National Institute of Health, Genetics Home Reference. Accessed November 21, 2019. 2. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474.3. Data on file using literature 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
