Small Caps - UK’s Post

Approved autologous CAR-T cell immunotherapies were in the spotlight of FDA last week. For BCMA- or CD19-directed chimeric antigen receptor T-cell therapies, the FDA announced it now requires a boxed warning. The warning to highlight the serious risk of T cell malignancies is based on investigations that started in November 2023, as well as issued label changes for all 6 relevant products made in January 2024. Based on an evaluation of data from postmarketing adverse event and clinical trial reports, FDA stated that mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes.  How this risk affects use of these products remains to be seen by oncology community in the future. https://lnkd.in/eFNRigSQ). #cancer therapy #cell therapy #CAR-T #FDA #clinical oncology #T cell

Pharmacist-Led Interventions Reduce Medication-Related Problems in Cancer Care Pharmacist-led interventions significantly improve treatment adherence and reduce medication-related problems (MRPs) in cancer patients, according to a systematic review and meta-analysis. The study found that these interventions increased treatment adherence by 4.79 times, decreased adverse drug events (ADEs) by 1.28, and reduced overall MRPs by 0.53. The findings suggest the importance of integrating pharmacists into cancer care teams. For more details please click the link! https://lnkd.in/dApMAm-Q #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

The Ministry of Food and Drug Safety of Republic of Korea (MFDS) has approved #Opdivo (#nivolumab) in combination with #cisplatin and #gemcitabine for the first-line treatment of unresectable or metastatic urothelial cell carcinoma (#UCC). This approval is backed by the Phase 3 CheckMate-901 trial, which showed significant improvements in median overall survival and progression-free survival compared to the standard chemotherapy regimen. #urothelialcellcarcinoma #immunotherapy #oncology https://lnkd.in/gicTnRqh

05/04/2024 — The U.S. FDA has granted Enhertu (fam-trastuzumab deruxtecan-nxki) accelerated approval for the treatment of adult patients with unresectable or metastatic HER2-positive solid tumours who have received prior systemic treatment and for whom no satisfactory alternative treatment is available. Fam-trastuzumab deruxtecan-nxki is a HER2-directed antibody and topoisomerase inhibitor conjugate that has 3 components: (1) a humanized anti-HER2 IgG1 monoclonal antibody that is covalently linked to a (2) topoisomerase inhibitor via a (3) tetrapeptide based cleavable linker. After binding to HER2 on tumour cells, the drug undergoes internalization and lysosomal enzymes cleave the linker. Upon release, the membrane-permeable topoisomerase inhibitor causes DNA damage and apoptotic cell death. The review of Enhertu was conducted under Project Orbis: an initiative of the FDA Oncology Centre of Excellence (OCE) established in 2019 to provide a framework for the submission and review of oncology products among international partners. The aim of this initiative is to allow cancer patients to receive earlier access to products in countries where there may be significant delays in regulatory submissions. For Enhertu, the U.S. FDA collaborated with the Australian TGA, the Brazilian ANVISA, Health Canada, and Singapore’s HSA. #regulatoryaffairs #regulatoryupdates #oncology

🚀 A New Horizon in Multiple Myeloma Treatment as FDA Approves First BCMA-Targeted Therapy CARVYKTI® by Johnson & Johnson The #FDA has approved CARVYKTI® as a treatment for relapsed or refractory multiple myeloma. It is the first B-cell maturation antigen (BCMA)-targeted therapy for this condition. The approval is based on the positive results from the Phase 3 CARTITUDE-4 study, which showed that CARVYKTI® significantly reduced the risk of disease progression or death compared to conventional treatments. CARVYKTI® uses the patient's immune system to target and eliminate BCMA-expressing cells. Despite safety concerns, the overall benefit-risk profile of CARVYKTI® is favorable. This approval provides hope for patients with multiple myeloma and highlights the importance of innovative therapies in #cancer treatment. For more details please click the link! https://lnkd.in/dEQNjhaT Author : Market Access Today #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical

📢 FDA accelerated approval for Genmab’s bi-specific epcoritamab for 3L r/r follicular lymphoma ➤ Efficacy evaluated in EPCORE NHL-1 an open-label single-arm trial (n=127) ➤ ORR was 82% inc. 60% CR. median DOR was not reached. The 12-month Kaplan-Meier estimate for DOR was 68.4% ➤ PMR to verify the clinical benefit is an ongoing Phase 3 trial (NCT05409066) evaluating rituximab + lenalidomide alone vs. in combination with epcoritamab in patients with 2L r/r FL. This study is currently >95% enrolled; PFS is the primary endpoint https://lnkd.in/eQPK_BV9 #oncology #biotech #biotechnology #regulatoryaffairs #pharma #drugdevelopment #oncologytrials #oncologyresearch #cancer #oncology #regulatoryintelligence #regulatoryprecedent

🔬𝗧𝗿𝗮𝘀𝘁𝘂𝘇𝘂𝗺𝗮𝗯 𝗕𝗶𝗼𝘀𝗶𝗺𝗶𝗹𝗮𝗿 𝗛𝗲𝗿𝘄𝗲𝗻𝗱𝗮® 𝗔𝗽𝗽𝗿𝗼𝘃𝗲𝗱 𝗯𝘆 𝗘𝗖!🚀   The European Commission (EC) has recently approved Herwenda®, a trastuzumab biosimilar developed by EirGenix, Inc. This is big news because trastuzumab is a key treatment for HER2-positive breast cancers, and having more options like Herwenda® can make a real difference for patients.💪   The approval came after positive results from clinical trials, showing that Herwenda® is just as effective as the original drug. It's great to see more options becoming available for patients in need.🎉💊 Read more: https://lnkd.in/dnPMWC4J   As soon it is available, you can order this biosimilar at Evidentic. If you're ready to use licensed drugs in your research, contact us.(Link in the comments) We're here to meet your needs and help you source the clinical-grade molecules you require.💡   #Evidentic #ClinicalGradeMolecules #ResearchAndDevelopment #DrugDevelopment #BetterHealth #PharmaInnovation #BiotechInnovation #HealthcareInnovation 🧪💊

Amgen criticized for keeping high-dose sotorasib on the market Sen. Dick Durbin (D-IL) is criticizing a move to keep high-dose sotorasib (Lumakras—Amgen) on the market instead of replacing it with a lower-dose option that is safer and more affordable but just as effective in treating non-small cell lung cancer. The manufacturer has not publicly accounted for the logic underpinning its decision to continue marketing the 960-mg dose, which consists of eight tablets that carry a list price of $100 each based on July 2023 data. Shifting to the 240-mg formulation, which works equally well, would require users to take only two tablets per dose. The less-potent dose curtails toxicity and adverse effects; however, it also would translate to a 75% drop in revenue. "The decision not to pursue the reduced dosage comes at a significant cost to patients and American taxpayers, as evidenced by the $124 million cost of Lumakras to just the Medicare program in 2022 alone," Durbin noted in a letter to Amgen CEO Robert Bradway. A spokeswoman with the company said Amgen will respond to the lawmaker's outreach, but she maintained that an FDA-mandated aftermarket study showed that the higher dose of Lumakras to have the better risk-benefit profile and to be more effective than the lower dose. https://lnkd.in/d4e7tvSa

Will European #oncologists embrace Pfizer's new bispecific antibody to treat #multiplemyeloma patients? To get a better understanding of how the market for these therapies is evolving we are fielding a short poll to European oncologists. Learn more here: https://lnkd.in/dpxQqsFa #myeloma #oncology #pharmaceuticals

Another cell therapy bumped up the treatment line showing confidence and more importantly safe and efficacious results in patients. With these therapies moving up to the frontline companies will have much larger patient populations they can treat, but then of course face new challenges (scale, throughput, skills, footprint etc etc) in making much more of them! #celltherapy #cart bluebird bio 2seventy bio #abecma *EDIT* I should really finish reading the news before I post...Carvykti has gained extended approval as well bringing it closer to more patients! The Janssen Pharmaceutical Companies of Johnson & Johnson Legend Biotech