The National Medical Products Administration ("NMPA") of China has approved the New Drug Application ("NDA") for zevorcabtagene autoleucel (R&D code: CT053, an autologous CAR-T product candidate against BCMA), for the treatment of adult patients with relapsed or refractory multiple myeloma who have previously progressed after at least 3 lines of therapy (including a proteasome inhibitor and immunomodulator agent). https://lnkd.in/gag2uvdh. #cartcelltherapy #multiplemyeloma
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A Game-Changer for Neuromyelitis Optica Spectrum Disorder Treatment: FDA Approves Ultomiris The FDA has approved Ultomiris for the treatment of adults with neuromyelitis optica spectrum disorder. The approval was based on positive results from a Phase III trial, in which Ultomiris demonstrated a significant reduction in relapse risk. Marc Dunoyer, CEO of Alexion, highlighted the potential of Ultomiris to transform the treatment landscape for this condition. The approval of Ultomiris in the US follows previous approvals in Japan and the EU. NMOSD is a rare condition that primarily affects women. Ultomiris is a long-acting C5 inhibitor that has shown promising results in reducing relapses. Overall, the approval of Ultomiris offers hope for improved outcomes for patients with NMOSD. For more details please click the link! https://lnkd.in/dyeswHtE #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical
New from JN Learning: Survival Benefit Associated With Participation in Clinical Trials of Anticancer Drugs https://lnkd.in/gZtJ_i86
STAT published this article highlighting the overwhelmingly positive data Dr. Christian Ruff from the TIMI Study Group presented this past weekend at #AHA23. “Bleeding or the fear of bleeding drives substantial under-treatment of patients, which results in devastating strokes,” said Christian Ruff, lead investigator of the trial and director of general cardiology at Brigham and Women’s Hospital. “The hope is if we had an even safer anticoagulant, that that would finally allow us to prescribe it to almost all patients and that they would actually take it, and that would be a substantial advance." #DYK, novel, dual-acting #FactorXI / XIa inhibitor, abelacimab, met its primary endpoint with a 67% reduction of major or clinically relevant non-major bleeding compared with rivaroxaban. The treatment, injected monthly, also reduced the risk of major bleeding events by 74% and gastrointestinal bleeding by 93%. Ruff also noted that "since abelacimab is a monoclonal antibody, it doesn’t have contrainteractions as many oral treatments do.” Read more from this informative interview with Elaine Chen. 👇
An investigational drug aimed at preventing strokes significantly reduced the risk of bleeding compared with a commonly used blood thinner for stroke prevention. https://trib.al/tZ9pzw7
https://buff.ly/3WK0hLy Endeavor's IPF drug ENV-101 showed significant improvements in lung function during phase 2 trials. 41 participants with IPF were given ENV-101 or a placebo daily. Results after 12 weeks indicated a 1.9% increase in percent predicted forced vital capacity (ppFVC) in the ENV-101 group versus a 1.3% decrease in the placebo group. Total lung capacity (TLC) also increased by 200 mL on average in the treatment group, while the placebo group saw a 56 mL decline. These findings suggest a positive impact of ENV-101 on IPF progression. #IPF #LungFunction
In a Phase III trial, the novel drug achieved near-complete stabilisation of TTR proteins in ATTR-CM patients #drugdevelopment #clinicaltrials #EPRTalks
𝐓𝐡𝐞 𝐅𝐃𝐀 𝐀𝐩𝐩𝐫𝐨𝐯𝐞𝐝 𝐗𝐎𝐋𝐑𝐄𝐌𝐃𝐈 (𝐦𝐚𝐯𝐨𝐫𝐢𝐱𝐚𝐟𝐨𝐫) 𝐂𝐚𝐩𝐬𝐮𝐥𝐞𝐬, 𝐅𝐢𝐫𝐬𝐭 𝐃𝐫𝐮𝐠 𝐈𝐧𝐝𝐢𝐜𝐚𝐭𝐞𝐝 𝐢𝐧 𝐏𝐚𝐭𝐢𝐞𝐧𝐭𝐬 𝐰𝐢𝐭𝐡 𝐖𝐇𝐈𝐌 𝐒𝐲𝐧𝐝𝐫𝐨𝐦𝐞 ✅ X4 Pharmaceuticals announced that the U.S. FDA has approved 𝐗𝐎𝐋𝐑𝐄𝐌𝐃𝐈 (𝐦𝐚𝐯𝐨𝐫𝐢𝐱𝐚𝐟𝐨𝐫) capsules for use in patients 12 years of age and older with 𝐖𝐇𝐈𝐌 𝐬𝐲𝐧𝐝𝐫𝐨𝐦𝐞 (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. 💊 XOLREMDI, a selective CXC chemokine receptor 4 (CXCR4) antagonist, is the 𝐟𝐢𝐫𝐬𝐭 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 specifically indicated in patients with WHIM syndrome. 💉 The FDA granted 𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐃𝐞𝐬𝐢𝐠𝐧𝐚𝐭𝐢𝐨𝐧 to mavorixafor in WHIM syndrome and evaluated the NDA under 𝐏𝐫𝐢𝐨𝐫𝐢𝐭𝐲 𝐑𝐞𝐯𝐢𝐞𝐰. The FDA approval of XOLREMDI was based on results of the pivotal, 4WHIM Phase 3 clinical trial, a global, randomized, double-blind, placebo-controlled, 52-week multicenter study in 31 people aged 12 years and older diagnosed with WHIM syndrome. 🗒 With the FDA approval of XOLREMDI, X4 has received a 𝐑𝐚𝐫𝐞 𝐏𝐞𝐝𝐢𝐚𝐭𝐫𝐢𝐜 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 𝐏𝐫𝐢𝐨𝐫𝐢𝐭𝐲 𝐑𝐞𝐯𝐢𝐞𝐰 𝐕𝐨𝐮𝐜𝐡𝐞𝐫 to obtain priority review for a subsequent application.
Pharmaxis (ASX: PXS) is pleased to report the FDA has cleared its protocol amendment to add PXS‐5505 to the treatment regimen for myelofibrosis patients who are already using the JAK inhibitor, ruxolitinib. These new patients will form a new combination arm of the Phase 2 clinical trials which reported interim data last month. Pharmaxis CEO Gary Phillips said, "This study will be run as an extra cohort of the ongoing monotherapy study and therefore we can get a very fast start with no need to set up new trial centres. This combination holds much promise for myelofibrosis patients with the potential to deliver a disease modifying effect." Click below to view the commentary in full within Pharmaxis' quarterly shareholder update. A video presentation covering the FDA news and June quarter can also be viewed here: https://lnkd.in/dKGRDJnz #fibrosis #myelofibrosis #clinicaltrials #fda #bonemarrowcancer
Oncternal Therapeutics Doses First Patient with ONCT-534 in the Fourth Cohort of P-I/II Study for Treating of R/R mCRPC #oncternaltherapeutics #onct534 #rrmcrpc #clinicaltrial #p-i #p-ii #safety #tolerability #efficacy #pk #src #arpis #enzalutamide #abiraterone #apalutamide #darolutamide
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