The nAbCyte cell-based neutralizing antibody assay detects AAVRh74var pre-existing neutralizing antibodies, which could impact patient safety and/or efficacy with Beqvez.
Hematological Disorders
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The approval was based on data from the randomized, double-blind, placebo-controlled phase 3 4WHILM trial.
Beqvez is a one-time AAV-based gene therapy designed to introduce in the transduced cells a functional copy of the FIX gene encoding a high-activity FIX variant.
Ninety-seven percent of patients were free from vaso-occlusive crises for at least 12 consecutive months.
Rilzabrutinib is an oral, reversible, covalent Bruton tyrosine kinase inhibitor.
NM5072 is a potential first-in-class monoclonal antibody that selectively blocks properdin, a protein of the complement alternative pathway.
The table below is a review of notable updates that occurred in March 2024 for investigational products in development.
The approval was based on data from the phase 3 ALPHA trial, which evaluated danicopan as add-on therapy to ravulizumab or eculizumab in adults with PNH and clinically significant EVH.
Preexposure prophylaxis approved for COVID-19; Nexletol and Nexlizet gain expanded approval; Novel Duchenne muscular dystrophy treatment; First blood test to screen for malaria in donors; Warning letter for OTC topical analgesic products.
Vafseo is an oral reversible inhibitor of hypoxia-inducible factor (HIF)-prolyl-4-hydroxylases (PH)1, PH2, and PH3.
The comparative efficacy of these therapies has been extended to patients with stage 3 chronic kidney disease, a common comorbidity in gout.
Cevidoplenib is an investigational SYK inhibitor designed to inhibit the downstream signaling of B-cell and Fc receptors resulting in increased platelet counts.
Increase seen in hemoglobin levels, and reduction seen in fatigue and in reticulocyte and bilirubin levels.
The designation is supported by data from an ongoing phase 2 trial that is assessing the effects of elritercept in patients with very low-, low-, or intermediate-risk MDS.
The table below is a review of notable updates that occurred in February 2024 for investigational products in development (not an inclusive list). Click on the status to view our full coverage.
Real-world data support the anemia reduction found in post hoc analyses of the CREDENCE and DAPA-CKD trials.
Insufficient evidence found for assessing benefits, harms of screening and iron supplementation for preventing adverse outcomes.
DISC-0974 is an investigational monoclonal antibody against hemojuvelin, a bone morphogenetic protein-signaling co-receptor.
The on-body injector includes features such as a retractable needle mechanism, an indicator, status light and auditory signal to help patients confirm the dose has been administered.
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