Drug Development
As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.
FEATURED STORIES
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
With immunology and inflammation blockbusters like AbbVie’s Skyrizi and Rinvoq reeling in nearly $7 billion combined in the third quarter, the pipeline-in-a-product strategy has never been more attractive.
The cholesterol-lowering drug is part of a suite of medicines that also includes MariTide and that Amgen Chief Medical Officer Paul Burton hopes will make the company the “undisputed leader in the management of cardiometabolic risk for patients” by 2030.
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Following the regulator’s denial of patisiran’s label expansion, Alnylam has published late-stage data for the RNAi therapeutic in The New England Journal of Medicine demonstrating its efficacy in ATTR-cardiomyopathy.
With the potential FDA approval of an MDMA-based therapy for PTSD on the horizon, biopharma stakeholders are eyeing psychedelics with fresh anticipation.
High multiplexed patient-centric assays could reduce patient burden
Protocol design optimization and timely engagement of regulators are the crux of optimized, patient-centric clinical trials.
New platforms are emerging to help biopharma companies fill their human studies more efficiently, but barriers remain to their successful implementation.
While the trial was designed to test safety and not efficacy, patients treated with Araclon Biotech’s experimental ABvac40 vaccine saw a 38% drop in disease progression compared to placebo.
The company’s respiratory syncytial virus vaccine Arexvy can elicit similar levels of immune protection in adults aged 50 to 59 as in its approved population, finds results from a late-stage study.
The regulator lifted a clinical hold on Verve Therapeutics’ CRISPR-based therapy clearing its Investigational New Drug application and plans to evaluate VERVE-101 in a Phase I heterozygous familial hypercholesterolemia trial.
The regulator has approved the first-ever subcutaneous version of infliximab, an IgG1 monoclonal antibody, commonly sold by Johnson & Johnson under the brand name Remicade.
The companies presented data from two pivotal studies of Dato-DXd, focusing on safety concerns and survival data for the investigational antibody-drug conjugate candidate in lung and breast cancer.