17 November 2023
17 November 2023
Vertex and CRISPR's Casgevy has received conditional approval in the UK for treating sickle cell disease and transfusion-dependent beta-thalassemia.
Trial data has shown that the combination treatment reduced mortality risk by 22% versus chemotherapy alone.
The approval for the new indication is based on interim data from Part 1 of the Phase III RUBY clinical trial.
Ardelyx scores another FDA win after Xphyozah was approved last month as an add-on therapy for chronic kidney disease patients.
The mRNA therapy may help to accelerate the regeneration of the urinary sphincter muscle, treating women with stress urinary incontinence.
Japan will use DS-5670 for its ongoing Covid-19 inoculation drive programme, launched in 2023.
Augtyro (repotrectinib) is the only FDA-approved tumour kinase inhibitor (TKI) for ROS1-positive non-small cell lung cancer (NSCLC).
Certa plans to discuss trial plans with the FDA in early 2024, with the trial set to begin later in the year.
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As PD therapy currently centers on symptomatic treatment, the need for DMTs is one of the greatest unmet needs. Several companies within the late-stage PD pipeline are developing drugs that target PD via novel MOAs. Targeting α-synuclein and other neurotoxic proteins is a key strategy in the late-stage pipeline for DMTs. However, the lack of validated predictive biomarkers of PD progression have made the development of DMTs and neuroprotective agents challenging.
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