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Duchenne gene therapy interim trial outcomes “striking”
The gene therapy demonstrated consistent evidence of positively changing disease trajectory in Duchenne muscular dystrophy, new interim data shows.
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Major trial indicates potential of novel hypertension drug
The demonstrated safety and efficacy of the aldosterone synthase inhibitor suggest its potential in helping patients with hypertension to reach their blood pressure goal, according to the data.
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FDA approval marks shift in diabetic retinopathy treatment
The ocular injection, now granted its third FDA approval, offers a more convenient treatment option for patients with vision loss.
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Measuring AI’s impact on the pharmaceutical industry
In this article, Updesh Dosanjh, Practice Leader, Pharmacovigilance Technology Solutions, IQVIA, explores how the pharmaceutical industry and its workforce can truly leverage the potential of artificial intelligence (AI) and measure the impact of this technology.
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Takeda investigational drug shows efficacy in narcolepsy
If approved, the oral medicine could provide a first-in-class treatment option for eligible individuals with the chronic 24-hour disease.
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Novel breast cancer treatment approach significantly enhances survival
With its detailed safety data for chemotherapy and olaparib to treat early-stage breast cancer, the trial findings could lead to improved patient outcomes.
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World-first approval supports AstraZeneca’s sustainability goals
Approval of the novel tool marks progress for both respiratory disease patients in the UK and reducing the global environmental impact of inhaled medicines.
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Innovative CAR T therapy could deliver in broader therapeutic settings, research suggests
The novel cell therapy utilises a shortened manufacturing process compared to standard production timelines, offering potential for enhanced therapeutic capability.
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FDA approves first-of-a-kind gene therapy for dystrophic epidermolysis bullosa
With clinical data showing the gene therapy can address large and challenging chronic wounds, the FDA’s decision will help improve quality of life for dystrophic epidermolysis bullosa (DEB) patients.
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CHMP meeting highlights – April 2025
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of a variety of new medicines, including several treatments for hereditary and rare conditions.
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AI-driven biomanufacturing: revolutionising production and quality in pharmaceuticals
This article explores how artificial intelligence (AI) is forming an essential pillar of staying competitive in pharmaceutical manufacturing, with its adoption ensuring greater efficiency of medicine production in this fast-evolving sector.
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Could investigational drug become first-in-class bronchiectasis therapy?
The small molecule treatment is currently being reviewed by the US Food and Drug Administration (FDA), under the Prescription Drug User Fee Act (PDUFA).
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Automated particle image analysis could optimise powder adhesion measurement
The research provides insight into effective measurement of particle adhesion, potentially enhancing pharmaceutical powder processing.
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Antibody treatment demonstrates potential in lupus kidney disease
The findings suggest that the biologic treatment could improve short-term outcomes for patients with lupus nephritis.
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FDA approves novel therapy for chronic skin condition
The US approval could improve outcomes for eligible patients with previously limited options for managing the inflammatory skin condition.
