How can real-world evidence support regulatory decision making?
Strategies to further accelerate real-world evidence generation to aid EU regulatory decision-making are needed, states a European Medicines Agency (EMA) report.
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First RNA CAR-T therapy autoimmunity trial shows potential
A novel RNA CAR-T cell therapy demonstrated long-term clinical benefit for most generalised myasthenia gravis (gMG) patients in a landmark study.
Reducing bioburden testing and sterility testing volumes for gene therapies
A reduced sampling plan was recommended in a Biophorum report for reducing required bioburden and sterility testing volumes for gene therapy batch yield.
Gene therapy approved for Duchenne muscular dystrophy
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
New approach for small molecule nanosimilar analysis reported
A study on the small molecule antibiotic rifabutin has highlighted benefits of mechanistic compartmental analysis in evaluating nanomedicines.
New mass spec technique identified for electron ionisation direct analysis
A paper has depicted extractive-liquid sampling electron ionisation-mass spectrometry (E-LEI-MS) as a new real-time MS technique for direct analysis.
First adult anti-inflammatory cardiovascular drug approved
An FDA-approved anti-inflammatory drug can reduce cardiovascular disease risk by an additional 31 percent in adults, on top of standard of care, clinical data has shown.
First-in-class oral small molecule trial data presented at 2023 ERA Congress
At the 2023 ERA Congress, Chinook Therapeutics revealed Phase I data for its first-in-class oral small molecule LDHA inhibitor.
How effective is nanopore sequencing for fungal identification?
A paper has described a DNA metabarcoding workflow that rapidly characterises fungal microbiota with high taxonomic resolution.
Could novel robotic oral pill replace traditional injections?
An osteoporosis study has demonstrated a high delivery success rate for a breakthrough technology that converts injections into an oral pill, data from ENDO 2023 showed.
How can AI be best harnessed for pharmacovigilance data?
A GSK-funded paper on pharmacovigilance stated that the pharma industry needs to harness the properties of data to allow optimal use of AI, otherwise "we will fail to do all we can for patient safety".
FDA approves off-the-shelf bispecific antibody cancer therapy
The US Food and Drug Administration (FDA) has approved the first bispecific antibody with a fixed-duration treatment in (R/R) diffuse large B-cell lymphoma (DLBCL).
R&D innovation advancing small molecule innovator CDMO market
Accelerating advancement in research and technology is generating opportunities for biopharma to develop innovative small molecule drugs, a report shows.
Why is microbiological testing in medical device manufacturing important?
Careful planning during manufacture means accurate microbiology testing methods can be developed, ensuring the safety and efficacy of medical devices, a paper reports.
6.1 percent CAGR expected for pharmaceutical sterility testing market
The rising importance of quality and sterility is expected to drive the pharmaceutical sterility testing market in the next decade, a report says.
