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Orphagen’s ACC therapy receives FDA rare pediatric disease status

Pharmaceutical Technology

Orphagen Pharmaceuticals has received a rare pediatric disease designation (RPDD) for OR-449 from the US Food and Drug Administration (FDA) to treat paediatric adrenocortical carcinoma (ACC). In paediatric ACC, the transcription factor is commonly amplified at the gene level.

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Erasca receives fast track designation for glioblastoma therapy

Pharmaceutical Technology

Erasca has received the US Food and Drug Administration’s (FDA) fast track designation (FTD) for ERAS-801 to treat adults with glioblastoma (GBM) with epidermal growth factor receptor (EGFR) gene alterations.

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Nanoparticle Formulation: A Paradigm Shift towards Novel Drug Delivery

Roots Analysis

Owing to their unique size and physicochemical properties ( surface roughness, surface area, surface energy, crystal structure and shape ), nanoparticles can be widely used as a contrasting agent in medical imaging, a vesicle to cross the blood-brain barrier and a carrier for targeted delivery of genes / drugs, proteins, vaccines and antibiotics.

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Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

percent received approval from the US Food and Drug Administration (FDA) to treat dry eye disease. Furthermore, the bioavailability of the active pharmaceutical ingredient (API) into the cornea is up to four times stronger with EyeSol technology. Attendees will understand best practices for novel drug delivery design and development.

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In the News: June 2021 Regulatory and Development Updates

Camargo

At Camargo, we have worked with hundreds of investigators who have found new targets for established drugs. It is exciting to find new uses of drugs with known safety because these drugs can reach new groups of patients in a relatively short timeframe.

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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Emerging as a new treatment option in rare and orphan disease areas, oligonucleotide therapeutics have matured into a drug class with a broad indication spectrum. Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake.

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Considerations for Neuroscience Trials with Intrathecal Delivery and Other Methods of Direct CNS Administration

XTalks

According to an analysis of the 2013 Medical Expenditure Panel Survey, one in six US adults reported taking a psychiatric drug at least once in 2013. CNS drugs treat a range of neurologic and psychiatric disorders, such as psychosis, depression, Parkinson’s disease, multiple sclerosis and epilepsy.

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