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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. A team of three cancer researchers, Michael Jung, Timothy Cloughesy and David Nathanson, designed and developed ERAS-801.

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Orphagen’s ACC therapy receives FDA rare pediatric disease status

Pharmaceutical Technology

OR-449 is a first-in-class, orally bioavailable, potent and selective small molecule antagonist of the orphan nuclear receptor steroidogenic factor-1 (SF-1, NR5A1). SF-1 is an important transcription factor for adrenal gland growth and development. In paediatric ACC, the transcription factor is commonly amplified at the gene level.

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Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

The clinical trials consistently showed significant therapeutic improvements in ocular surface damage and associated symptoms, while pioneering visual function improvement analysis”, said John D. Additionally, the clinical trials underscored the favorable tolerability profile of Vevye.

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A Complex Chromosomal Abberation Called Adrenoleukodystrophy

Delveinsight

Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. A novel, brain penetrant, orally bioavailable and selective PPAR?

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Lilly adds to pain pipeline with Asahi Kasei licensing deal

pharmaphorum

Lilly is paying $20 million upfront for exclusive rights to AK1780, a P2X7 receptor antagonist in early-stage clinical development for neuroinflammatory pain conditions, outside Japan and China. While tanezumab is an antibody and has to be delivered by subcutaneous injection, AK1780 is orally bioavailable.