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Pharming licenses orphan drug from Novartis

Novartis AG’s small molecule phosphoinositide 3-kinase delta (PI3Kẟ) blocker CDZ173, which is in a registration-enabling study, is expected to reach the market in 2022 lastest. Activated Phosphoinositide 3-kinase Delta Syndrome (APDS) is an ultra-rare, debilitating primary immune deficiency which makes carriers of the underlying defect in the PIK3CD gene unable to fight of infections. Currently there is no approved treatment available for PIK3CD overactivity, which occurrs approximately 1-2 per million.

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