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Novartis: ultra-rare disease drug gets EMA's priority review

(CercleFinance.com) - Swiss drugmaker Novartis said on Friday that its iptacopan pill has received priority designation from the European Medicines Agency (EMA) as a treatment for C3 glomerulopathy, an ultra-rare disease.


Novartis said that the EMA has granted a so-called "Prime" designation for iptacopan for this severe form of primary glomerulonephritis, which is commonly diagnosed in adolescents and young adults.

The disease has a poor prognosis, as about 50% of patient progress to end-stage renal disease within 10 years. It has a worldwide annual incidence of 1-2 per million.

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