EMA accelerates assessment of drug for rare disease APDS

The European Medicines Agency building

A potential new treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) has been validated for scientific evaluation under an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP). 

Pharming’s investigational drug leniolisib, a selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, will be evaluated as a therapy in adolescents and adults 12 years or older. 

The accelerated assessment reduces the review timeframe from 210 days to 150 days. The European Medicines Agency (EMA) will grant an accelerated assessment if they decide the product is of major interest for public health. 

Marketing authorisation for leniolisib in Europe is expected early next year. 

The application is supported by data from a Phase II/III study of leniolisib, which met its co-primary endpoints of reduction in lymph node size and increase in percentage of naïve B cells in patients with APDS. 

Anurag Relan, Chief Medical Officer of Pharming, commented: “We anticipate that leniolisib will fill an unmet need for patients with APDS, who currently rely on supportive therapies to treat their primary symptoms. This review constitutes a key milestone in Pharming’s effort to give healthcare providers and their patients global access to leniolisib.”  

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