Pharming announces first patient enrolled in pediatric clinical trial of leniolisib
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Tuesday, February 21, 2023
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LEIDEN, Netherlands, Feb. 21, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM)/(Nasdaq: PHAR) announces that the first patient has been enrolled in its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children with activated phosphoinositide 3-kinase delta syndrome (APDS). There is currently no approved treatment for this complex and progressive disease caused by genetic variants.
Key Points:
- Pharming plans to initiate a similar clinical trial in the third quarter of 2023 that will include children aged 1 to 6 years, with APDS, to evaluate a new pediatric formulation of leniolisib.
- Eligible patients enrolled in either of the pediatric trials will continue to receive leniolisib for a year after the initial 12-week treatment period through an open-label extension trial.
- Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
"I am pleased we have initiated the first trial in our Phase III pediatric clinical program evaluating leniolisib in children with APDS, and I look forward to our second pediatric trial getting underway. - Pharming expects that the CHMP will issue its opinion on the leniolisib MAA in 2H 2023.