PHGUF - Pharming gets EMA accelerated review of leniolisib for rare immunodeficiency disorder
- The European Medicines Agency (EMA) validated Pharming's ( NASDAQ: PHAR ) application seeking approval of leniolisib to treat patients aged 12 years and older with activated phosphoinositide 3-kinase (PI3K) delta syndrome (APDS).
- The company's marketing authorisation application (MAA) for leniolisib was validated for evaluation under an accelerated assessment by the EMA's Committee for Medicinal Products for Human Use (CHMP).
- In August the leniolisib MAA was granted accelerated assessment by EMA's CHMP.
- The MAA is backed by data from a phase 2/3 trial which met its main goals.
- "EMA's validation for review of our MAA under an accelerated assessment pathway highlights Pharming's ongoing commitment to advance leniolisib as a targeted treatment for adults and adolescents 12 years of age or older with APDS," said Pharming Chief Medical Officer Anurag Relan.
- APDS is a rare immunodeficiency disease that affects 1 to 2 people per million and is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells, according to the company.
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Pharming gets EMA accelerated review of leniolisib for rare immunodeficiency disorder