Dutch biopharmaceutical company Pharming Group N.V. (PHAR) announced that the US Food and Drug Administration has accepted for priority review its New Drug Application or NDA for leniolisib to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome or APDS in adults and adolescents 12 years of age and older in the U.S.
The FDA has assigned a Prescription Drug User Fee Act or PDUFA goal date of March 29, 2023, aligned with a Priority Review classification.
leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor.
APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. It is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells.
Pharming submitted the NDA on July 29 based on positive data from a Phase II/III study of leniolisib, which met its co-primary endpoints of reduction in index lymph node size and correction of immunodeficiency in the target population.
The company noted that the trial results demonstrated the efficacy of leniolisib over placebo with a statistically significant reduction from the baseline size of participants’ index lymphadenopathy lesions and normalization of their immune function.
Anurag Relan, Chief Medical Officer of Pharming, said, “With FDA’s review, leniolisib moves further along the regulatory pathway as a potential disease-modifying targeted treatment for APDS in adults and adolescents 12 years of age and older in the US, who currently rely on supportive therapies such as antibiotics and immunoglobulin replacement therapy.”
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