Pharming gets US FDA Acceptance for Priority Review of its NDA for Leniolisib


Pharming Group N.V., announced that it has got acceptance from the US Food and Drug Administration (FDA) for priority review of Pharming’s New Drug Application (NDA) for leniolisib, an oral and selective phosphoinositide 3-kinase delta inhibitor. The inhibitor is used to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents above 12 years of age in the United States.

Pharming’s New Drug Application (NPA) was supported by positive data from a Phase 2 and Phase 3 study of leniosib that met its co-primary endpoints of reduction in index lymph node and correction of the immunodeficiency in the people.

Anurag Relan, chief medical officer of Pharming, said that this acceptance from the US FDA for priority review for Pharming’s NDA for leniolisib is a milestone that shows their commitment to addressing unmet needs for patients with rare diseases. With review from the US FDA, leniolisib moves further along the regulatory pathway as a potential disease-targeted treatment for APDS in adults and adolescents over 12 years old in the United States, who currently rely on the supportive therapies like immunoglobin replacement therapy and antibiotics.

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Anurag Relan added that they are looking forward to continuing to work closely with the US FDA, as well as with the regulatory authorities across the globe, to make the drug available to haematologists, immunologists, and APDS patients.