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Rare Immunodeficiency Treatment Receives FDA Priority Review

The FDA granted a Priority Review to the leniolisib New Drug Application (NDA) indicated for individuals with activated phosphoinositide 3-kinase delta syndrome (APDS) 12 years and older.

Today, Pharming shared that the FDA granted priority review status for its oral selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, leniolisib. The drug candidate is indicated for adults and adolescents with activated phosphoinositide 3-kinase delta syndrome (APDS) 12 years and older.

“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," Anurag Relan, MD, MPH, Chief Medical Officer, Pharming, said in a statement. "We look forward to continuing to work closely with the FDA, as well as with regulatory authorities across the globe, to make leniolisib available to immunologists, hematologists, and their APDS patients.”

Positive phase 2/3 data supported the candidate's New Drug Application (NDA) by meeting the co-primary endpoints of the study. Leniolisib decreased APDS disease markers and was well-tolerated according to safety data.

Efficacy was exhibited by a significant reduction in index lymph node size (p=0.006), in addition to immumodeficiency correction among individuals with APDS. The normalization of the patients' immune systems was demonstrated by an elevation in the amount of naïve B cells from the baseline (p=0.002).

Activated PI3K delta syndrome causes lymphoproliferation, immune dysfunction and an increased risk of lymphoma in those living with the condition discovered less than a decade ago.

"The FDA’s acceptance for priority review of Pharming’s New Drug Application for leniolisib is a milestone that demonstrates our commitment to addressing unmet needs for patients with rare diseases," Relan stated.