FDA Accepts NDA for Therapy for Rare Immunodeficiency Disease


The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, for a decision on leniolisib as a treatment for APDS, a rare primary immunodeficiency.

The FDA has accepted for priority review Pharming’s new drug application (NDA) for leniolisib to treat patients 12 years and older with a rare primary immunodeficiency disease. The disease, activated phosphoinositide 3-kinase delta syndrome (APDS), affects 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. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023.

Leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor. Leniolisib inhibits the production of PIP3, which is an important cellular messenger and it regulates cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism.

The NDA was supported by positive data from a phase 2/3 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 findings indicate a reduction in disease markers associated with APDS, which include significant lymphoproliferation and immune dysfunction, as well as increased risk of lymphoma.

Leniolisib was well tolerated by participants. Also submitted as part of the application were data from a long-term, open-label extension clinical trial including 38 patients with APDS who were treated with leniolisib for a median of 102 weeks.

Additionally, the CDC has issued a new diagnosis code for APDS — D81.82 Activated Phosphoinositide 3-kinase Delta Syndrome (APDS) — which will be effective starting Oct. 1, 2022.

Anurag Relan, M.D.

Anurag Relan, M.D.

“By assigning this ICD-10-CM code, the CDC is formally recognizing APDS as a discrete immunological disease, and that will make a life-altering difference for people affected by the condition,” Anurag Relan, M.D., chief medical officer of Pharming, said in a press release. “By using the unique diagnostic code to identify both established and new patients with APDS, physicians will increase care options for affected individuals while helping to boost the world’s understanding of the prevalence, mechanisms, and outcomes of this progressively debilitating disease.

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