Drugs

Complete Response on Pharming’s Pediatric sNDA for Joenja

FDA has issued Pharming Group a complete response letter for its supplemental NDA (sNDA) seeking expanded approval of Joenja (leniolisib) for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). The drug is currently approved for treating APDS in patients aged 12 years and older.

The FDA letter cited concerns about potential underexposure in lower-weight pediatric patients and requested additional pharmacokinetic data to reassess the proposed pediatric dosing regimen, according to the company. The agency reportedly wants confirmation that children in lower-weight dose groups can achieve drug exposure levels comparable to those seen in adolescents and adults currently approved for the therapy.

In addition to the clinical pharmacology issue, FDA identified a problem with one of the analytical methods used for production batch testing and requested further data and clarification, the company says.

Pharming say it plans to work with FDA to determine next steps and intends to request a Type A meeting with the agency to discuss a potential resubmission. “While we are disappointed in the FDA’s response, we remain dedicated to making Joenja available to pediatric patients aged 4–11 with APDS,” it adds.

The sNDA was based on data from an open-label, multinational Phase 3 study in children aged 4 to 11 years with APDS, Pharming says. The trial showed improvements over 12 weeks in two key disease hallmarks — reduced lymphadenopathy and increased naïve B cells — indicating correction of the underlying immune defect, it says.. Safety data from up to eight months of treatment were also included in the submission.

Pharming reports that improvements were observed across all four dose levels studied and were consistent with results previously seen in adolescent and adult patients. All treatment-emergent adverse events were mild to moderate, with no drug-related serious adverse events reported and no discontinuations during the 12-week treatment period.

APDS is described as a rare primary immunodeficiency caused by genetic variants affecting the PI3Kδ pathway, leading to immune dysfunction and recurrent infections, lymphoproliferation, autoimmunity, and other complications.

Joenja is an oral selective phosphoinositide 3 kinase delta inhibitor. It inhibits the “production of phosphatidylinositol-3-4-5- trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism,” according to the company.

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