Pharming: Positive EMA Opinion for Joenja, European Decision Pending

Targeting Hyperactivity in PI3K? Pathway

Joenja targets the hyperactivity of the PI3K? pathway responsible for APDS (Activated Phosphoinositide 3-Kinase Delta Syndrome), a rare primary immunodeficiency characterized by severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy. Leniolisib, the active ingredient in Joenja, is an oral inhibitor of PI3K? that regulates various cellular functions including proliferation, differentiation, and cytokine production. If approved in Europe, Joenja would become the first authorized treatment for APDS in the European Union. The centralized marketing authorization would be valid across all 27 EU member states, as well as in Norway, Iceland, and Liechtenstein.

Positive CHMP Opinion Based on Clinical Trial Results

The positive opinion from the CHMP is based on the results of a Phase II/III clinical trial, which was randomized, triple-blind, and placebo-controlled, involving 31 patients aged 12 years and older diagnosed with APDS. This trial demonstrated a statistically significant impact on immune dysregulation and immunodeficiency. Data from an open-label follow-up study involving 37 patients who received leniolisib for a median of three years were also submitted. Joenja is already approved and marketed in the United States, the United Kingdom, and Japan for patients aged 12 years and older with APDS. Leniolisib is also being evaluated in two Phase II clinical trials for other primary immunodeficiencies with immune dysregulation.