FDA approves Pharming’s Joenja for children
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The US FDA has granted approval for Pharming’s Joenja (leniolisib) to treat children aged 4 to 11 with activated phosphoinositide 3-kinase delta syndrome (APDS).
The US Food and Drug Administration (FDA) has approved Dutch biopharma firm Pharming’s supplemental New Drug Application (sNDA) for Joenja (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
With this approval, Joenja becomes the first FDA-approved treatment for children aged 4 to 11 years with APDS in the USA. The newly approved Joenja doses are expected to be available to eligible pediatric patients in the USA in October through Pharming’s established specialty distribution network and patient-support infrastructure.
Joenja is a medicine used to treat activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents from 12 years of age and weighing 45 kg or more.
Activated PI3K delta syndrome (APDS) is characterized by a spectrum of clinical manifestations involving the immune system leading to increased susceptibility to infections (e.g., otitis media, sinusitis, bronchitis, and pneumonia), autoimmune/autoinflammatory manifestations including autoimmune cytopenias, gastrointestinal manifestations resembling Crohn-like colitis, intussusception, and lymphoproliferation (e.g., lymphadenopathy, hepatosplenomegaly, and nodular lymphoid hyperplasia), and an increased risk of developing B-cell lymphomas and other malignancies.
APDS is an autosomal dominant disorder. Approximately 80% of individuals diagnosed with APDS have an affected parent and 20% of individuals have the disorder as the result of a de novo PIK3CD gain-of-function variant (for APDS1) or de novo PIK3R1 loss-of-function variant (for APDS2). Once the PIK3CD or PIK3R1 pathogenic variants have been identified in an affected family member, carrier testing for at-risk relatives and prenatal/preimplantation genetic testing are possible.
APDS is a rare, inherited disease in which the immune system (the body’s natural defences) does not work properly, making patients more susceptible to bacterial and viral infections. The condition can also lead to autoimmune disorders and lymphoma (cancer of lymphocytes, a type of white blood cell).
APDS is rare, and Joenja was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 19 October 2020.
