Joenja (leniolisib) — Medica

Initial criteria

• age ≥ 12 years
• patient weighs ≥ 45 kg
• patient has a genetic phosphoinositide 3-kinase delta (PI3Kδ) pathogenic variant in the PIK3CD and/or PIK3R1 genes
• patient has at least one clinical finding or manifestation consistent with APDS (e.g., recurrent sinopulmonary infections, recurrent herpesvirus infections, lymphadenopathy, hepatomegaly, splenomegaly, nodular lymphoid hyperplasia, autoimmunity, cytopenias, enteropathy, bronchiectasis, or organ dysfunction)
• Joenja is prescribed by or in consultation with an immunologist or a physician who treats patients with primary immune deficiencies

Reauthorization criteria

• patient has been established on therapy for at least 6 months
• age ≥ 12 years
• patient weighs ≥ 45 kg
• patient has a genetic phosphoinositide 3-kinase delta (PI3Kδ) pathogenic variant in the PIK3CD and/or PIK3R1 genes
• patient has had a positive clinical response in the signs and manifestations of APDS (e.g., reduction of lymph node size, spleen size, immunoglobulin replacement therapy use, infection rate, or immunoglobulin M (IgM) levels)

Approval duration

initial: 6 months; reauthorization: 1 year