WHA
 

Joenja (leniolisib) - PA, NF

Indications for Prior Authorization

Joenja (leniolisib)
  • For diagnosis of Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome
    Indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

Criteria

Joenja

Prior Authorization (Initial Authorization)

Length of Approval: 6 Month(s) [A]

  • Diagnosis of activated phosphoinositide 3-kinase delta syndrome (APDS)
    • AND
  • Disease is confirmed by the presence of mutations in the PIK3CD or PIK3R1 gene as detected by a FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
    • AND
  • Patient is 12 years of age or older
    • AND
  • Patient weighs greater than or equal to 45kg
    • AND
  • Both of the following:
    • Presence of nodal and/or extranodal proliferation (e.g., lymphadenopathy, splenomegaly, hepatomegaly)
    • Presence of other clinical findings and manifestations consistent with APDS (e.g., recurrent sino-pulmonary infections, bronchiectasis, enteropathy)
    AND
  • Trial and failure, contraindication, or intolerance to at least one standard of care treatment for APDS (e.g., Immunoglobulin replacement therapy, antimicrobial prophylaxis [e.g., azithromycin, bactrim], rituximab, tacrolimus, etc.) [3]
    • AND
  • Prescribed by or in consultation with one of the following:
    • Hematologist
    • Immunologist
    • Geneticist
Joenja

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

  • Patient demonstrates positive clinical response to therapy (e.g., reduced lymph node size, increased naïve B-cell percentage, decreased severity or frequency of infections/hospitalizations)
Joenja

Non Formulary

Length of Approval: 6 Month(s) [A]

  • Submission of medical records (e.g., chart notes) confirming diagnosis of activated phosphoinositide 3-kinase delta syndrome (APDS)
    • AND
  • Both of the following:
    • Submission of medical records (e.g., chart notes) confirming disease by the presence of mutations in the PIK3CD or PIK3R1 gene
      • AND
    • Provider attests that mutation was detected by a FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
    AND
  • Patient is 12 years of age or older
    • AND
  • Submission of medical records (e.g., chart notes) confirming patient weighs greater than or equal to 45kg
    • AND
  • Submission of medical records (e.g., chart notes) confirming both of the following:
    • Presence of nodal and/or extranodal proliferation (e.g., lymphadenopathy, splenomegaly, hepatomegaly)
    • Presence of other clinical findings and manifestations consistent with APDS (e.g., recurrent sino-pulmonary infections, bronchiectasis, enteropathy)
    AND
  • Submission of medical records (e.g., chart notes) or paid claims confirming trial and failure, contraindication, or intolerance to at least one standard of care treatment for APDS (e.g., Immunoglobulin replacement therapy, antimicrobial prophylaxis [e.g., azithromycin, bactrim], rituximab, tacrolimus, etc.) [3]
    • AND
  • Prescribed by or in consultation with one of the following:
    • Hematologist
    • Immunologist
    • Geneticist

  • Guideline ID
    GL-683390

  • Formulary
    Premium

  • Effective date
    Sep 1, 2026

  • P&T approval date
    Jun 21, 2023

P & T Revisions

2026-08-06, 2026-04-29, 2025-12-18, 2025-05-02, 2024-05-01, 2023-11-02, 2023-05-26

  1. Joenja Prescribing Information. Pharming Technologies, Inc. Foster City, CA. June 2025.
  2. Per clinical consultation with allergist/immunologist specialist, May 10, 2023.
  3. Rao VK, Webster S, Šedivá A, et al. Randomized, Placebo-Controlled, Phase 3 Trial of PI3Kδ Inhibitor Leniolisib for Activated PI3Kδ Syndrome. Blood. Published online November 18, 2022.
  1. Per consult with specialist, extending initial authorization to 6 months will give providers more time to assess efficacy and see those surrogate outcomes such as reduced lymph node size. [2]
  • 2026-08-06: Drug specific non-formulary criteria created.
  • 2026-04-29: 2026 Annual Review - updated gene mutation language and references
  • 2025-12-18: no criteria changes, added IL statute operational note
  • 2025-05-02: 2025 Annual Review - no changes
  • 2024-05-01: 2024 Annual Review - Update to add geneticist as an additional specialist option
  • 2023-11-02: Program update to standard reauthorization language. No changes to clinical intent.
  • 2023-05-26: New Program