JOENJA (leniolisib)

JOENJA (leniolisib) 

Self-Administration – oral tablets

Diagnosis considered for coverage:

• 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.

Coverage Criteria:

For diagnosis of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome:

• Diagnosis of activated phosphoinositide 3-kinase delta syndrome (APDS); AND
• Molecular genetic testing confirms mutations in the PIK3CD or PIK3R1 gene; 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.); AND
• Prescribed by or in consultation with a hematologist or immunologist

Reauthorization Criteria:

For diagnosis of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome:

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

Dosing:

• 70 mg tablet taken orally, twice daily.
  • For patients weighing less than 45kg, there is no recommended dosage. 

Coverage Duration: 

• Initial: 6 months
• Reauthorization: 1 year

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information: 

• Precaution: JOENJA may cause fetal harm. Verify pregnancy status of patients of reproductive potential. Advise women of reproductive potential to use effective contraception during treatment and for 1 week after last dose.
• Currently available treatments are supportive and aimed at disease symptoms and complications. These treatments include prophylactic antibiotics, immune globulin replacement therapy, and immunosuppressive agents (e.g., systemic corticosteroids, off-label p110/mTOR inhibitors).  
• JOENJA is the first treatment approved specifically to treat the underlying pathogenesis of APDS. It was FDA-approved in March 2023. 

Policy Updates:

• 12/01/2023 – New policy for Joenja approved by WHA P&T Committee. (P&T, 11/14/2023)

References:

1. Jamee M, Moniri S, Zaki-Dizaji M, et al. Clinical, Immunological, and genetic features in patients with activated PI3Kδ syndrome (APDS): a systematic review. Clin Rev Allergy Immunol. 2020;59(3):323-333.
2. Joenja Prescribing Information. Pharming Technologies, Inc. Foster City, CA. March 2023. 
3. Per clinical consultation with allergist/immunologist specialist, May 10, 2023. 
4. 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.