Joenja - NC Standard

Restricted Product(s)

• Joenja® (leniolisib)

FDA Approved Use

• For the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older 

Criteria for Approval of Restricted Product(s)

Initial Coverage for Approval

1. The patient is 12 years of age or older; AND 
2. The patient has genetically confirmed diagnosis of activated phosphoinositide 3- kinase delta syndrome (APDS) with presence of APDSassociated genetic PI3Kδ mutation documented in either PIK3CD or PIK3R1 variant (medical record documentation required); AND 
3. The patient has clinical manifestations of APDS prior to therapy with the requested agent (e.g., recurrent sinopulmonary infections, recurrent herpesvirus infections, lymphadenopathy, hepatomegaly, splenomegaly, nodular lymphoid hyperplasia, autoimmunity, cytopenia, enteropathy, bronchiectasis, organ dysfunction) (medical record documentation required); AND 
4. The patient will not be using the requested medication in combination with immunosuppressive therapy [other than corticosteroids or immunoglobulin G (IgG) therapy]; AND 
5. The prescribed dose and quantity are appropriate based on intended use and FDA labeling; AND 
6. The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g. immunologist); AND 
7. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies. 

Duration of Approval: 12 weeks

Continuation Criteria for Approval

1. The patient has been previously approved with Blue Cross and Blue Shield of North Carolina (Blue Cross NC) or would have met initial criteria for approval upon the start of therapy; AND 
2. The patient continues to be managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g immunologist); AND 
3. The patient exhibits signs of therapeutic benefit (i.e. improvement or stabilization in sinopulmonary infections, herpesvirus infections, lymphadenopathy, hepatomegaly, splenomegaly, nodular lymphoid hyperplasia, autoimmunity, cytopenia, enteropathy, bronchiectasis, organ dysfunction) from baseline (medical record documentation required). 

Duration of Approval: 365 days (1 year) 

Quantity Limit Exception Criteria

1. The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90-day titration period); AND 
2. The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND 
3. The quantity (dose) requested does not exceed the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert; OR 
4. If the quantity (dose) requested exceeds the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert, then the prescriber must submit documentation in support of therapy with a higher dose for the intended diagnosis (submitted documentation may include medical records OR fax form which reflects medical record documentation that shows the length of time the requested dose has been used, and what other medications and doses have been tried and failed). 

Duration of Approval: 365 days (1 year)

References

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information

Criteria and treatment protocols are reviewed annually by the Blue Cross NC P&T Committee, regardless of change. This policy is reviewed in Q2 annually.

August 2024: Criteria change: Removed lymphoproliferation and immunophenotype baseline assessment requirement and the measurement requirement of naïve B cells out of total B cells. Allow documentation of the general clinical findings of APDS for approval in the initial and continuation review sections.

April 2023: Original utilization management criteria issued.