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Fabhalta (iptacopan) Prior Authorization with Quantity Limit Program Summary
Policy Number: PH-1218
This program applies to Blue Partner, Commercial, GenPlus, NetResults A series, SourceRx and Health Insurance Marketplace formularies.
POLICY REVIEW CYCLE
Effective Date |
Date of Origin |
07-01-2024 |
07-01-2024 |
FDA LABELED INDICATIONS AND DOSAGE
Agent(s) |
FDA Indication(s) |
Notes |
Ref# |
Fabhalta® (iptacopan) Capsule |
Treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) |
|
1 |
See package insert for FDA prescribing information: https://dailymed.nlm.nih.gov/dailymed/index.cfm
CLINICAL RATIONALE
Paroxysmal Nocturnal Hemoglobinuria |
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, life-threatening, rare, multi-systemic disease developing as a result of somatic mutation of hematopoietic stem cells, and characterized by clonal, complement-mediated intravascular hemolysis. PNH is mainly a disease of adults with a median age of onset in the thirties. High Precision Flow Cytometry is the most useful and accepted diagnostic test to confirm the diagnosis of PNH. Flow cytometry is performed by incubating the patient’s peripheral blood cells with fluorescently-labeled monoclonal antibodies that bind to glycosylphosphatidylinositol (GPI) anchored proteins, which are reduced or absent on blood cells in PNH. Since different blood cell lineages display different combinations of GPI-linked proteins, and some proteins bind to cell surfaces via both GPI-linked and GPI-independent mechanisms, it is recommended that at least two independent flow cytometry reagents be used on at least two cell lineages (e.g., RBCs and WBCs) to establish the diagnosis of PNH.(2) Lack of the complement inhibitor CD59 on the red blood cells surface is mostly responsible for the clinical manifestations in PNH. These patients manifest with chronic intravascular hemolysis, paroxysmal flares of hemolysis and a propensity for thrombosis. Intravascular hemolysis leads to release of free hemoglobin (Hb) into the blood. Free hemoglobin, in turn, can cause various toxic effects, including hypercoagulability, changes in vascular tone from reduction of circulating nitric oxide and renal damage.(3) Extravascular hemolysis also occurs in patients with PNH because C3 fragments that are not destroyed by the membrane attack complex (MAC) intravascularly can accumulate on the GPI-negative red blood cell (lacking CD55) surface and these fragments opsonize the red blood cells, causing reticuloendothelial destruction in the liver and spleen.(3) The main clinical situations or diseases that should be considered in the differential diagnosis of PNH are:(3)
PNH is classified into three different categories:(3)
Patients with PNH have a median survival of ten years after diagnosis. The approach to therapy depends on the severity of symptoms and the degree of hemolysis. The treatment options for PNH are supportive care, allogenic hematopoietic stem cell transplantation (HCT) and a complement blockade.(2,3) |
Efficacy |
Iptacopan binds to Factor B of the alternative complement pathway and regulates the cleavage of C3, generation of downstream effectors, and the amplification of the terminal pathway. In PNH, intravascular hemolysis is mediated by the downstream membrane attack complex, while extravascular hemolysis is facilitated by C3b opsonization. Iptacopan acts proximally in the alternative pathway of the complement cascade to control both C3b-mediated EVH and terminal complement mediated IVH.(1) The efficacy of Fabhalta in adults with PNH was evaluated in a multi-center, open-label, 24-week active comparator-controlled trial (APPLY-PNH; NCT04558918). The study enrolled adults with PNH and residual anemia (hemoglobin < 10 g/dL) despite previous treatment with a stable regimen of anti-C5 treatment (either eculizumab or ravulizumab) for at least 6 months prior to randomization. Efficacy primary endpoints were established based on demonstration of superiority of switching to Fabhalta compared to continuing on anti-C5 therapy in achieving hematological response after 24 weeks of treatment, without a need for transfusion, by assessing the proportion of patients demonstrating:(1)
Secondary endpoints included:(1)
Patients with sustained increase of hemoglobin levels greater than or equal to 2 g/dL in the Fabhalta arm had an 82.3% response rate (95% CI) and the response rate in the Anti-C5 arm was 0%. Patients with sustained hemoglobin level greater than or equal to 12 g/dL in the absence of tranfusions in the Fabhalta arm had a 67.7% response rate (95% CI) and the response rate in the Anti-C5 arm was 0%.(1) Fabhalta was studied in a single arm study in adults with PNH who were not previously treated with a complement inhibitor (APPOINT-PNH; NCT04820530). Adult patients with PNH (RBC clone size greater than or equal to 10%), hemoglobin less than 10 g/dL, and LDH greater than 1.5 times the upper limit of normal received Fabhalta during the 24-week open-label core treatment period. In total, 77.5% (95% CI: 61.5%, 89.2%) of patients achieved a sustained increase (between Day 126 and Day 168) in hemoglobin levels from baseline of greater than or equal to 2 g/dL in the absence of RBC transfusions.(1) |
Safety |
Fabhalta has a boxed warning about the increased risk of serious and life-threatening infections, caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B:(1)
Fabhalta is contraindicated in:(1)
Fabhalta is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called FABHALTA REMS.(1) |
REFERENCES
Number |
Reference |
1 |
Fabhalta prescribing information. Novartis Pharmaceuticals Corporation. December 2023. |
2 |
Sahin Fahri, Meltem Akay O, Ayer M, et al. Pesg PNH diagnosis, follow-up and treatment guidelines. Am J Blood Res. 2016; 6(2): 19-27. |
3 |
Rodolfo D. Cançado, Aderson da Silva Araújo, Alex Freire Sandes, Celso Arrais, Clarisse Lopes de Castro Lobo, Maria Stella Figueiredo, Sandra Fátima Menosi Gualandro, Sara Teresinha Olalla Saad, Fernando Ferreira Costa. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematology, Transfusion and Cell Therap. 2020. |
POLICY AGENT SUMMARY PRIOR AUTHORIZATION
Target Brand Agent(s) |
Target Generic Agent(s) |
Strength |
Targeted MSC |
Available MSC |
Final Age Limit |
Preferred Status |
|
||||||
Fabhalta |
iptacopan hcl cap |
200 MG |
M ; N ; O ; Y |
N |
|
|
POLICY AGENT SUMMARY QUANTITY LIMIT
Target Brand Agent Name(s) |
Target Generic Agent Name(s) |
Strength |
QL Amount |
Dose Form |
Day Supply |
Duration |
Addtl QL Info |
Allowed Exceptions |
Targeted NDCs When Exclusions Exist |
|
|||||||||
Fabhalta |
iptacopan 200 mg capsules |
200 MG |
60 |
Capsules |
30 |
DAYS |
|
|
|
CLIENT SUMMARY – PRIOR AUTHORIZATION
Target Brand Agent Name(s) |
Target Generic Agent Name(s) |
Strength |
Client Formulary |
Fabhalta |
iptacopan hcl cap |
200 MG |
Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx |
CLIENT SUMMARY – QUANTITY LIMITS
Target Brand Agent Name(s) |
Target Generic Agent Name(s) |
Strength |
Client Formulary |
Fabhalta |
iptacopan 200 mg capsules |
200 MG |
Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx |
PRIOR AUTHORIZATION CLINICAL CRITERIA FOR APPROVAL
Module |
Clinical Criteria for Approval |
|
Initial Evaluation Target Agent(s) will be approved when ALL of the following are met:
Length of Approval: 6 months NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria. *Step therapy requirement may not apply if a prior health plan paid for the medication - documentation of a paid claim may be required.
Renewal Evaluation Target Agent(s) will be approved when ALL of the following are met:
Length of Approval: 12 months NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria |
QUANTITY LIMIT CLINICAL CRITERIA FOR APPROVAL
Module |
Clinical Criteria for Approval |
|
Quantity limit for the Target Agent(s) will be approved when ONE of the following is met:
Length of Approval: Initial approval up to 6 months Renewal approval up to 12 months |
This pharmacy policy is not an authorization, certification, explanation of benefits or a contract. Eligibility and benefits are determined on a case-by-case basis according to the terms of the member’s plan in effect as of the date services are rendered. All pharmacy policies are based on (i) information in FDA approved package inserts (and black box warning, alerts, or other information disseminated by the FDA as applicable); (ii) research of current medical and pharmacy literature; and/or (iii) review of common medical practices in the treatment and diagnosis of disease as of the date hereof. Physicians and other providers are solely responsible for all aspects of medical care and treatment, including the type, quality, and levels of care and treatment.
The purpose of Blue Cross and Blue Shield of Alabama’s pharmacy policies are to provide a guide to coverage. Pharmacy policies are not intended to dictate to physicians how to practice medicine. Physicians should exercise their medical judgment in providing the care they feel is most appropriate for their patients.
Neither this policy, nor the successful adjudication of a pharmacy claim, is guarantee of payment.
ALBP _ Commercial _ CSReg _ Fabhalta_iptacopan__PAQL _ProgSum_ 07-01-2024 _ © Copyright Prime Therapeutics LLC. May 2024 All Rights Reserved1