Fabhalta®
Drug - Fabhalta® (iptacopan) [Novartis Pharmaceuticals Corporation]
January 2025
Therapeutic area - PNH Agents
Initial approval criteria
- Patient is ≥ 18 years of age; AND
- Patient has a diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) confirmed by detection of PNH clones of at least 10% by flow cytometry diagnostic testing; AND
- Demonstrate the presence of at least 2 different glycosylphosphatidylinositol (GPI) protein deficiencies (e.g., CD55, CD59, etc.) within at least 2 different cell lines (e.g., granulocytes, monocytes, erythrocytes); AND
- Patient's hemoglobin (Hb) is < 10 g/dL (lab tests required); AND
- If the patient has NOT received prior treatment with complement inhibitor therapy (e.g., pegcetacoplan, eculizumab, or ravulizumab-cwvz) BOTH of the following (lab tests required) must be met:
- Patient has RBC clone size ≥ 10%; AND
- Patient has a lactate dehydrogenase (LDH) level > 1.5 times the upper limit of normal (ULN); AND
- Patient will NOT use Fabhalta in combination with another complement inhibitor (e.g., pegcetacoplan, eculizumab, or ravulizumab); AND
- Patient does NOT have an unresolved serious infection and if infection by an unencapsulated bacteria occurs, the medication will be stopped; AND
- Patient does NOT have serious hypersensitivity to iptacopan or any of the excipients; AND
- Patient has complete or updated vaccination for encapsulated bacteria (based on ACIP recommendations for patients receiving a complement inhibitor) ≥ 2 weeks prior to the first dose of Fabhalta, unless the risks of delaying Fabhalta outweigh the risk of developing a serious infection; AND
- Patient is NOT on concomitant cytochrome P450 2C8 (CYP2C8) inhibitors (e.g., gemfibrozil); AND
- Patient does NOT have severe renal impairment (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2) with or without hemodialysis; AND
- Patient does NOT have severe hepatic impairment (Child-Pugh class C); AND
- Fabhalta has been prescribed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., hematologist); AND
- Prescriber will monitor total cholesterol, low-density lipoprotein-cholesterol (LDL-C), and serum triglycerides during Fabhalta treatment; AND
- Prescriber will monitor for signs of hemolysis for a minimum of 2 weeks after Fabhalta discontinuation; AND
- Prescriber must be registered with the Fabhalta REMS program.
- Initial approval is for 6 month
Renewal criteria
- Patient must continue to meet the above criteria; AND
- Patient has demonstrated improvement or stabilization of PNH from baseline (e.g., decreased requirement of RBC transfusions, Hb stabilization or improvement, LDH reduction, symptom improvement or stabilization, reduction in thrombotic events); AND
- Patient has not experienced treatment restricting adverse effects (e.g., encapsulated bacterial infection, uncontrolled hyperlipidemia).
- Renewal approval is for 12 months
Quantity limits
- 200 mg capsules: 68 capsules per 34 days
Questions?
Provider Call Center (844) 575-7887