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CurrentExcellus Health Plan IncPolicy PHARMACY-98

Rare Diseases Clinical Review Prior Authorization (CRPA) Drug Policy

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This policy governs coverage and prior authorization criteria for specified rare-disease drugs under applicable Excellus Health Plan lines of business. It affects providers requesting coverage for formulary drugs and does not apply when the member’s subscriber contract excludes the drug or service; non-formulary drugs are directed to the Non-Formulary Medication Exception Review Policy.

Policy Summary

PayerExcellus Health Plan Inc

PolicyRare Diseases Clinical Review Prior Authorization (CRPA) Drug Policy

Policy CodePolicy PHARMACY-98

Change TypeNo material change

Effective DateJanuary 15, 2021

Last ReviewNov 10, 2026

Next Review DateN/A

Key ActionSubmit prior authorization requests with drug-specific diagnosis, age, prescriber, clinical history, laboratory or diagnostic evidence, and treatment-response documentation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

<200,000Rare disease definition

10Covered therapies in this window

6 monthsTypical initial approval period

12 monthsTypical recertification period

EpysqliPreferred eculizumab formulation

14 daysTzield treatment course

Drug-Specific Coverage Criteria

Drug-Specific Covered Indications, Exclusions, and Medical Necessity Criteria

Coverage is available only when the applicable drug- and indication-specific medical-necessity requirements below are satisfied. The requested indication, specialist involvement, diagnostic confirmation, age or weight criteria, prior-treatment requirements, laboratory or imaging findings, dosing, and any required response must be documented.

Covered rare-disease drug indication: Actimmune: chronic granulomatous disease confirmed by neutrophil-function testing with appropriate specialist prescribing and antibiotic/antifungal therapy, or severe malignant osteopetrosis confirmed radiologically by an appropriate specialist; dose is 50 mcg/m2 subcutaneously three times weekly and doses above 50 mcg/m2 are not authorized. Adzynma: hematologist involvement and congenital TTP confirmed by ADAMTS13 activity below 10% plus pathogenic homozygous or compound-heterozygous ADAMTS13 findings; new prophylactic starts require a prior TTP event or current plasma-based prophylaxis. Aldurazyme: Hurler, Hurler-Scheie, or Scheie MPS I confirmed by alpha-L-iduronidase enzyme analysis, metabolic specialist follow-up, qualifying family history or symptoms, and age at least 6 months.Adzynma ADAMTS13 <10%; Aldurazyme age ≥6 months

Use FDA-labeled dosing and provide current weight and regimen where required.

Hematologic and inflammatory indications:

Policy Summary

PayerExcellus Health Plan Inc

PolicyRare Diseases Clinical Review Prior Authorization (CRPA) Drug Policy

Policy CodePolicy PHARMACY-98

Change TypeNo material change

Effective DateJanuary 15, 2021

Last ReviewNov 10, 2026

Next Review DateN/A

Key ActionSubmit prior authorization requests with drug-specific diagnosis, age, prescriber, clinical history, laboratory or diagnostic evidence, and treatment-response documentation.

SourceLink

On This Page

Aqvesme: adults with laboratory-confirmed alpha- or beta-thalassemia, hemoglobin at or below 10 g/dL, and either non-transfusion-dependent status (no more than 5 RBC units in 24 weeks and no transfusion in 8 weeks) or transfusion-dependent status (6–20 units in 24 weeks and no transfusion-free interval longer than 6 weeks); transfusion-dependent beta-thalassemia new starts generally require Reblozyl failure or serious adverse effects unless prior splenectomy. Arcalyst: qualifying CAPS, DIRA with IL1RN mutation and Kineret benefit, or recurrent pericarditis with qualifying recurrence, inflammation, specialist involvement, and planned taper of anti-inflammatory therapy. Ilaris: CAPS, active sJIA, AOSD, periodic fever syndrome, or acute gout under the applicable age, specialist, prior-treatment, dose, and safety criteria. Gamifant: primary HLH with genetic/family-history confirmation or at least 5 diagnostic criteria plus refractory, recurrent, progressive disease or severe intolerance to conventional therapy; or Still-disease-associated HLH/MAS meeting active MAS criteria and steroid-failure, intolerance, contraindication, or recurrent-MAS criteria.

Metabolic, hepatic, endocrine, and genetic indications: Bylvay: molecularly confirmed PFIC types 1–6 or ALGS with age, specialist, cholestasis, bile-acid, pruritus, and prior-treatment requirements. Camzyos or Myqorzo: adults with symptomatic obstructive HCM confirmed by imaging, qualifying wall thickness and LVOT gradient, NYHA II/III symptoms, LVEF at least 55%, conventional cardiac-therapy trials or exceptions, and REMS compliance. Crenessity: classic CAH from confirmed 21-hydroxylase deficiency in a patient at least 4 years old receiving supraphysiologic glucocorticoids. Imcivree: acquired hypothalamic obesity, qualifying POMC/PCSK1/LEPR-related obesity, or BBS-related obesity with the applicable anthropometric, genetic, injury, or feature criteria. Increlex: severe primary IGF-1 deficiency or GH-gene deletion with neutralizing GH antibodies. Isturisa or Recorlev: adults with symptomatic endogenous Cushing syndrome, elevated UFC, failed or unsuitable surgery, and required medication failures. Joenja: APDS with qualifying PIK3CD or PIK3R1 variant and clinical or imaging manifestations. Juxtapid or Evkeeza: HoFH with qualifying genetic or clinical evidence, persistent LDL-C elevation, and age-appropriate lipid-lowering treatment failure. Korlym: adults with endogenous Cushing syndrome plus type 2 diabetes or glucose intolerance after failed or unsuitable surgery.Camzyos/Myqorzo LVEF ≥55%; Evkeeza LDL-C >70 mg/dL with ASCVD or >100 mg/dL without ASCVD; Isturisa/Recorlev UFC ≥1.5×ULN

HeFH, nonclassic CAH, closed epiphyses for Increlex, and other excluded diagnoses are not covered.

Renal, complement, and immune indications: Empaveli, Fabhalta, Filspari, Tarpeyo, Trutakna, Vanrafia, or Voyxact may be covered for the applicable PNH, C3G, IC-MPGN, IgAN, or FSGS pathway when biopsy or flow-cytometry confirmation, nephrology or hematology involvement, ACE inhibitor/ARB therapy or documented exception, required prior-treatment failure, proteinuria, and eGFR criteria are met. Enjaymo: adults over 39 kg with laboratory-confirmed primary CAD, chronic hemolysis, qualifying titer and hemoglobin, recent symptoms, and rituximab failure or urgent-treatment exception. Revcovi: ADA-SCID with abnormal ADA testing, elevated erythrocyte dATP or dAdo nucleotides, inability to undergo or failure of HCT, and no severe thrombocytopenia. Rethymic: congenital athymia in a patient under 18 with low T-cell counts and qualifying FOXN1 or Complete DiGeorge findings plus surgical, HLA, infection-control, and prophylaxis safeguards. Ryplazim: plasminogen deficiency type 1 with baseline plasminogen activity below 45% and compatible lesions.Renal pathways generally require eGFR >30 or ≥30 mL/min/1.73 m2 and specified proteinuria; Revcovi platelets ≥50,000/microliter; Ryplazim plasminogen <45%

Ongoing duplicate complement inhibition is limited to defined transitions and is otherwise not authorized.

Neurologic, ophthalmic, wound, and other specialty indications: Daybue: classic or typical Rett syndrome with MECP2 mutation, age at least 2 years, and weight above 9 kg. Firdapse: LEMS confirmed by electromyography or calcium-channel antibody testing in patients at least 6 years old. Hyftor: tuberous-sclerosis-associated facial angiofibroma with at least 3 red papules at least 2 mm each. Imaavy, Rystiggo, eculizumab, Ultomiris, Uplizna, or Vyvgart/Vyvgart Hytrulo: antibody-positive gMG with qualifying MGFA class, baseline MG-ADL, specialist involvement, and immunosuppressive-treatment failure or rescue-therapy history. Enspryng, Uplizna, or eculizumab/Ultomiris: NMOSD with positive AQP4 testing and a qualifying relapse. Filsuvez or Vyjuvek: genetically confirmed DEB/JEB with documented qualifying wounds. Luxturna: biallelic RPE65 retinal dystrophy with viable retinal cells and certified ophthalmic administration. Scenesse: adult EPP with biochemical or genetic confirmation and phototoxicity. Skyclarys: genetically confirmed Friedreich ataxia in patients at least 16 years old. Skysona: eligible male patients age 4–17 with early active CALD and all genetic, laboratory, infection, conditioning, and transplant safeguards. Sohonos: FOP with ACVR1 R206H mutation and radiographic HO. Tepezza or Lumvoa: moderate-to-severe TED with qualifying thyroid status. Tzield: Stage 2 type 1 diabetes with at least two qualifying islet autoantibodies and dysglycemia without overt hyperglycemia. Vijoice: severe or life-threatening PROS with PIK3CA mutation and measurable target lesion. Voxzogo: genetically confirmed achondroplasia with open epiphyses or AGV above 1.5 cm/year. Vykat XR: genetically confirmed PWS with hyperphagia and current weight.Tzield age ≥1; gMG MG-ADL generally ≥5 or ≥6 by product; Tepezza/Lumvoa lid retraction >2 mm or proptosis >3 mm above normal; Voxzogo AGV >1.5 cm/year

Product-specific age, dosing, combination, administration, and renewal requirements apply.

Exclusions and non-covered uses: Non-FDA-approved indications are excluded. Additional product-specific exclusions include: Adzynma for immune-mediated or acquired TTP; Aqvesme for sickle hemoglobin or hemoglobin C diagnoses; Bylvay or Livmarli for specified nonfunctional BSEP variants, cirrhosis, portal hypertension, or hepatic decompensation; Camzyos for mimicking infiltrative/storage disorders and continued therapy with LVEF below 50%, symptomatic heart failure, worsening status, or prohibited CYP therapy; Filspari for nephrotic-syndrome FSGS or use with RAAS inhibitors, ERAs, or aliskiren; Recorlev for pituitary/adrenal carcinoma or fungal infection; Scenesse for listed skin cancers, melanoma history, non-EPP photodermatoses, or dosing more often than every 2 months; Rethymic retreatment or treatment of SCID; Skysona retreatment or prior HSCT/gene therapy; current intubation for gMG; STEC-HUS for aHUS; prohibited combinations among complement inhibitors or gMG biologics; Tepezza, Lumvoa, or Tzield retreatment as specified; and wound-specific combination use of Filsuvez, Vyjuvek, or Zevaskyn without documented justification.

Subscriber-contract exclusions and non-formulary status are handled under the applicable contract or exception policy.

Initial Therapy Criteria

Initial authorization requires all core criteria for the requested product and indication. Where an indication has alternatives, at least one complete pathway must be met; nested prior-treatment and diagnostic alternatives are shown below.

Initial hematologic, enzyme, hepatic, and inflammatory therapies: Actimmune requires the applicable CGD or severe malignant osteopetrosis diagnosis, specialist, diagnostic confirmation, and approved dose. Adzynma requires hematologist involvement, congenital TTP with ADAMTS13 activity below 10% and pathogenic molecular confirmation, plus a qualifying prior TTP event or plasma prophylaxis for new prophylactic use. Aldurazyme requires confirmed MPS I, metabolic specialist care, qualifying family history or symptoms, age at least 6 months, weight, and regimen. Aqvesme requires one of the non-transfusion-dependent or transfusion-dependent thalassemia pathways. Arcalyst requires one of CAPS, DIRA, or recurrent-pericarditis pathways. Bylvay requires molecularly confirmed PFIC or ALGS with disease activity and prior conventional therapy. Crenessity requires classic CAH with 21-hydroxylase deficiency, glucocorticoid co-therapy, and baseline glucocorticoid and androstenedione values. Penicillamine requires an FDA-approved indication; cystinuria additionally requires specialist diagnosis and failure of conservative measures. Daybue requires specialist prescribing, age at least 2, weight above 9 kg, MECP2 mutation, and classic or typical Rett syndrome.See indication-specific thresholds in the policy

Initial approval periods and dosing are product-specific.

Initial renal, lipid, complement, and immune therapies: Empaveli, Fabhalta, Filspari, Tarpeyo, Trutakna, Vanrafia, and Voyxact require their respective biopsy-confirmed renal diagnosis, eGFR and proteinuria thresholds, ACEi/ARB treatment or exception, and required SGLT2, glucocorticoid, immunosuppressive, or comparator failure pathway. Fabhalta IgAN requires Filspari or Tarpeyo failure; Vanrafia requires Filspari or Tarpeyo failure; Fabhalta C3G and Empaveli renal indications require steroid or mycophenolate failure or exception. Evkeeza and Juxtapid require HoFH, LDL-C above threshold, and age-appropriate lipid-lowering failure. Enjaymo requires primary CAD criteria, qualifying hemolysis and symptoms, and rituximab failure or immediate-response exception. Revcovi requires ADA-SCID, abnormal ADA-related laboratory findings, HCT unsuitability or failure, and platelet count at least 50,000/microliter. Ryplazim requires type 1 plasminogen deficiency, baseline activity below 45%, and compatible lesions. Rethymic requires congenital athymia, low T-cell count, qualifying genetic or DiGeorge pathway, and administration safeguards.Renal eGFR generally >30 or ≥30 mL/min/1.73 m2; IgAN proteinuria >0.5 g/day or UPCR >0.44 g/g

Use the same laboratory parameter at recertification when the policy requires it.

Initial neurologic, ophthalmic, and specialty therapies: Imaavy, Rystiggo, eculizumab, Ultomiris, Uplizna, Zilbrysq, or Vyvgart/Vyvgart Hytrulo require antibody-positive gMG with qualifying MGFA class, baseline MG-ADL, specialist involvement, and a qualifying immunosuppressive failure or rescue-therapy pathway; new starts for applicable products require Vyvgart Hytrulo PFS failure. Enspryng, Uplizna, Ultomiris, or eculizumab for NMOSD require positive AQP4 testing, specialist prescribing, and a qualifying relapse requiring rescue therapy. Gamifant requires primary HLH or Still-disease-associated MAS criteria, specialist prescribing, active disease, and required conventional therapy failure or steroid pathway. Filsuvez and Vyjuvek require genetic EB confirmation and wound documentation. Luxturna requires biallelic RPE65 disease, viable retina, baseline FST, and certified ophthalmic administration. Skysona requires all eligibility, laboratory, infection, conditioning, donor, vaccination, transplant, and one-time-administration safeguards. Tzield requires Stage 2 type 1 diabetes with at least two autoantibodies and qualifying OGTT dysglycemia. Tepezza, Lumvoa, Scenesse, Skyclarys, Sohonos, Vijoice, Voxzogo, Vykat XR, and Tryngolza require their respective specialist, disease-confirmation, severity, laboratory, genetic, or anthropometric pathways.gMG MG-ADL ≥5 or ≥6 by product; Tzield fasting glucose >110 and <126 mg/dL, 2-hour OGTT >140 and <200 mg/dL, or timed OGTT >200 mg/dL

Product-specific exclusions, dosing, benefit routing, and site-of-care rules remain applicable.

Required nested alternatives: For qualifying pathways that allow alternatives, satisfy one complete route: primary HLH may be established genetically, by compatible family history, or by at least 5 diagnostic criteria; gMG may qualify through corticosteroid failure, non-steroidal immunosuppressant failure, or recent rescue therapy; IgAN renal products may qualify through SGLT2 failure, systemic glucocorticoid failure, or the specified high-risk proteinuria/eGFR route; FCS may qualify through biallelic pathogenic variants, FCS score, NAFCS score, or the complete clinical pathway; POMC/PCSK1/LEPR or BBS obesity requires the applicable genetic or syndromic pathway; and Tryngolza may qualify through FCS or severe hypertriglyceridemia criteria.FCS score ≥10; NAFCS score ≥45; primary HLH ≥5 diagnostic criteria

Each alternative must be supported by the documentation specified for that pathway.

Continuation and Recertification Criteria

Continuation and recertification require documented tolerance and ongoing clinical benefit. Unless a drug-specific period is stated, the request must also continue to meet current dosing, formulary, and safety requirements.

Hematologic, hepatic, inflammatory, and enzyme-replacement response: Adzynma requires response and discontinuation or non-routine use of plasma prophylaxis. Aqvesme requires a hemoglobin increase of at least 1 g/dL for non-transfusion-dependent disease or reduced RBC transfusion burden for transfusion-dependent disease. Arcalyst recurrent pericarditis requires no recurrence and reduced or discontinued NSAID, colchicine, or corticosteroid therapy. Bylvay and Livmarli require tolerance plus reduced pruritus and/or bile acids. Camzyos and Myqorzo require positive clinical response and repeat echocardiographic LVEF monitoring. Crenessity requires reduced glucocorticoid dose or androstenedione. Penicillamine or tiopronin requires urinary cystine below 250 mg/L or decreased stone formation. Daybue requires tolerance and ongoing improvement or stability. Gamifant requires complete, partial, or improved HLH response, or for Still-disease-associated MAS, improvement in MAS signs and symptoms plus at least one objective laboratory response. Hyftor requires improvement or maintained response in facial angiofibromas.Aqvesme Hb increase ≥1 g/dL; Gamifant ferritin decrease ≥80% or <2,000 ng/mL; Myqorzo continued therapy not approved with LVEF <40%

Response must be documented at the applicable review interval.

Renal, lipid, complement, and hematologic response: Empaveli, Fabhalta, Filspari, Trutakna, Vanrafia, and Voyxact require reduction in the specified urine protein or UPCR measure with preserved eGFR. Filspari FSGS requires improved UPCR and continued absence of nephrotic syndrome. Evkeeza and Juxtapid require LDL-C reduction from baseline. Enjaymo requires hemoglobin normalization above 12 g/dL or increase above 2 g/dL, with no RBC transfusion after week 5, subject to the policy's clinical-benefit exception. Empaveli, Fabhalta, Piasky, Voydeya, Ultomiris, or eculizumab for PNH require tolerance and response shown by fewer transfusions, improved hemoglobin, normalized LDH, or symptom improvement. Revcovi requires improved immune or clinical status or normalized ADA-related laboratory markers. Ryplazim requires lesion resolution or clinical efficacy and, when applicable, trough plasminogen activity more than 10% above baseline. Reblozyl requires reduced RBC transfusion burden and must be stopped when the applicable maximum-dose response period is not met.Renal eGFR >30 or ≥30 mL/min/1.73 m2 as applicable; Ryplazim trough activity >10% above baseline; Reblozyl no response after specified 9-week maximum-dose period

The same baseline laboratory parameter may be required for recertification.

Neurologic, endocrine, oncologic, wound, and genetic response: Imaavy, Rystiggo, Uplizna, Ultomiris, eculizumab, and Vyvgart/Vyvgart Hytrulo require more than a 2-point MG-ADL improvement or provider-attested clinical benefit. Ilaris acute-gout renewal requires optimized urate-lowering therapy and at least 12 weeks between doses. Imcivree requires the applicable 5% or 10% weight/BMI response and later maintenance. Isturisa and Recorlev require UFC normalization and symptom improvement. Joenja requires improvement in APDS manifestations. Mycapssa, Palsonify, and Signifor require stable or improved GH/IGF-1 or disease-specific biochemical, imaging, or clinical response. Filsuvez and Vyjuvek require wound improvement with incomplete closure, reopening, or a new untreated wound requiring therapy. Scenesse requires reduced phototoxicity or improved light exposure/quality of life plus skin-lesion monitoring. Skyclarys requires ongoing benefit; Sohonos requires provider-attested FOP benefit; Sylvant requires no progression; Tavneos requires remission or meaningful improvement while continuing standard care; Vijoice requires objective target-lesion response; Voxzogo requires recent growth evidence and increased AGV; Vykat XR requires current weight and clinical benefit.MG-ADL improvement >2 points; Imcivree POMC/PCSK1/LEPR 5% then 10%, BBS 5%; Voxzogo AGV >1.5 cm/year

Accelerated-approval products may require confirmatory-trial verification of clinical benefit.

Time limits and discontinuation safeguards: Product-specific renewal periods apply, including 3-month Gamifant review intervals, 6-month cycles for many complement and biologic products, 9-month renal-product cycles where specified, 12-month renewals for many chronic therapies, and two-year Revcovi renewal. Tzield is limited to one 14-day course; Luxturna is limited to one dose per eye per lifetime; Skysona and Rethymic are not authorized for retreatment; Tepezza and Lumvoa retreatment is excluded as specified. Continued therapy is not authorized when required response, safety, renal function, cardiac function, or clinical stability is not maintained.Tzield 14 consecutive days; Luxturna one lifetime dose per eye; Skysona one-time dose

Requests must continue to satisfy product-specific combination and safety exclusions.

Prior Authorization and Provider Requirements

Prior Authorization

Obtain prior authorization and satisfy prerequisites

Submit prior authorization for the drugs and indications addressed by the drug-specific criteria. Approval depends on the applicable diagnosis, age, prescriber or specialist involvement, clinical history, laboratory or diagnostic evidence, treatment prerequisites, and response requirements; unless individual criteria specify otherwise, approval periods follow the policy guidelines.

Document required prior treatment failure, serious adverse effects, intolerance, or contraindication when step therapy applies.
Track authorization periods and submit recertification evidence, including tolerance, clinical response, laboratory response, or reduced treatment burden as applicable.
Examples include initial approval of 6 months with 12-month recertification for Adzynma and Aqvesme, and initial approval of 2 months with 2-month continuation review for Gamifant in Still disease-associated HLH/MAS.

Documentation Required

Submit complete clinical documentation

Include complete clinical and treatment documentation with each request. Depending on the drug and indication, this may include diagnostic confirmation, specialist information, baseline laboratory or imaging results, current weight and requested dose, prior-treatment history, administration details, and objective response at recertification.

Provide required diagnostic evidence, such as flow cytometry, biopsy, molecular or genetic testing, enzyme analysis, antibody testing, imaging, or electrophysiology.
Submit baseline and follow-up measurements when required, including hemoglobin, transfusion history, eGFR, proteinuria or UPCR, LVEF, MG-ADL, urinary free cortisol, triglycerides, wound measurements, or growth data.
For continuation requests, document the policy-defined response, such as fewer transfusions, improved hemoglobin, reduced proteinuria, improved symptoms, clinical benefit, or objective imaging or laboratory improvement.

Denial Risk

Screen for denial risks before requesting coverage

Review the indication, dose, treatment combinations, and safety restrictions before submitting a request. Coverage may be denied for non-FDA-approved indications, excluded diagnoses or clinical conditions, excessive dosing, prohibited combinations, inadequate response, or failure to meet required monitoring or treatment restrictions.

Do not request coverage for a subscriber contract exclusion; policy criteria are not applied in that circumstance.
Avoid prohibited concomitant therapies, including specified complement-inhibitor combinations, unstudied generalized myasthenia gravis combinations, and same-wound use of Filsuvez with Vyjuvek or Zevaskyn.
Observe product-specific dose and administration limits, including Actimmune doses no higher than 50 mcg/m², Scenesse administration no more frequently than every 2 months, and Tzield’s single 14-day course.

Coding and Clinical Thresholds

HCPCSHCPCS

J1931

HCPCS code listed for Aldurazyme (laronidase)

Enjaymo HCPCSHCPCS

J1302

HCPCS code listed for Enjaymo (sutimlimab-jome)

Evkeeza HCPCSHCPCS

J1305

HCPCS code listed for Evkeeza (evinacumab-dgnb)

HCPCSHCPCS

J9210

HCPCSHCPCS

J0638

HCPCSHCPCS

J9256

Luxturna HCPCS CodeHCPCS

J3398

HCPCS code listed for Luxturna

Lanreotide and Somatuline Depot CodesHCPCS

J1930	Code listed for lanreotide acetate or Somatuline Depot
J1932	Code listed for lanreotide acetate

Lanreotide/Somatuline reference medical benefit codesHCPCS

J1930	Lanreotide acetate or Somatuline Depot
J1932	Lanreotide acetate

Piasky medical benefit codeHCPCSCovered

J1307

Piasky (crovalimab-akkz)

Reblozyl medical benefit codeHCPCSCovered

J0896

Reblozyl (luspatercept-aamt)

Rystiggo HCPCSHCPCSCovered

J9333

Rystiggo (rozanolixizumab-noli)

Lanreotide and Somatuline reference codesHCPCS

J1930	Lanreotide acetate or Somatuline Depot
J1932	Lanreotide acetate

Sandostatin/octreotide HCPCSHCPCSCovered

J2353

Sandostatin LAR and octreotide intramuscular injection

Scenesse HCPCSHCPCSCovered

J7352

Scenesse (afamelanotide)

Codes referenced for required lanreotide step therapyHCPCS

J1930	Lanreotide acetate or Somatuline Depot billing code referenced in acromegaly step therapy
J1932	Lanreotide acetate billing code referenced in acromegaly step therapy

Signifor LAR HCPCSHCPCS

J2502

HCPCS code listed for Signifor LAR

HCPCSHCPCS

J3387

HCPCS code listed in the policy window

Eculizumab HCPCS codesHCPCSCovered

J1300	Injection, eculizumab, 10 mg (effective until 3/31/2025)
J1299	Injection, eculizumab, 2 mg (effective 4/1/2025)
Q5151	Injection, eculizumab-aagh (Epysqli), biosimilar, 2 mg (effective 4/1/2025)
Q5152	Injection, eculizumab-aeeb (Bkemv), biosimilar, 2 mg (effective 4/1/2025)

Sylvant HCPCS codeHCPCSCovered

J2860

HCPCS code listed for Sylvant (siltuximab)

Tepezza HCPCSHCPCSCovered

J3241

HCPCS code listed for Tepezza (teprotumumab-trbw)

Tzield HCPCSHCPCSCovered

J9381

HCPCS code listed for Tzield (teplizumab-mzwv)

HCPCSHCPCS

J9381

HCPCS : J9381

HCPCSHCPCS

J1303

HCPCS: J1303

HCPCSHCPCS

J1823

HCPCS : J1823

Drug-specific clinical thresholds

ADAMTS13 activity for congenital TTP<10%

Aqvesme hemoglobin≤10.0 g/dL, averaged over at least 2 measurements separated by ≥7 days

Aqvesme transfusion-independent exposure≤5 RBC units in the previous 24 weeks and no RBC transfusion in the previous 8 weeks

Recurrent pericarditis activityPericardial pain >1 day with NRS pain score >4 and CRP ≥1 mg/dL

Camzyos LVEF≥55% for initiation; continued therapy is not approved if <50%

Empaveli renal thresholdFor C3G or IC-MPGN, baseline UPCR >1.0 g/g and eGFR ≥30 mL/min/1.73 m²

Primary HLH clinical diagnosisAt least 5 of the specified diagnostic criteria when genetic or family-history confirmation is unavailable

Required Prior Treatment

Drug or indication	Step	Required prerequisite or pathway	Failure, intolerance, or exception
Aqvesme — transfusion-dependent beta-thalassemia
1
Reblozyl before a new start
Document serious adverse effects or drug failure with Reblozyl. This requirement does not apply after prior splenectomy.
Arcalyst — DIRA
1
Kineret (anakinra) before Arcalyst
Document demonstrated clinical benefit, such as resolution of rash, bone pain, or fever; normal CRP; absence of rash; no active bone disease on imaging; or reduced corticosteroid use.
Arcalyst — recurrent pericarditis
1
NSAIDs, colchicine, or corticosteroids before Arcalyst
Disease must recur despite these therapies; the prescriber must attest that anti-inflammatory therapy will be tapered and discontinued.
Bylvay — PFIC or ALGS
1
At least one conventional medication for cholestasis or pruritus; ALGS also requires Livmarli
Document serious adverse effects or drug failure with the required conventional therapy and, for ALGS, with Livmarli.
Camzyos
1
Separate trials of a non-vasodilating beta blocker and a non-dihydropyridine calcium-channel blocker
Document serious adverse effects or drug failure; a trial may be bypassed when medically inappropriate.
Cuprimine — brand penicillamine
1
Generic penicillamine capsules, then generic penicillamine tablets
Document serious adverse effects or drug failure with both generics, unless clinically inappropriate with adequate justification.
Penicillamine tablets
1
Generic penicillamine capsules
Document serious adverse effects or drug failure, unless capsules are not clinically appropriate with adequate justification.
Empaveli — C3G or IC-MPGN
1
Maximally recommended or tolerated ACE inhibitor or ARB for at least 3 months, plus systemic oral glucocorticoid or mycophenolate therapy
ACE inhibitor/ARB use may be replaced by documented intolerance or contraindication. Glucocorticoid or mycophenolate therapy may be replaced by documented serious adverse effects or contraindication; otherwise document treatment failure.
Enjaymo
1
Rituximab-containing product before Enjaymo
Document serious adverse effects or drug failure. The prerequisite may be waived when severe hemolysis or symptoms require a more immediate response.
Evkeeza — age 10 years and older
1
Maximally tolerated high-potency statin plus ezetimibe and a PCSK9 inhibitor for at least 8 weeks
Must fail to reach target LDL-C. PCSK9 inhibitor trial is not required with two LDL-receptor-negative alleles.
Evkeeza — ages 1 to 9 years
1
At least one lipid-lowering therapy for at least 8 weeks, such as a statin, ezetimibe, or lipoprotein apheresis
Must fail to reach target LDL-C.
Fabhalta — IgAN
1
Filspari or Tarpeyo before Fabhalta
Document failure of Filspari or Tarpeyo.
Fabhalta — C3G
1
Systemic oral glucocorticoids or mycophenolate mofetil/mycophenolate sodium
Document treatment failure, unless serious adverse effects or contraindication are documented.
Filspari — IgAN
1
SGLT2 inhibitor for at least 3 months
Document failure, unless serious adverse effects or contraindication are documented.
Filspari — IgAN
2
Systemic oral glucocorticoid for at least 6 weeks
Document failure, unless serious adverse effects or contraindication are documented. A high-proteinuria or reduced-eGFR pathway may alternatively qualify.
Filsuvez — previously treated wound
1
Vyjuvek or Zevaskyn previously used on the same wound
Document inadequate response or treatment failure.
Gamifant — primary HLH
1
Conventional HLH therapy, such as etoposide/dexamethasone, cyclosporine A, antithymocyte globulin, or intrathecal methotrexate
Active disease must be refractory, recurrent, or progressive, or severe intolerance to at least one conventional therapy must be documented.
Ilaris — active sJIA or AOSD
1
Approved DMARD or anti-inflammatory therapy, such as methotrexate, NSAIDs, analgesics, or corticosteroids
Document failure to respond or intolerance; therapies may be used alone or in combination.
Ilaris — acute gout
1
NSAIDs, colchicine, and oral or injectable corticosteroids
Document serious adverse effects, failure at maximally tolerated doses, or contraindication to each therapy.
Imaavy — generalized myasthenia gravis
1
Corticosteroids for at least 3 months
Document serious adverse effects or drug failure.
Imaavy — generalized myasthenia gravis
1
Non-steroidal immunosuppressive therapy for at least 6 months, such as azathioprine, mycophenolate mofetil, or cyclosporine
Document serious adverse effects or drug failure.
Imaavy — generalized myasthenia gravis
2
Plasma exchange, plasmapheresis, or IVIG within the prior 12 months despite at least one immunosuppressant
This is an alternative rescue-therapy pathway.
Imaavy — adult new start
2
Vyvgart Hytrulo prefilled syringe
Document serious adverse effects or drug failure.
Isturisa — Cushing disease
1
Oral ketoconazole and at least two of Signifor, Lysodren, and cabergoline
Document serious adverse effects or failure. Only one of the listed agents is required if prior metyrapone failure is documented.
Juxtapid
1
Age-appropriate lipid-lowering therapy
For ages 10 years and older, fail target LDL-C on maximally tolerated high-potency statin plus ezetimibe and PCSK9 inhibitor for at least 8 weeks. Ages 2–9 require failure of at least one lipid-lowering therapy for at least 8 weeks.
Juxtapid — PCSK9 pathway
2
PCSK9 inhibitor with maximally tolerated statin plus ezetimibe
The PCSK9 inhibitor trial is not required when two LDL-receptor-negative alleles are documented.
Periodic paralysis therapies
1
Potassium supplementation when applicable for hypokalemic disease and oral acetazolamide
Document failure to improve attack severity or frequency; potassium supplementation failure is required when applicable.
Keveyis or Ormalvi
1
Generic dichlorphenamide
Document serious adverse effects or drug failure.
Korlym — brand
1
Generic mifepristone 300 mg tablets
Document serious adverse effects or drug failure with the generic product.
Livmarli — ALGS
1
At least one conventional medication for cholestasis or pruritus
Document serious adverse effects or drug failure.
Livmarli — PFIC
1
At least one conventional medication plus Bylvay
Document serious adverse effects or drug failure with both required therapies.
Mycapssa
1
Injectable octreotide or lanreotide for at least 6 months, stable for the last 3 months
Document clinical response and tolerance before switching to Mycapssa.
Myqorzo
1
Separate trials of a non-vasodilating beta blocker and a non-dihydropyridine calcium-channel blocker
Document serious adverse effects or drug failure; a documented medical reason may prevent use.
Palsonify
1
Maximally tolerated lanreotide acetate or Somatuline Depot
Document serious adverse effects or drug failure.
Palsonify
1
Pituitary surgery and/or radiation, unless not a candidate, plus lanreotide/Somatuline
Document inadequate response after surgery/radiation or inability to undergo those interventions, and failure or serious adverse effects with lanreotide/Somatuline.
Procysbi
1
Immediate-release cysteamine (Cystagon)
Document severe nausea, vomiting, anorexia, fever, or lethargy interfering with activities of daily living. Nonadherence alone does not qualify unless its cause is resolved.
Recorlev — Cushing syndrome
1
Cushing-specific surgery and oral ketoconazole; pituitary disease also requires Signifor, Lysodren, and/or cabergoline
Document surgery failure or contraindication and serious adverse effects or drug failure with ketoconazole and the required prior agents.
Reblozyl — MDS or MDS/MPN-RS-T
1
Erythropoiesis-stimulating agent pathway
Document no prior ESA, ESA refractoriness, EPO ≥500 mU/mL, or ESA intolerance, adverse event, or contraindication. ESA refractoriness is assessed after 6–8 weeks.
Redemplo — inconclusive genetic testing
1
Standard triglyceride therapies: statins, fibrates, or high-dose omega-3 fatty acids
Document refractoriness and rule out other causes of hypertriglyceridemia; the clinical pathway also requires pancreatitis or recurrent abdominal pain.
Rystiggo — generalized myasthenia gravis
1
Corticosteroids for at least 3 months or non-steroidal immunosuppressive therapy for at least 6 months
Document serious adverse effects or drug failure. Alternatively, document rescue therapy with plasma exchange, plasmapheresis, or IVIG within 12 months despite immunosuppressant treatment.
Rystiggo — new start
2
Vyvgart Hytrulo prefilled syringe
Document serious adverse effects or drug failure; requirement applies to new starts in all lines of business.
Sandostatin LAR or octreotide — selected new starts
1
Lanreotide acetate or Somatuline Depot
Document serious adverse effects or drug failure for applicable acromegaly, neuroendocrine-tumor, or adrenal-tumor indications.
Signifor LAR — acromegaly
1
Maximally tolerated lanreotide acetate or Somatuline Depot
Must fail to achieve full biochemical control, defined as GH <2.5 mcg/L and normal IGF-1.
Soliris or Bkemv
1
Preferred Epysqli
Use Epysqli unless medical justification establishes that it cannot be used; Medicare Advantage applies the preference to new starts only.
Eculizumab — generalized myasthenia gravis
1
Corticosteroids for at least 3 months
Document serious adverse effects or drug failure.
Eculizumab — generalized myasthenia gravis
2
Non-steroidal immunosuppressive therapy for at least 6 months
Document serious adverse effects or drug failure.
Eculizumab — generalized myasthenia gravis
3
Plasma exchange, plasmapheresis, or IVIG within the prior 12 months despite at least one immunosuppressant
Alternative rescue-therapy pathway.
Eculizumab — adult gMG new start
2
Vyvgart Hytrulo prefilled syringe
Document serious adverse effects or drug failure.
Eculizumab — PNH or aHUS new start
1
Ultomiris
Document serious adverse effects or drug failure with Ultomiris.
Trientine products — Wilson disease
1
Penicillamine, followed by generic trientine 250 mg capsules for higher-strength or branded products
Document serious adverse effects or drug failure with penicillamine. Generic trientine 250 mg may be bypassed only with adequate clinical justification.
Tarpeyo
1
Maximum tolerated ACE inhibitor/ARB for at least 3 months plus an SGLT2 inhibitor or systemic oral glucocorticoid trial
SGLT2 inhibitor failure requires at least 3 months; glucocorticoid failure requires at least 6 weeks. Serious adverse effects or contraindication may substitute; qualifying proteinuria/eGFR may provide an alternative pathway.
Trutakna
1
Maximum or maximally tolerated ACE inhibitor/ARB for at least 3 months
Document intolerance or contraindication when the prerequisite cannot be used; also meet the SGLT2/glucocorticoid or high-risk renal pathway.
Trutakna
1
SGLT2 inhibitor for at least 3 months or systemic oral glucocorticoid for at least 6 weeks
Document drug failure; serious adverse effects or contraindication may substitute. High-risk proteinuria or reduced eGFR may qualify instead.
Tryngolza — severe hypertriglyceridemia
1
Maximally tolerated high-potency statin plus omega-3 fatty acids or a fibric acid derivative for at least 4 weeks
Document failure of combination therapy; the policy specifies goal doses of atorvastatin 40–80 mg/day or rosuvastatin 20–40 mg/day.
Tryngolza — FCS
1
Dietary fat restriction and, where applicable, standard triglyceride therapies
Document appropriate dietary restriction and refractoriness to standard therapy when required by the selected FCS confirmation pathway.
gMG biologic new starts, including Ultomiris and Uplizna
1
Corticosteroids or non-steroidal immunosuppressive therapy; recent plasma exchange, plasmapheresis, or IVIG may be an alternative
Document serious adverse effects or drug failure after corticosteroids for at least 3 months or non-steroidal therapy for at least 6 months, or qualifying rescue therapy within 12 months.
NMOSD — Ultomiris or Uplizna
1
Enspryng; Ultomiris also requires Uplizna
Document serious adverse effects or drug failure with the required prior therapy; a qualifying relapse requiring rescue therapy within the prior 12 months is also required.
Vanrafia
1
Maximum or maximally tolerated ACE inhibitor/ARB for at least 3 months, followed by Filspari or Tarpeyo
Document ACE inhibitor/ARB intolerance or contraindication when applicable and drug failure with Filspari or Tarpeyo.
Voxzogo — age 2 years and older
1
Yuviwel
Document serious adverse effects or drug failure. Voxzogo cannot be used in combination with Yuviwel.
Voydeya
1
Ravulizumab or eculizumab for at least 6 months
Voydeya must be used with one of these agents; other complement-inhibitor combinations are not authorized.
Voyxact
1
Maximum or maximally tolerated ACE inhibitor/ARB for at least 3 months
Document intolerance or contraindication when applicable; an SGLT2 inhibitor or glucocorticoid pathway is also required.
Voyxact
2
SGLT2 inhibitor for at least 3 months
Document drug failure, unless serious adverse effects or contraindication are documented.
Voyxact
2
Systemic oral glucocorticoid for at least 6 weeks
Alternative pathway; document drug failure, unless serious adverse effects or contraindication are documented.
Vyjuvek — previously treated wound
1
Filsuvez or Zevaskyn previously used on the same wound
Document inadequate response or treatment failure.
Vyvgart/Vyvgart Hytrulo
1
Corticosteroids for at least 3 months or non-steroidal immunosuppressive therapy for at least 6 months
Document serious adverse effects or drug failure. Alternatively, document plasma exchange, plasmapheresis, or IVIG within the prior 12 months despite immunosuppressant therapy.

Quantity and Dose Limits

Quantity limits and exceptions

Actimmune1 vial per 28 days; quantity is limited to the minimum number of vials needed for FDA-approved body-surface-area dosing

Arcalyst4 vials per 28 days; one-time loading-dose override of 5 vials for CAPS, FCAS, MWS, or recurrent pericarditis; up to 8 vials per 28 days for DIRA requiring two weekly injections

Cuprimine or penicillamine180 capsules per 30 days; exceptions allow 480 capsules for cystinuria or 240 capsules for Wilson disease

Empaveli8 vials per 30 days; exception of 10 vials per 30 days when 1,080 mg is dosed every 3 days

Enspryng1 syringe per 28 days; initial loading allowance is 3 syringes per 28 days for the first 4 weeks

Filspari30 tablets per 30 days; exception of 60 tablets per 30 days for 400 mg FSGS dosing in patients weighing ≥50 kg

Pyrukynd taper packs5 mg taper pack: 7 tablets per 365 days; 20 mg-5 mg and 50 mg-20 mg taper packs: 14 tablets per 365 days

Administration and Benefit Routing

Billing Rule

Use the correct benefit and administration site

Route and administer the drug according to the applicable benefit and site-of-care rule. Pharmacy-benefit, medical-benefit, home, office, infusion-center, hospital, and certified-treatment-center requirements vary by product and by who administers the therapy.

Self-administered therapies generally use the pharmacy benefit; medical-benefit review may be available when inability to self-administer or lack of a caregiver is documented.
Healthcare-professional administration may require the medical benefit, such as Reblozyl, Scenesse, and professional-administered Revcovi.
Site-specific administration is required for certain therapies: Luxturna at a certified treatment center by an ophthalmic surgeon, Rethymic as surgery by an experienced surgeon, and Tzield by IV infusion for 14 consecutive days.
Prior authorization applies to Gamifant regardless of inpatient or outpatient administration site.

Policy Definitions

Clinical and disease definitions

Rare diseaseA disease affecting fewer than 200,000 people in the United States.

Non-transfusion-dependent thalassemiaAlpha- or beta-thalassemia with no more than 5 RBC units transfused in the previous 24 weeks and no RBC transfusion in the previous 8 weeks.

Transfusion-dependent thalassemiaAlpha- or beta-thalassemia with 6 to 20 RBC units transfused in the previous 24 weeks and a transfusion-free period no longer than 6 weeks.

Recurrent pericarditisA subsequent pericarditis episode after a symptom-free interval of at least 4 to 6 weeks.

Molecularly confirmed PFIC or ALGSPFIC types 1-6 and Alagille syndrome confirmed by molecular genetic testing as specified in the policy.

Active macrophage activation syndromeFerritin >684 ng/mL plus at least two of: platelets ≤181×10^9/L, AST >48 U/L, triglycerides >156 mg/dL, or fibrinogen ≤360 mg/dL.

MGFA class II-IVaMyasthenia Gravis Foundation of America clinical classification class II to IVa.

Preferred Products and Product Substitution

Step Therapy

Use preferred formulations and strengths

Select preferred Epysqli for eculizumab requests. Soliris and Bkemv require Epysqli use or medical justification explaining why Epysqli cannot be used; the preferred-product requirement applies to new starts and existing users for all lines of business except Medicare Advantage, where it applies only to new starts.

Epysqli is the preferred eculizumab formulation.
If requesting Soliris or Bkemv instead, include medical justification for why Epysqli cannot be used.
For trientine products, use generic trientine 250 mg capsules before trientine 500 mg capsules, Cuvrior, or Syprine unless the prescriber adequately justifies that the 250-mg generic is not clinically appropriate.

Rare Diseases Clinical Review Prior Authorization (CRPA) Drug Policy

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Drug-Specific Coverage Criteria

Prior Authorization and Provider Requirements

Coding and Clinical Thresholds

Required Prior Treatment

Quantity and Dose Limits

Administration and Benefit Routing

Policy Definitions

Rare-Disease Policy Background

Preferred Products and Product Substitution

Scope and Contract Applicability