Voydeya (danicopan)

POLICY NUMBER

L.5.01.618

Voydeya (danicopan tablet)

Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

DESCRIPTION

Danicopan is a small molecule Factor D inhibitor that selectively inhibits the alternative complement pathway. Danicopan prevents cleavage of complement Factor B into the Ba and Bb fragments that are required for formation of the alternative pathway complement component C3 convertase (C3bBb), the generation of downstream effectors including C3 fragment opsonization, and the amplification of the terminal pathway. In PNH, intravascular hemolysis (IVH) is mediated by the terminal membrane attack complex (MAC), while extravascular hemolysis (EVH) is facilitated by C3 fragment opsonization. Danicopan acts proximally in the alternative pathway of the complement cascade to control preferentially C3 fragment-mediated EVH, while co-administered ravulizumab or eculizumab is anticipated to maintain control over MAC-mediated IVH.

Voydeya (danicopan) is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH).

Related medical policies - 

• Complement Inhibitors

POLICY

Prior authorization is required.

The use of samples by an individual will not be considered current or stable therapy to satisfy Medical Policy requirements. 

Initial Criteria

Voydeya (danicopan) may be considered medically necessary when ALL of the following criteria are met:

1. The individual is at least 18 years of age;

2. There is no evidence of an unresolved serious infection caused by encapsulated bacteria, including Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae type B;

3. ONE of the following:

   1. The individual has been vaccinated against encapsulated bacteria (e.g., Streptococcus pneumoniae, Neisseria meningitidis) at least two weeks prior to administration of the first dose of the requested agent; OR

   2. The individual has received two weeks of antibacterial drug prophylaxis prior to administration of the first dose if the requested agent must be initiated immediately or if vaccines are administered less than 2 weeks before starting the requested agent;

4. The individual has a documented diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) and ALL of the following:

   1. Clinical documentation supporting the diagnosis includes BOTH of the following:

      1. Flow cytometry confirmation of at least 2 different glycosylphosphatidylinositol (GPI)-linked protein deficiencies (e.g., CD55, CD59, etc.) within at least 2 different cell lines (e.g., granulocytes, monocytes, erythrocytes); AND

      2. Laboratory results, signs, and or/symptoms attributed to PNH (e.g., anemia, extreme fatigue, dyspnea, transfusion-dependence, episodic hemοglοbinuria, thrombosis, pain, and/or organ dysfunction);

   2. The individual has clinically significant extravascular hemolysis (EVH) as indicated by BOTH of the following:

      1. Hemoglobin less than or equal to 9.5 g/dL; AND

      2. Absolute reticulocyte count greater than or equal to 120 x 10^9/L with or without transfusion support;

   3. The individual has received a stable dose of eculizumab or ravulizumab for at least 6 months;

   4. The requested agent will be used concomitantly as add-on therapy with Soliris (eculizumab) or Ultomiris (ravulizumab-cwvq); AND

   5. The requested agent will NOT be used in combination with a complement protein C3 inhibitor or a complement factor B inhibitor for the treatment of PNH;

5. The prescriber is a specialist in the area of the individual’s diagnosis or has consulted with a specialist in the area of the individual’s diagnosis (e.g., hematologist/oncologist);

6. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

7. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Length of Approval: 6 months 

Renewal Criteria

Voydeya (danicopan) may be approved for RENEWAL when ALL of the following criteria are met:

1. The individual been previously approved for the requested agent through the BCBSMS PA process;

2. The individual has documented clinical response/improvement (i.e., stabilization/improvement of hemoglobin levels from pretreatment baseline, decrease in serum LDH from pretreatment baseline, decrease in packed RBC transfusion requirement, etc.);

3. The individual continues to receive the requested agent in combination with Soliris (eculizumab) or Ultomiris (ravulizumab);

4. The prescriber is a specialist in the area of the individual’s diagnosis or has consulted with a specialist in the area of the individual’s diagnosis (e.g., hematologist/oncologist);  

5. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent;

6. The requested agent is NOT used in combination with a complement protein C3 inhibitor or a complement factor B inhibitor for the treatment of PNH; AND

7. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Voydeya (danicopan) is considered not medically necessary when used for all other indications not outlined in this policy including, but not limited to, use as monotherapy.

POLICY EXCEPTIONS

State Health Plan (State and School Employees): The prescription drug(s) in this medical policy may be covered under a prescription drug benefit plan administered by the State Health Plan’s Pharmacy Benefit Manager. Please perform a formulary drug search at https://www.dfa.ms.gov/cvs-caremark and submit any required Prior Authorization Requests for coverage determination to the Plan’s Pharmacy Benefit Manager. Services related to delivery and/or administration of a medication determined to be not medically necessary will also be considered not medically necessary. Services related to delivery and/or administration of a self-administered drug are not covered.

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

A.  consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

B.  appropriate with regard to standards of good medical practice; and

C.  not solely for the convenience of the Member, his or her Provider; and

D.  the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

Investigative is defined as the use of any treatment procedure, facility, equipment, drug, device, or supply not yet recognized as a generally accepted standard of good medical practice for the treatment of the condition being treated and; therefore, is not considered medically necessary. For the definition of Investigative, “generally accepted standards of medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. In order for equipment, devices, drugs or supplies [i.e, technologies], to be considered not investigative, the technology must have final approval from the appropriate governmental bodies, and scientific evidence must permit conclusions concerning the effect of the technology on health outcomes, and the technology must improve the net health outcome, and the technology must be as beneficial as any established alternative and the improvement must be attainable outside the testing/investigational setting.

Medication Failure

Medication failure is defined as disease progression at generally accepted doses (for ≥3 months use) as appropriate for the disease state being treated. Dosages below the recommended dose for the specific condition being treated and/or experience of common side effects of medication will not be considered medication failure or lack of response for the purpose of this review.

BCBSMS determines patient medication trial and adherence by a review of pharmacy claims data over the preceding twelve months. Additional information may be requested on a case-by-case basis to allow for proper review. If member is new to BCBSMS and pharmacy records are needed to confirm medication trials and adherence, it is the responsibility of the member and/or requesting provider to obtain said records and to submit them to BCBSMS upon request. Medical records from the provider that list previously prescribed medications will not be sufficient to show medication trials or adherence.

POLICY HISTORY

03/31/2025: New policy added. Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Effective 06/01/2025.

SOURCE(S)

1. Brodsky RA. Paroxysmal nocturnal hemoglobinuria: Treatment and prognosis. UpToDate, Connor RF (Ed), Wolters Kluwer.

   https://www.uptodate.com/contents/paroxysmal-nocturnal-hemoglobinuria-treatment-and-prognosis

2. Voydeya prescribing information. Alexion Pharmaceuticals Inc. December 2024. Last accessed January 2025.

CODE REFERENCE

None