Koselugo (selumetinib)

POLICY NUMBER

L.5.01.562

Koselugo (selumetinib)

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

Koselugo (selumetinib) is a kinase inhibitor that blocks mitogen-activated protein kinases 1 and 2 (MEK1/2). MEK1/2 regulates the extracellular signal-related kinase (ERK) pathway, and both are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers. Koselugo (selumetinib) is indicated for the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

Neurofibromatosis is a genetic disorder characterized by nerve cell tumors (neurofibromas) that can lead to skin changes and bone deformities. NF1 is the most common type of neurofibromatosis and can present as early as birth and nearly always by age 10. Symptoms include light brown spots on the skin, two or more growths on the iris of the eye, a tumor on the optic nerve, a larger than normal head circumference, and abnormal development of the spine, a skull bone, or the tibia. Standard treatment of NF1 may include surgery, radiation, or chemotherapy. Koselugo (selumetinib) is the first FDA approved drug treatment for NF1.

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.

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

1. The individual is between 2 and 18 years of age;

2. The individual has a diagnosis of neurofibromatosis type 1 (NF1) with BOTH of the following:

   1. The individual is symptomatic; AND

   2. The individual has inoperable plexiform neurofibromas (PN);

3. If female, the individual is not pregnant;

4. The individual does not have any contraindication(s) to therapy with the requested agent;AND

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

Length of Approval: 12 months

Renewal Evaluation

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

1. The individual has been previously approved through the BCBSMS PA process;

2. There is no evidence of disease progression or unacceptable toxicity;

3. The individual does not have any contraindication(s) to therapy with the requested agent; AND

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

Length of Approval: 12 months

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.

Medication failure is defined as disease progression despite maximally tolerated dose (≥3 months use) as appropriate for disease state being treated. Experience of common side effects of medication will not be considered medication failure for the purpose of this review.

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 [ie, 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.

POLICY HISTORY

08/18/2020: New policy added. Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

10/30/2020: Code Reference section updated to add HCPCS code J8499.

02/01/2023: Policy Exceptions updated to add the following: 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.

03/20/2025: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy statement revised to state that the use of samples by an individual will not be considered current or stable therapy to satisfy Medical Policy requirements. Policy statement criteria updated regarding age requirement, contraindications to therapy, and dose requirements. Policy Guidelines updated to change "Nervous/Mental Conditions" to "Mental Health Disorders." Sources updated.

SOURCE(S)

1. Koselugo prescribing information. AstraZeneca Pharmaceuticals LP. January 2024. Last accessed January 2025.

2. Neurofibromatosis Information Page. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/Disorders/All-Disorders/Neurofibromatosis-Information-Page . Last accessed January 2025.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

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