Kuvan (sapropterin)

POLICY NUMBER

L.5.01.444

Javygtor powder for oral solution (sapropterin dihydrochloride)

Javygtor tablets (sapropterin dihydrochloride)

Kuvan tablets (sapropterin dihydrochloride)

Kuvan powder for

oral

solution (sapropterin dihydrochloride)

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

Sapropterin is a phenylalanine hydroxylase activator indicated in conjunction with a phenylalanine-(Phe) restricted diet to reduce blood Phe levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-(BH4-) responsive Phenylketonuria (PKU).

DOSING:The recommended starting dose is dependent on age and ranges from 10-20mg/kg once daily. Not all patients with PKU respond to treatment with sapropterin. Response to treatment cannot be predetermined by laboratory testing and can only be determined by a therapeutic trial of sapropterin. Patients with PKU who are being treated with sapropterin should also be treated with a Phe-restricted diet, including dietary protein and Phe restriction. Responsive patients may receive a dosage within the range of 5-20mg/kg as outlined within the policy.

Recommended Maintenance Phenylalanine Levels for Classical PKU According to the National Institutes of Health (NIH) Consensus Statement

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 Evaluation:

Sapropterin may be considered medically necessary when ALL of the following criteria are met:

1. The individual is one month of age or older and has a diagnosis of phenylketonuria (PKU);

2. The individual is currently on and will continue with a phenylalanine-(Phe) restricted diet; 

3. Baseline blood Phe level (measured within 2 weeks prior to initiation of sapropterin therapy) is greater than 360 µmol/L;

4. The prescriber has verified the individual's dietary protein and Phe intake will NOT be modified during the initial evaluation period of sapropterin therapy;

5. The prescriber is a specialist with knowledge and expertise in metabolic diseases or genetic diseases;

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

7. The individual is not receiving the requested medication in combination with Palynziq (pegvaliase-pqpz); AND

8. The dose is within the FDA-labeled dose range of 5 to 20 mg/kg/day based on the individual's recent weight (within the last 90 days).

 Length of Approval: 2 months 

Renewal Evaluation

Sapropterin may be approved for RENEWAL when ALL of the following criteria are met:

1. The individual has been previously approved for treatment with sapropterin through BCBSMS PA process; 

2. The individual has been successfully treated with sapropterin as defined by ONE of the following:

   1. The individual's blood Phe level is being maintained within the acceptable range; OR

   2. The individual has a documented ≥30% decrease in blood Phe level from baseline;

3. The prescriber has verified that the individual's diet was NOT modified in any way during the initial 1-month trial of sapropterin therapy;

4. The prescriber is a specialist with knowledge and expertise in metabolic diseases or genetic diseases;

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

6. The individual is not receiving the requested medication in combination with Palynziq (pegvaliase-pqpz); AND

7. The dose is within the FDA-labeled dose range of 5 to 20mg/kg/day.

 Length of Approval: 6 months

POLICY EXCEPTIONS

Kuvan® Prior Authorization is not required for Federal Employee Program (FEP) members.

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. 

BCBSMS may request medical records for determination of medical necessity. When medical records are requested, letters of support and/or explanation are often useful, but are not sufficient documentation unless all specific information needed to make a medical necessity determination is included.

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. 

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

12/14/2015: New policy added.

05/27/2016: Policy number L.5.01.444 added.

06/13/2016: Approved by Pharmacy & Therapeutics (P&T) Committee.

05/16/2017: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

03/27/2018: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

06/06/2019: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Added drug names to the top of the policy. Policy statements unchanged. Sources updated.

11/01/2020: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Policy description updated. Revised policy statement regarding initial evaluation. Policy statement criteria for renewal evaluation revised to state that the patient has been previously approved for treatment with Kuvan (sapropterin) through BCBSMS PA process. Policy Guidelines updated to change "Nervous/Mental Conditions" to "Mental Health Conditions." Added information regarding BCBSMS request for medical records. Sources updated.

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 updated to add Javygtor powder for oral solution (sapropterin dihydrochloride) and Javygtor tablets (sapropterin dihydrochloride) to the list of products. Policy description and policy statements updated to change "Kuvan (Sapropterin)" to "Sapropterin." Policy description updated regarding indications for sapropterin. 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. Revised initial and renewal criteria for coverage of sapropterin. Sources updated.

SOURCE(S)

1. Javygtor prescribing information. Dr. Reddys Laboratories Inc. November 2022. Last accessed January 2025.

2. Kuvan prescribing information. BioMarin Pharmaceutical Inc. August 2024. Last accessed January 2025.

3. Phenylketonuria: Screening and Management. NIH Consensus Statement Online 2000 October 16-18;17(3):1-27. Last accessed January 2025 at:

   http://consensus.nih.gov/2000/2000phenylketonuria113html.htm

   .

4. Vockley J, Andersson HC, Antshel KM, et al. Phenylalanine hydroxylase deficiency: diagnosis and management guideline. Genet Med 2014; 16:188.

CODE REFERENCE

None