Fosdenopterin (Nulibry™)
IMPORTANT REMINDER
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.
POLICY
I. INDICATIONS
The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.
FDA-Approved Indication
Nulibry is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.
All other indications are considered experimental/investigational and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization review:
A. Initial requests: genetic testing results documenting a mutation in the molybdenum cofactor synthesis 1 (MOSC1) gene, where applicable
B. Continuation requests (where applicable):
1. Genetic testing results documenting a mutation in the molybdenum cofactor synthesis gene 1 (MOSC1).
2. Chart notes or medical records documenting a benefit from therapy (e.g., improvement, stabilization, or slowing of disease progression for encephalopathy, seizure activity, improved or normalized uric acid, urinary S-sulfocysteine, and xanthine levels).
III. CRITERIA FOR INITIAL APPROVAL
Molybdenum cofactor deficiency (MoCD) Type A
A. Authorization 12 months may be granted when the diagnosis of MoCD Type A was confirmed by genetic testing documenting a mutation in the molybdenum cofactor synthesis gene 1 (MOSC1).
B. Authorization of 3 months may be granted when both of the following criteria are met:
1. Member has a presumed diagnosis of MoCD Type A and genetic test results are pending.
2. Member has clinical signs and symptoms associated with MoCD Type A (e.g., encephalopathy, intractable seizures, developmental delay, decreased uric acid levels, elevated urinary S-sulfocysteine and/or xanthine levels).
IV. CONTINUATION OF THERAPY
Authorization of 12 months may be granted for members with an indication listed in Section III when one of the following is met:
A. The member has received less than 12 months of therapy and has genetic testing results documenting a mutation in the molybdenum cofactor synthesis gene 1 (MOSC1).
B. Member has received 12 months of therapy or more and is experiencing benefit from therapy (e.g., improvement, stabilization, or slowing of disease progression for encephalopathy, seizure activity, improved or normalized uric acid, urinary S-sulfocysteine, and xanthine levels).
APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
ADDITIONAL INFORMATION
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
REFERENCES
1. Nulibry [package insert]. Boston, MA: Origin Biosciences, Inc.; October 2022.
2. Atwal PS, Scaglia F. Molybdenum cofactor deficiency. Mol Genet Metab. 2016;117(1):1-4.
3. Schwahn BC, Van Spronsen FJ, Belaidi AA, et al. Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study. Lancet. 2015; 386: 1955-1963.
4. ClinicalTrials.gov. Study of ORGN001 (formerly ALXN1101) in neonates with molybdenum cofactor deficiency (MOCD) type A. Available at: https://clinicaltrials.gov/ct2/show/NCT02629393. Accessed: October 25, 2023.
5. ClinicalTrials.gov. Safety & efficacy study of ORGN001 (formerly ALXN1101) in pediatric patients with MoCD type A currently treated with rcPMP. Available at: https://clinicaltrials.gov/ct2/show/NCT02047461. Accessed: October 25, 2023.
ORIGINAL EFFECTIVE DATE: 6/2/2021
MOST RECENT REVIEW DATE: 6/11/2024
ID_CHS
Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
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