71336-1000-XX ONPATTRO 10MG/5ML Solution (ALNYLAM PHARMACEUTICALS)
Patisiran is a double-stranded small interfering ribonucleic acid (siRNA) which is formulated as a lipid complex. This makes it deliverable to hepatocytes where it specifically binds to mutant and wild-type transthyretin (TTR) messenger RNA (mRNA). This causes degradation of TTR mRNA through RNA interference resulting in a reduction of serum TTR protein. Mean serum TTR was reduced by approximately 80% within 10 to 14 days after a single dose. Ultimately it leads to diminished TTR protein deposits in tissues.
Patisiran for the treatment of polyneuropathy due to Hereditary Transthyretin-Mediated Amyloidosis (hATTR amyloidosis)/Familial Amyloidotic Polyneuropathy (FAP) is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Patisiran for the treatment of other conditions/diseases is considered investigational.
Patisiran is considered medically appropriate if ALL of the following criteria are met:
Individual is 18 years of age or older
Must not be used in combination with other transthyretin (TTR) reducing agents (e.g., inotersen, tafamidis, etc.)
Diagnosis of Hereditary Transthyretin-Mediated Amyloidosis (hATTR amyloidosis)/Familial Amyloidotic Polyneuropathy (FAP) confirmed by ANY ONE of the following:
Tissue biopsy identifying amyloid deposition
Molecular Genetic Testing identifying pathogenic variation of the TTR gene
Used for the treatment of polyneuropathy as demonstrated by at least TWO of the following criteria:
Subjective symptoms are suggestive of neuropathy
Abnormal nerve conduction studies are consistent with polyneuropathy
Abnormal neurological examination is suggestive of neuropathy
Peripheral neuropathy is attributed to hATTR/FAP and other causes of neuropathy have been excluded
Baseline in strength/weakness has been documented using an objective clinical measuring tool (e.g., Medical Research Council (MRC) muscle strength, etc.)
Individual has not been the recipient of an orthotopic liver transplant (OLT)
Individual is receiving supplementation with vitamin A at the recommended daily allowance
Patisiran is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet initial approval criteria
Absence of any serious or life-threatening infusion-related reaction, e.g., severe infusion-related reactions, ocular symptoms related to hypovitaminosis A, etc.
Disease response compared to pre-treatment baseline as evidenced by stabilization or improvement in one or more of the following:
Signs and symptoms of neuropathy
MRC muscle strength
|INDICATION(S)||DOSAGE & ADMINISTRATION|
Polyneuropathy of hATTR/FAP
Weight < 100 kg - 0.3 mg/kg intravenously every 3 weeks
Weight ≥ 100 kg - 30 mg intravenously every 3 weeks
Preparing for Therapy:
Dosing is based on actual body weight
Individual should be premedicated with a corticosteroid, acetaminophen and antihistamines.
Infusion should be filtered and diluted and infused, via a pump, over at least 80 minutes.
Individual should receive vitamin A supplementation.
LENGTH OF AUTHORIZATION
Coverage will be provided for 12 months and may be renewed
Refer to DOSAGE LIMITS below
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.
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, the express terms of the health plan will govern.
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).
Lexicomp Online. (2020). AHFS DI. Patisiran. Retrieved October 16, 2020 from Lexicomp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2020, April). Patisiran. Retrieved October 16, 2020 from MICROMEDEX Healthcare Series.
Sekijima, Y. (2001, November 5, updated 2018, December). Hereditary Transthyretin Amyloidosis. In: Adam, M. P., Ardinger, H. H., Pagon, R.A., et al., eds., GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2019. Retrieved October 17, 2019 from https://www.ncbi.nlm.nih.gov/books/NBK1194/pdf/Bookshelf_NBK1194.pdf/.U. S. Food and Drug Administration. (2020, April). Center for Drug Evaluation and Research. ONPATTRO® (patisiran) lipid complex injection, for intravenous use. Retrieved October 16, 2020 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/210922s007lbl.pdf.
ORIGINAL EFFECTIVE DATE: 1/31/2019
MOST RECENT REVIEW DATE: 12/8/2020
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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Maximum billable units per dose and over time by indication as a Medical Benefit; 1 billable unit = 0.1 mg