027 Form

Pharmacy Medical Policy

Antisense Oligonucleotide Medications

Table of Contents • Policy: Commercial • Information Pertaining to All Policies • Forms • Policy: Medicare • References • Policy History

Policy Number: 027 BCBSA Reference Number: None

Related Policies • N/A

Policy Commercial Members: Managed Care (HMO and POS), PPO, and Indemnity

Note: All requests for outpatient retail pharmacy for indications listed and not listed on the medical policy guidelines may be submitted to BCBSMA Clinical Pharmacy Operations by completing the Prior Authorization Form on the last page of this document. Physicians may also call BCBSMA Pharmacy Operations department at (800)366-7778 to request a prior authorization/formulary exception verbally. Patients must have pharmacy benefits under their subscriber certificates.

Prior Authorization Information ☒ Prior Authorization ☐ Step Therapy ☒ Quality Care Dosing

Pharmacy Operations: Tel: 1-800-366-7778 Fax: 1-800-583-6289 Policy last updated 3/15/2026 Pharmacy (Rx) or Medical (MED) benefit coverage ☒ Rx ☐ MED To request for coverage: Providers may call, fax, or mail the attached form (Formulary Exception/Prior Authorization form) to the address below.
Blue Cross Blue Shield of Massachusetts Pharmacy Operations Department 25 Technology Place Hingham, MA 02043

Individual Consideration: Policy for requests that do not meet clinical criteria of this policy, see section labeled Individual Consideration
Policy applies to Commercial members with BCBSMA formulary:
• Managed Care (HMO/POS) • PPO/EPO
• Indemnity
• MEDEX with Rx plans • Managed Blue for Seniors Policy does NOT apply to: • Medicare Advantage Provider Documentation Requirements: Documentation from the provider to support a reason preventing trial of formulary alternative(s) must include the name and strength of alternatives tried and failed (if alternatives were tried, including dates if available) and specifics regarding the treatment failure. Documentation to support clinical basis preventing switch to formulary alternative should also provide specifics around clinical reason. We may also use prescription claims records to establish prior use of formulary alternatives or to show if step therapy criteria has been met. We will require the provider to share additional information when prescription claims

data is either not available or the medication fill history fails to establish use of preferred formulary medications or that step therapy criteria has been met. Other documentation requirements, if any, are outlined in prior authorization criteria.
See Appendix for additional information.

Please refer to the chart below for the formulary and step status of the medications affected by this policy.

Drug Formulary Status Amondys 45 (casimersen)
PA
Exondys 51 (eteplirsen) PA
Givlaari (givosiran) PA
Qalsody (tofersen)
PA, Medical Viltepso (viltolarsen) PA
Vyondys 53 (golodirsen) PA

PA – Prior Authorization; Medical -- This medication is excluded from the pharmacy benefit. It may be covered by the medical benefit. Approval Length: See Clinical Guideline Coverage Criteria

Clinical Coverage Criteria: Amondys 45 (casimersen)

Initial Approval

We may cover Amondys 45 (casimersen) for the treatment Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping when ALL of the following criteria are met:

1. Confirmed diagnosis of Duchenne muscular dystrophy (DMD) which will benefit from Exon 45 skipping AND
2. Documentation of ambulation without assistance or devices AND
3. Concurrent use of glucocorticoids, unless clinically contraindicated AND
4. The prescription is written by a board certified / board eligible Neurologist AND

5. Dose is limited to FDA approved dosing of 30 mg/kg administered once weekly (weight and calculated dose required)

   Continuation of therapy Reauthorization will require the same criteria above. Clinical Coverage Criteria: Exondys 51 (eteplirsen)

   Initial Approval

   We may cover Exondys 51 (eteplirsen) for the treatment Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping when all of the following criteria are met: Length of Approval: If approved, a prior authorization will be granted for up to 6 months.

1. Confirmed diagnosis of Duchenne muscular dystrophy (DMD) which will benefit from Exon 51 skipping AND
2. Documentation of ambulation without assistance or devices AND
3. Concurrent use of glucocorticoids, unless clinically contraindicated AND
4. The prescription is written by a board certified / board eligible Neurologist AND

5. Dose is limited to FDA approved dosing of 30 mg/kg administered once weekly (weight and calculated dose required)

   Continuation of therapy Reauthorization will require the same criteria above.

   Clinical Coverage Criteria: Givlaari (givosiran)

   Initial Approval We may cover Givlaari (givosiran) for the treatment of adults with acute hepatic porphyria (AHP) when ALL of the following criteria are met:

6. Confirmed diagnosis of acute hepatic porphyria (AHP) [including acute intermittent porphyria (AIP), variegate porphyria (VP), aminolaevulinic acid dehydratase deficiency porphyria (ALAD), and hereditary coproporphyria (HCP)] AND
7. Patient is ≥ 12 years of age AND
8. Elevated urinary or plasma porphobilinogen (PBG) or ALA values within the past year AND
9. Patient has active disease, with at least 2 documented porphyria attacks within the last 6 months or 1 attack involving CNS AND

10. Patient is not anticipating a liver transplantation.

    Continuation of therapy Reauthorization will require the same criteria above.

    Clinical Coverage Criteria: Qalsody (tofersen)

    Initial Approval We may cover Qalsody (tofersen) for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene when ALL of the following criteria are met: Length of Approval: If approved, a prior authorization will be granted for up to 6 months. Length of Approval: If approved, continuation of therapy prior authorizations will be granted for up to Length of Approval: If approved, an initial prior authorization will be granted for up to 6 months.

1. Confirmed diagnosis of amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene
   AND
2. The previous or concurrent use of any form of Riluzole or Radicava (edaravone) AND

3. Patient is ≥ 18 years of age.

   Continuation of therapy Reauthorization will require the same criteria above.

   Clinical Coverage Criteria: Vyondys 53 (golodirsen), Viltepso (viltolarsen) Initial Approval We may cover Vyondys 53 (golodirsen) or Viltepso (viltolarsen) for the treatment Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping when ALL of the following criteria are met:

   1.Confirmed diagnosis of Duchenne muscular dystrophy (DMD) which will benefit from Exon 53 skipping AND

4. Documentation of ambulation without assistance or devices AND
5. Documentation of a recent (within four weeks of request) pre-treatment 6-Minute Walk Time of at least 300 meters while walking independently (e.g., without assist, cane, walker, wheelchair) AND
6. Concurrent use of glucocorticoids, unless clinically contraindicated AND
7. Member has stable pulmonary and Cardiac function AND
8. The prescription is written by a board certified / board eligible Neurologist AND
9. Member is not concurrently enrolled in a clinical trial to receive an experimental therapy for DMD AND

10. Dose is limited to FDA approved dosing of 30 mg/kg administered once weekly (weight and calculated dose required)

    Continuation of therapy Reauthorization will require the same criteria above.

    We do not cover the medications listed above for other conditions not listed above.

    CPT Codes / HCPCs Codes / ICD Codes Inclusion or exclusion of a code does not constitute or imply member coverage or provider reimbursement. Please refer to the member’s contract benefits in effect at the time of service to determine coverage or non- coverage as it applies to an individual member.

    Providers should report all services using the most up-to-date industry-standard procedure, revenue, and diagnosis codes, including modifiers where applicable.

    Length of Approval: If approved, prior authorizations will be granted for up to 12 months. Length of Approval: If approved, a prior authorization will be granted for up to 6 months.

The following codes are included below for informational purposes only; this is not an all-inclusive list.

The above medical necessity criteria MUST be met for the following codes to be covered for Commercial Members: Managed Care (HMO and POS), PPO, and Indemnity: HCPCS Codes HCPCS codes: Code Description C9071 Injection, viltolarsen (Viltepso), 10 mg C9399 Unclassified drugs or biologicals (This code should only be used for drugs and biologicals that are approved by the FDA on or after January 1, 2004) (Hospital Outpatient Use ONLY) J0223 Injection, givosiran, 0.5 mg (Givlaari) J1304 Injection, tofersen, 1 mg (Qalsody) J1428 Injection, eteplirsen, 10 mg (Exondys 51) J1429 Injection, golodirsen, 10 mg (Vyondys 53) J3490 Unclassified drugs J3590 Unclassified biologics

Appendix Member cost share consideration A higher non-preferred cost share may be applied if an exception request is approved for coverage of a non-preferred or a non-formulary/non-covered drug. Criteria Documentation Provider must submit supporting documentation (e.g., chart notes, lab results or other clinical information) to show that the member has met all approval criteria. Forms To request prior authorization using the Massachusetts Standard Form for Medication Prior Authorization Requests (eForm), click the link below: https://www.bluecrossma.org/medical- policies/sites/g/files/csphws2091/files/acquiadam- assets/023%20E%20Form%20medication%20prior%20auth%20instruction%20prn.pdf OR Print and fax, Massachusetts Standard Form for Medication Prior Authorization Requests #434 Individual Consideration (for Atypical Patients) Our medical policies are written for most people with a given condition. Each policy is based on peer reviewed clinical evidence. We also take into consideration the needs of atypical patient populations and diagnoses.
If the coverage criteria outlined is unlikely to be clinically effective for the prescribed purpose, the health care provider may request an exception to cover the requested medication based on an individual’s unique clinical circumstances. This is also referred to as “individual consideration” or an “exception request.”
Some reasons why you may need us to make an exception include: therapeutic contraindications; history of adverse effects; expected to be ineffective or likely to cause harm (physical, mental, or adverse reaction).
To facilitate a thorough and prompt review of an exception request, we encourage the provider to include additional supporting clinical documentation with their request. This may include: • Clinical notes or supporting clinical statements; • The name and strength of formulary alternatives tried and failed (if alternatives were tried) and specifics regarding the treatment failure, if applicable; • Clinical literature from reputable peer reviewed journals; • References from nationally recognized and approved drug compendia such as American Hospital Formulary Service® Drug Information (AHFS-DI), Lexi-Drug,

Clinical Pharmacology, Micromedex or Drugdex®; and • References from consensus documents and/or nationally sanctioned guidelines

Providers may call, fax or mail relevant clinical information, including clinical references for individual patient consideration, to:

Blue Cross Blue Shield of Massachusetts Pharmacy Operations Department 25 Technology Place Hingham, MA 02043 Phone: 1-800-366-7778 Fax: 1-800-583-6289 Samples Requests based exclusively on the use of samples will not meet coverage criteria for exception. Additional clinical information demonstrating medical necessity of the desired medication must be submitted by the requesting prescriber for review. Specialty Blue Cross Blue Shield of Massachusetts (BCBSMA*) members (other than Medex®; Blue MedicareRx, Medicare Advantage plans that include prescription drug coverage) obtaining the medication from the Pharmacy benefit instead of the Medical benefit will be required to fill their prescriptions for medications listed as specialty at one of the providers in our retail specialty pharmacy network, see link below: Link to Specialty Pharmacy List

Policy History Date Action 3/15/2026 Annual Medical Policy review: No criteria changes; updated formatting and references. 1/2024 Clarified coding information. 9/2023 Updated to include Qalsody ™ to the policy and updated IC to align with 118E MGL § 51A. 7/2023 Reformatted Policy. 7/2021 Updated to add Amondys 45 to the policy.
1/2021 Updated to add Viltepso to the policy 4/2020 Updated to add Vyondys-53 to the Policy. 2/2020 Updated to change the name of the policy and to add Givlaari to the policy. 2/2019 BCBSA National medical policy review. No changes to policy statements. New references added. 10/2018 Clarified coding information. 1/2018 Clarified coding information. 10/2017 Updated to change Walgreens Specialty Name. 7/2017 Updated to add AllCare to Pharmacy Specialty list. 5/2017 Implementation of a new policy including the medication Exondys -51 TM.

References

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3. Center for Disease Control and Prevention. Muscular Dystrohpy: MD STARnet Data and Statistics. 2016; http://www.cdc.gov/ncbddd/musculardystrophy/data.html. Accessed November 12, 2018.
4. Falzarano MS, Scotton C, Passarelli C, Ferlini A. Duchenne muscular dystrophy: from diagnosis to therapy. Molecules. Oct 07 2015;20(10):18168-18184. PMID 26457695
5. Food and Drug Administration. Accelererated Approval Letter to Sarepta Therapeutics: NDA 206488. 2016, September 19; https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2016/206488Orig1s000ltr.pdf. Accessed November 12, 2018.

5. McDonald CM, Henricson EK, Abresch RT, et al. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve. Sep 2013;48(3):343-356. PMID 23681930
6. Henricson E, Abresch R, Han JJ, et al. The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year. PLoS Curr. Jul 8 2013;5. PMID 23867975
7. Sarepta Therapeutics Inc. Prescribing Label: EXONDYS 51 (eteplirsen) injection, for intravenous use. 2016; https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/206488lbl.pdf. Accessed November 12, 2018.
8. Mendell JR, Rodino-Klapac LR, Sahenk Z, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. Nov 2013;74(5):637-647. PMID 23907995
9. Ruff S. Sarepta Presentations for the April 25, 2016 Meeting of the Peripheral and Central Nervous System Drugs Advisory Committee 2016; https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/Peripheraland CentralNervousSystemDrugsAdvisoryCommittee/UCM500822.pdf. Accessed November 12, 2018.
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11. Center for Drug Evaluation and Research. Application Number: 206488orig1s000. Summary Review. 2016; http://www.accessdata.fda.gov/drugsatfda_docs/nda/2016/206488_summary%20review_Redacted.pdf. Accessed November 7, 2017.
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24. Gloss D, Moxley RT, 3rd, Ashwal S, Oskoui M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. Feb 2 2016;86(5):465-472. PMID 26833937
25. Exondys 51® [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc.; August 2025.
26. Givlaari™ [package insert]. Cambridge, MA: Alnylam Pharmaceuticals, Inc.; September 2025.
27. Alnylam Pharmaceuticals. ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias. NCT03338816 accessed 1/9/2020. https://www.clinicaltrials.gov/ct2/results?cond=&term=NCT03338816. Accessed January 09, 2020.
28. Vyondys 53 ® [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc.; June 2024.
29. Amondys 45 ® [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc.;July 2024.
30. Qalsody ™ [package insert]. Cambridge, MA: Biogen MA Inc.; April 2023.