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Table 76: Neuromuscular Agents – Duchenne Muscular Dystrophy and Spinal Muscular Atrophy


A    B    C    D    E    F    G    H    I    J    K    L    M    N    O    P    Q    R    S    T    U    V    W    X    Y    Z


Drug Category: Genetic/Developmental Disorder

Medication Class/Individual Agents: Neuromuscular

I. Prior-Authorization Requirements

 Neuromuscular Agents - Duchenne Muscular Dystrophy Agents

Clinical Notes

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

casimersen Amondys 45 PA  
delandistrogene moxeparvovec-rokl Elevidys PA   CO, MB
eteplirsen Exondys 51 PA  
golodirsen Vyondys 53 PA  
viltolarsen Viltepso PA  

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

 

Casimersen, eteplirsen, golodirsen, and viltolarsen

  • Casimersen, eteplirsen, golodirsen, and viltolarsen are intravenously (IV) administered antisense oligonucleotides directed at dystrophin pre-messenger ribonucleic acid (mRNA) with amenable mutations
  • FDA-approved dosing: 
    • Casimersen, eteplirsen, golodirsen: 30 mg/kg weekly
    • Viltolarsen: 80 mg/kg weekly
  • The same motor function measures must be used for both the pre-treatment baseline and current test
  • Quarterly objective testing is required for recertification

Nusinersen and risdiplam

  • Nusinersen and risdiplam target survival motor neuron-2 (SMN2)
  • Use of nusinersen in combination with risdiplam has not been evaluated
  • Examples of baseline motor function tests to include: Hammersmith Functional Motor Scale [HFMSE], Hammersmith Infant Neurological Examination [HINE], Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders [CHOP INTEND], World Health Organization [WHO] Motor Milestones, etc.

Onasemnogene abeparvovec-xioi

  • MassHealth Drug Utilization Review will be reaching out to prescribers after PA approval to verify administration date and at ongoing intervals for long-term monitoring of response.
 

 Neuromuscular Agents - Spinal Muscular Atrophy Agents

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

nusinersen Spinraza PA   MB
onasemnogene abeparvovec-xioi Zolgensma PD PA   CO, MB
risdiplam Evrysdi PA  
Table Footnotes
CO Carve-Out. This agent is listed on the Acute Hospital Carve-Out Drugs List and is subject to additional monitoring and billing requirements.
 
PD Preferred Drug. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class.
 
MB This drug is available through the health care professional who administers the drug or in an outpatient or inpatient hospital setting. MassHealth does not pay for this drug to be dispensed through the retail pharmacy. If listed, PA does not apply through the hospital outpatient and inpatient settings. Please refer to 130 CMR 433.408 for PA requirements for other health care professionals. Notwithstanding the above, this drug may be an exception to the unified pharmacy policy; please refer to respective MassHealth Accountable Care Partnership Plans (ACPPs) and Managed Care Organizations (MCOs) for PA status and criteria, if applicable.
 

II. Therapeutic Uses

FDA-approved, for example:

  • Duchenne muscular dystrophy (Amondys 45, Elevidys, Exondys 51, Viltepso, Vyondys 53)
  • Spinal muscular atrophy (Evrysdi, Spinraza, Zolgensma)

Note: The above list may not include all FDA-approved indications.

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III.  Evaluation Criteria for Approval

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

  • All prior-authorization requests must include clinical diagnosis, drug name, dose, and frequency.
  • Dispensing in a 90-day supply of medication may be mandated or allowable for agents in this therapeutic class (designated by M90 or A90, respectively, in the Drug Notes section above). Applicable quantity limits are described below as units per day, per month, per 28 days, or as clinically appropriate, and may be extrapolated for fills of longer day supply. 
  • A preferred drug may be designated for this therapeutic class. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. Additional information about these agents, including PA requirements and preferred products, can be found within the MassHealth Drug List at www.mass.gov/druglist.
  • For recertification requests, approval may require submission of additional documentation including, but not limited to, documentation of: some or all criteria for the original approval; response to therapy; clinical rationale for continuation of use; status of member’s condition; appropriate diagnosis; appropriate age; appropriate dose, frequency, and duration of use for requested medication; complete treatment plan; current laboratory values; and member’s current weight.
  • Additional criteria may apply depending upon diagnosis and/or requested medication (see below). Other factors, including rebate and FDA-approval status, may change independently of scheduled MassHealth updates, which may result in additional restrictions.

 

Amondys 45

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • confirmed out-of-frame deletion in the DMD gene that is amenable to exon 45 skipping; and
    • prescriber is a neuromuscular neurologist or consult notes from a neuromuscular neurology office are provided; and
    • member is ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • appropriate dosing (30 mg/kg intravenously every week); and
    • one of the following:
      • member has received a corticosteroid for at least six months prior and member will continue to use a corticosteroid in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has at least a baseline measurement for each of the following timed function tests as shown in medical records (tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds); and
    • member has not previously received treatment with a gene therapy for DMD.

 

  • For recertification requests, documentation of all of the following is required:
    • member remains ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • member has a stable or improving pattern of 6MWTs as shown in medical records with results of a pretreatment baseline and all interim results (all previous 6MWTs results must be included); and
    • dosing remains appropriate; and
    • one of the following:
      • member continues to utilize corticosteroids in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has a stable or improving pattern of observed performance on at least two of the following five timed function tests as shown in medical records (all results for all tests must be included with the date of performance; tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds); and
    • member has not previously received treatment with a gene therapy for DMD.

  

Elevidys

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ four years of age and < six years of age at the time of administration; and
    • prescriber is a neuromuscular specialist; and
    • copy of genetic test with a confirmed mutation in the DMD gene; and
    • member does not have any deletion in exon 8 or exon 9 of the DMD gene; and
    • copy of baseline anti-AAVrh74 total binding antibody titers < 1:400; and
    • member has a baseline measurement for both of the following:
      • North Star Ambulatory Assessment, including scores and times on individual items (within the past three months); and
      • Six-minute walk test (within the past three months); and
    • member is ambulatory as defined by a current six-minute walk test (6MWT – distance walked in six minutes in meters) ≥ 200 meters; and
    • member is on a stable dose of corticosteroid; and
    • member has not previously been treated with delandistrogene moxeparvovec; and
    • member is not currently utilizing antisense oligonucleotides; and
    • appropriate dosing.

  

Evrysdi

  • Documentation of all of the following is required:
    • appropriate diagnosis [type 1, 2, or 3 spinal muscular atrophy (SMA)]; and
    • one of the following:
      • if pre-symptomatic, member has two copies of SMN2; or
      • if SMA type 1, member has two copies of SMN2; or
      • if SMA type 2 or 3, member is non-ambulatory; and
    • genetic test confirming diagnosis of SMA; and
    • prescriber is a neurologist or consult notes from a neurologist are provided; and
    • baseline motor function test; and
    • appropriate dosing for age and weight; and 
    • requested quantity is ≤ 5 mg (6.67 mL) per day; and
    • member does not have evidence of permanent ventilator dependence (defined as any of the following: member has an endotracheal tube, member has a tracheotomy tube, member had at least 14 days of continuous respiratory assistance for at least 16 hours per day); and
    • requested agent will not be used in combination with Spinraza.

 

  • For recertification requests, documentation of all of the following is required:
    • one of the following: 
      • current (within the past three months) motor function test documenting positive response to therapy; or
      • medical necessity for continuing therapy; and
    • member does not have evidence of permanent ventilator dependence (defined as any of the following: member has an endotracheal tube, member has a tracheotomy tube, member had at least 14 days of continuous respiratory assistance for at least 16 hours per day).

  

Exondys 51

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • confirmed out-of-frame deletion in the DMD gene that is amenable to exon 51 skipping; and
    • prescriber is a neuromuscular neurologist or consult notes from a neuromuscular neurology office are provided; and
    • member is ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • appropriate dosing (30 mg/kg intravenously every week); and
    • one of the following:
      • member has received a corticosteroid for at least six months prior and member will continue to use a corticosteroid in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has at least a baseline measurement for each of the following timed function tests as shown in medical records (tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds; and
    • member has not previously received treatment with a gene therapy for DMD.

 

  • For recertification requests, documentation of all of the following is required:
    • member remains ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • member has a stable or improving pattern of 6MWTs as shown in medical records with results of a pretreatment baseline and all interim results (all previous 6MWTs results must be included); and
    • dosing remains appropriate; and
    • one of the following:
      • member continues to utilize corticosteroids in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has a stable or improving pattern of observed performance on at least two of the following five timed function tests as shown in medical records (all results for all tests must be included with the date of performance; tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds); and
    • member has not previously received treatment with a gene therapy for DMD.

  

Spinraza

  • Documentation of all of the following is required:
    • appropriate diagnosis (type 1, 2, or 3 SMA); and
    • one of the following:
      • if pre-symptomatic, member has two copies of SMN2; or
      • if SMA type 1, member has two copies of SMN2; or
      • if SMA type 2 or 3, member is non-ambulatory; and
    • genetic test confirming diagnosis of SMA; and
    • prescriber is a neurologist or consult notes from a neurologist are provided; and
    • baseline motor function test; and
    • member does not have evidence of permanent ventilator dependence (defined as any of the following: member has an endotracheal tube, member has a tracheotomy tube, member had at least 14 days of continuous respiratory assistance for at least 16 hours per day); and
    • requested agent will not be used in combination with Evrysdi; and
    • appropriate dosing.

 

  • For recertification requests, documentation of all of the following is required:
    • one of the following:
      • current (within the past three months) motor function test documenting positive response to therapy; or
      • medical necessity for continuing therapy; and
    • member does not have evidence of permanent ventilator dependence (defined as any of the following: member has an endotracheal tube, member has a tracheotomy tube, member had at least 14 days of continuous respiratory assistance for at least 16 hours per day).

  

Viltepso

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • confirmed out-of-frame deletion in the DMD gene that is amenable to exon 53 skipping; and
    • prescriber is a neuromuscular neurologist or consult notes from a neuromuscular neurology office are provided; and
    • member is ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • appropriate dosing (80 mg/kg intravenously every week); and
    • one of the following:
      • member has received a corticosteroid for at least three months prior and member will continue to use a corticosteroid in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has at least a baseline measurement for each of the following timed function tests as shown in medical records (tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds); and
    • member has not previously received treatment with a gene therapy for DMD.

 

  • For recertification requests, documentation of all of the following is required:
    • member remains ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • member has a stable or improving pattern of 6MWTs as shown in medical records with results of a pretreatment baseline and all interim results (all previous 6MWTs results must be included); and
    • dosing remains appropriate; and
    • one of the following:
      • member continues to utilize corticosteroids in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has a stable or improving pattern of observed performance on at least two of the following five timed function tests as shown in medical records (all results for all tests must be included with the date of performance; tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds); and
    • member has not previously received treatment with a gene therapy for DMD.

  

Vyondys 53

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • confirmed out-of-frame deletion in the DMD gene that is amenable to exon 53 skipping; and
    • prescriber is a neuromuscular neurologist or consult notes from a neuromuscular neurology office are provided; and
    • member is ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • appropriate dosing (30 mg/kg intravenously every week); and
    • one of the following:
      • member has received a corticosteroid for at least six months prior and member will continue to use a corticosteroid in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has at least a baseline measurement for each of the following timed function tests as shown in medical records (tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds); and
    • member has not previously received treatment with a gene therapy for DMD.

 

  • For recertification requests, documentation of all of the following is required:
    • member remains ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 200 meters (test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); and
    • member has a stable or improving pattern of 6MWTs as shown in medical records with results of a pretreatment baseline and all interim results (all previous 6MWTs results must be included); and
    • dosing remains appropriate; and
    • one of the following:
      • member continues to utilize corticosteroids in combination with the requested agent; or
      • contraindication to corticosteroids; and
    • member has a stable or improving pattern of observed performance on at least two of the following five timed function tests as shown in medical records (all results for all tests must be included with the date of performance; tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner):
      • timed ten-meter walk/run (time in seconds); and
      • timed floor (supine) to stand (time in seconds); and
      • timed four-step descend (time in seconds); and
      • timed four-step climb (time in seconds); and
      • timed sit to stand (time in seconds); and
    • member has not previously received treatment with a gene therapy for DMD.

  

Zolgensma

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • prescriber is a neuromuscular specialist; and
    • member is < two years of age; and
    • genetic test confirming diagnosis of bi-allelic mutation in the SMN1 gene; and
    • genetic test confirming the member has two, three, or four copies of the SMN2 gene; and
    • baseline AAV9 antibody test confirming titers < 1:50; and 
    • pre-treatment baseline Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score; and
    • member does not have evidence of complete paralysis of limbs; and
    • member does not have evidence of permanent ventilator dependence (defined as any of the following: member has an endotracheal tube, member has a tracheotomy tube, member had at least 14 days of continuous respiratory assistance for at least 16 hours per day) at the time the requested agent is to be administered.


Original Effective Date: 05/2018

Last Revised Date: 03/2024


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Last updated 05/16/24

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