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Drug Category: Genetic/Developmental Disorder

Medication Class/Individual Agents: Neuromuscular

I. Prior-Authorization Requirements

 Neuromuscular Agents

Clinical Notes

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

eteplirsen Exondys 51 PA  
nusinersen Spinraza 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.

 

Eteplirsen

  • Eteplirsen is an intravenously (IV) administered antisense oligonucleotide directed at exon 51 of dystrophin pre-messenger ribonucleic acid (mRNA)
  • FDA-approved dosing: 30 mg/kg weekly
  • The same motor function measures must be used for both the pre-treatment baseline and current test

Nusinersen

  • Nusinersen is an intrathecally (IT) administered survival motor neuron-2 (SMN2) directed antisense oligonucleotide
  • FDA-approved dosing:
    • Initial: 12 mg IT every 14 days for three doses then, a fourth loading dose of 12 mg 30 days after the third dose
    • Maintenance: 12 mg intrathecally every four months thereafter
  • 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.
  • Recommended lab testing at baseline and before each dose: platelet count, prothrombin time and/or activated partial thromboplastin time, quantitative spot urine protein testing
 
Table Footnotes

II. Therapeutic Uses

FDA-approved, for example:

  • Duchenne muscular dystrophy (Exondys 51)
  • Spinal muscular atrophy (Spinraza)

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.
  • 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 the member’s condition and requested medication (see below).

    

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
    • one of the following:
      • 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
    • dosing is appropriate (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).

    

  • 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 (30 mg/kg intravenously every week); and
    • one of the following:
      • member continues to utilize corticosteroids in combination with the requested agent; or
      • contraindication to corticosteroid; 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).

  

Spinraza

  • Documentation of all of the following is required:
    • appropriate diagnosis (type 1, 2, or 3 spinal muscular atrophy); and
    • genetic test confirming diagnosis of spinal muscular atrophy; and
    • prescriber is a neurologist or consult notes from a neurology office are provided; and
    • baseline motor function test; and
    • appropriate dose.
  • For recertification requests, medical records documenting positive response to therapy (e.g., follow up information on motor function tests and/or member’s improvement or stability of function) is required.


Original Effective Date: 05/2018

Last Revised Date: 08/2019


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Last updated 11/08/19