FDA-approved, for example:

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

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

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.

Duvyzat

• Documentation of all of the following is required:
  • appropriate diagnosis; and 
  • copy of genetic test showing mutation in the DMD gene confirming the diagnosis; and
  • member is ≥ six years of age; and
  • prescriber is a neuromuscular neurologist or consult notes from a neuromuscular neurologist office are provided; and
  • member is ambulatory as defined by a current six-minute walk test (6MWT – distance walked in six minutes in meters) ≥ 200 meters; and
  • baseline timed 4-step climb ≤ 8 seconds; and
  • baseline time to rise from floor < 10 seconds; and
  • member has at least a baseline measurement for each of the following timed function tests as shown in medical records:
    • timed 10-meter walk/run (time in seconds); and
    • timed floor (supine) to stand (time in seconds); and
    • timed 4-step descend (time in seconds); and
    • timed 4-step climb (time in seconds); and
    • timed sit to stand (time in seconds); and
  • one of the following:
    • member is on a stable dose of corticosteroid; or
    • attestation that the member will initiate corticosteroid and continue to utilize chronic corticosteroids in combination with Duvyzat; or
    • demonstrated contraindication to corticosteroids; and
  • requested agent will not be used in combination with other disease-modifying therapies for DMD; and
  • member has not previously received treatment with a gene therapy for DMD; and
  • appropriate dose.

• 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 timed 4-step climb ≤ 8 seconds; and
  • member has time to rise from floor < 10 seconds; 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 4-step descend (time in seconds); and
    • timed sit to stand (time in seconds); and
  • requested agent will not be used in combination with other disease-modifying therapies for DMD (e.g., exon-skipping therapies); 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
  • one of the following:
    • member is on a stable dose of corticosteroid; or
    • attestation that the member will continue to utilize chronic corticosteroids after Elevidys infusion; or
    • demonstrated contraindication to corticosteroids; and
  • member has not previously received treatment with a gene therapy for DMD; and
  • infusion will take place in a qualified treatment facility; and
  • member is not currently utilizing antisense oligonucleotides; and
  • appropriate dosing.

Evrysdi

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • one of the following:
    • copy of genetic test confirming member has 4 copies of SMN2 and one of the following:
      • member is symptomatic; or
      • member is a pre-symptomatic infant diagnosed via newborn screening; or
    • copy of genetic test confirming member has 2 or 3 copies of SMN2; and
  • genetic test confirming diagnosis of SMA; and
  • prescriber is a neurologist or consult notes from a neurologist are provided; and
  • current motor function test; and
  • if the member has previously received Zolgensma, all of the following:
    • attestation that the member has had an inadequate response to Zolgensma; and
    • pre-treatment baseline motor function test (prior to treatment with any SMA agent); and
    • pre-Zolgensma baseline motor function test (if different than pre-treatment baseline); and
    • post-Zolgensma motor function tests; 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 both of the following is required:
  • one of the following: 
    • current 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; and
  • one of the following:
    • copy of genetic test confirming member has 4 copies of SMN2 and one of the following:
      • member is symptomatic; or
      • member is a pre-symptomatic infant diagnosed via newborn screening; or
    • copy of genetic test confirming member has 2 or 3 copies of SMN2; and
  • genetic test confirming diagnosis of SMA; and
  • prescriber is a neurologist or consult notes from a neurologist are provided; and
  • current motor function test; and
  • if the member has previously received Zolgensma, all of the following:
    • attestation that the member has had an inadequate response to Zolgensma; and
    • pre-treatment baseline motor function test (prior to treatment with any SMA agent); and
    • pre-Zolgensma baseline motor function test (if different than pre-treatment baseline); and
    • post-Zolgensma motor function tests; 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 both of the following is required:
  • one of the following:
    • current 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; and
  • member does not have active viral infection, including human immunodeficiency virus [HIV] or positive serology for hepatitis B or C, or Zika virus; and 
  • member has not previously received treatment with a gene therapy for spinal muscular atrophy.