SPECIAL AUTHORIZATION

For patients diagnosed with 5q Spinal Muscular Atrophy (SMA) under the care of a specialist with experience in the diagnosis and management of SMA, if the following clinical criteria are met:

 

1) Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or compound heterozygote, AND

2) Patients who:

– are pre-symptomatic with two or three copies of SMN2, OR

– have had disease duration of less than six months, two copies of SMN2, and symptom onset after the first week after birth and on or before seven months of age, OR

– are under the age of 18 with symptom onset after six months of age, regardless of the ability to walk independently.

AND

3) Patient is not currently requiring permanent invasive ventilation*, AND

4) A baseline assessment using an age-appropriate scale (the Hammersmith Infant Neurological Examination [HINE] Section 2, Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders [CHOP INTEND], or Hammersmith Functional Motor Scale-Expanded [HFMSE]) must be completed prior to initiation of nusinersen treatment.

 

Other patients who do not meet the expanded funding criteria may be considered in exceptional cases.

 

Initial coverage may be approved for three 12 mg doses at Day 0, Day 14 and Day 28, followed by one 12 mg dose at Day 63.

 

Patients will be limited to receiving one dose of nusinersen per prescription at their pharmacy.

 

For continued coverage, the patient must meet the following criteria:

1) There is demonstrated achievement or maintenance of motor milestone function (as assessed using age-appropriate scales: the [HINE] Section 2), CHOP INTEND, or HFMSE) since treatment initiation in patients who were pre-symptomatic at the time of treatment initiation; OR

There is demonstrated maintenance of motor milestone function (as assessed using age-appropriate scales: the HINE Section 2, CHOP INTEND, or HFMSE) since treatment initiation in patients who were symptomatic at the time of treatment initiation;

AND

2) Patient does not require permanent invasive ventilation*.

 

Continued coverage may be considered for one 12 mg maintenance dose at a time, to be administered at 4-month intervals.

Each maintenance dose cannot be considered prior to 4 months elapsing from the date of the previous dose.

Treatment should be discontinued if, prior to the fifth dose or every subsequent dose of nusinersen, the above renewal criteria are not met.

 

*Permanent invasive ventilation is defined as the use of tracheostomy and a ventilator due to progression of SMA that is not due to an identifiable and reversible cause.

 

SMA drug therapy and adeno-associated virus (AAV) vector-based gene therapy may not be used concomitantly. Additionally, use of a SMA drug therapy after administration of an AAV vector-based gene therapy will not be permitted, and coverage will not be approved when any SMA drug therapies are to be used in combination.

 

Patients currently receiving SMA drug therapy may be eligible to switch to an alternate SMA drug therapy; however, patients will not be permitted to switch back to a previously trialed SMA drug.”

 

All requests (including renewal requests) for nusinersen must be completed using the Nusinersen/Risdiplam Special Authorization Request Form (ABC 60064).

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Available: