New Brunswick
Treatments

Evrysdi – Risdiplam

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Available:

Effective May 24- , 2022 – According to Bulletin 1008, EVRYSDIⓇ (risdiplam) will be covered through the New Brunswick Drug Formulary. For the treatment of 5q spinal muscular atrophy (SMA), if the following criteria are met:
• Genetic documentation of 5q SMA homozygous gene deletion, or compound heterozygous mutation; and
• Patient is not requiring permanent invasive ventilation; and
• Patient who is symptomatic with two or three copies of the SMN2 gene and is:
– 2 months to 7 months of age, or
– 8 months to 25 years of age and non-ambulatory.

Discontinuation Criteria:
• There is failure to demonstrate maintenance in motor milestone function as assessed using
age-appropriate scales since treatment initiation; or
• permanent invasive ventilation is required Clinical Notes: 1. An age-appropriate scale is defined as 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). New Brunswick Drug Plans 5 May 2022 2. A baseline assessment using an age-appropriate scale must be completed prior to initiation of treatment. 3. Yearly assessments must be completed using an age-appropriate scale no more than 12 weeks prior to the renewal date. 4. 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.

Spinraza – Nusinersen

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Available:

Pediatric:

Initiation Criteria
1. Pre-symptomatic patients with 2 or 3 copies of the SMN2 gene,
OR
2. Had the disease for <6 months, 2 copies of SMN2, and symptom onset after the first week after birth and on, or before seven months of age.
OR
3. Patients under the age of 18 with symptom onset > 6 months of age, and never achieved the ability to walk independently. †

Stopping Criteria
1. For those pre-symptomatic at initiation: no improvement/maintenance on HINE-2, CHOP INTEND, or HFMSE, OR
2. For those symptomatic at initiation: no maintenance in HINE-2, CHOP INTENT, or HFMSE, OR
3. Permanent invasive ventilation required

Adult:

“Requests for patients who do not meet the above criteria may be considered on a case-by-case basis.”

Zolgensma – Onasemnogene abeparvovec

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Available:

  • Genetic documentation of 5q SMA with biallelic mutations in the survival motor neuron 1 (SMN1) gene; and
  • Patient is 180 days of age or younger at the time onasemnogene abeparvovec is administered; and
  • Patient is pre-symptomatic or symptomatic with one to three copies of the survival motor neuron 2 (SMN2) gene; and
  • Patient does not require permanent ventilatory support (invasive or non-invasive) or a permanent feeding tube.

Clinical Note:

  • Permanent ventilatory support is defined as the need for a tracheostomy or requirement of 16 hours or more of respiratory assistance per day (via non-invasive ventilatory support) for 14 or more consecutive days in the absence of an acute reversible illness excluding perioperative ventilation.

Claim Notes:

  • The patient must be under the care of a specialist experienced in the diagnosis and treatment of SMA.
  • No treatment with nusinersen, risdiplam or other medications indicated for the treatment of SMA will be considered after the patient has received a dose of onasemnogene abeparvovec.
  • Approvals will be limited to one lifetime administraton of 1.1 x 1014 vector genomes/kg.
  • Patients who have received a prior dose of onasemnogene abeparvovec accessed by any mechanism (e.g. private insurance plan, clinical trial, compassionate access) will not be funded.
  • Patients with 4 or more copies of the SMN2 gene will not be funded.