New Brunswick treatments

ROCHE: Evrysdi – Risdiplam

https://www2.gnb.ca/content/dam/gnb/Departments/h-s/pdf/en/NBDrugPlan/FormularyUpdates/NBDrugPlansBulletin-1078.pdf

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.

BIOGEN: Nusinersen – Spinraza

https://www2.gnb.ca/content/dam/gnb/Departments/h-s/pdf/en/NBDrugPlan/NewBrunswickDrugPlansFormulary.pdf

Available:

Initiation Criteria
1. Pre-symptomatic patients with two or three copies of the SMN2 gene, OR

2. Had the disease for <6 months, two 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 on HINE-2, CHOP INTEND, or HFMSE, OR

2. For those symptomatic at initiation: no improvement in HINE-2, CHOP INTENT, or HFMSE, OR

3. Permanent invasive ventilation required

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

NOVARTIS: Onasemnogene abeparvovec – Zolgensma

Available