ROCHE: Evrysdi – Risdiplam
Effective February 25, 2022 – 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 or compound heterozygote, AND
2) Patients who:
– are symptomatic with two or three copies of SMN2, AND
– are between 2 months and 7 months (inclusive), OR
– are aged 8 months and up to 25 years inclusive, and are non-ambulatory.
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 risdiplam treatment.
Patients will be limited to receiving one month of risdiplam per prescription at their pharmacy.
Coverage for risdiplam may be approved for 12 months as follows:
– 0.2 mg/kg/day for patients from 2 months to <2 years of age, or 0.25 mg/kg/day for patients >/=2 years of age and weighing less than 20 kg, or 5 mg/day for patients >/=2 years of age and weighing >/= 20 kg.
For continued coverage, the patient must meet the following criteria:
1) 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;
2) Patient does not require permanent invasive ventilation*.
*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.”
BIOGEN: Nusinersen – Spinraza
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.†
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
“Other patients who do not meet the expanded funding criteria may be considered in exceptional cases.””
NOVARTIS: Onasemnogene abeparvovec – Zolgensma
Alberta Health Services, hospital formulary