Evrysdi (risdiplam) is considered medically necessary for members meeting the
following criteria:
Spinal Muscular Atrophy
(SMA)
1)
Diagnosis of SMA with
documentation of both of the following (a and b):
a.
Genetic testing
quantifying number of copies of SMN2 gene ≥ 1 but ≤ 4;
b.
Member is symptomatic;
2)
Genetic testing confirms
the presence of one of the following (a, b, or c):
a.
Homozygous deletions of
SMN1 gene (e.g., absence of the SMN1 gene);
b.
Homozygous mutation in
the SMN1 gene (e.g., biallelic mutations of exon 7);
c.
Compound heterozygous
mutation in the SMN1 gene [e.g., deletion of SMN1 exon 7 (allele 1) and mutation
of SMN1 (allele 2)];
3)
Prescribed by or in
consultation with a neurologist;
4)
Age ≥ 2 months;
5)
Documentation of one of
the following baseline scores (a or b):
a. For age < 2 years:
Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorder
(CHOP-INTEND) score or Hammersmith Infant Neurological Examination (HINE)
Section 2 motor milestone score;
b. For age ≥ 2 years:
Hammersmith functional motor scale expanded (HFMSE) score, Revised Hammersmith
Scale (RHS), Upper Limb Module (ULM), Revised Upper Limb Module (RULM), or
6-Minute Walk Test (6MWT);
6)
Member does not require tracheostomy or invasive ventilation;
7)
Evrysdi is not prescribed concurrently with Spinraza® and/or Zolgensma®;
8)
If the member is currently on Spinraza, documentation of prescriber attestation
of Spinraza discontinuation;
9)
If the member has a history of treatment with Zolgensma, must meet both of the
following (a and b):
a. Provider must submit
evidence of poor response to Zolgensma (e.g., sustained decrease in CHOP-INTEND
score over a period of 6 months);
b. Documentation of
prescriber attestation of clinical deterioration;
10)
Request meets one of the following (a, b, or c):
a. If 2 months of age to
less than 2 years of age, dose does not exceed 0.2 mg/kg per day;
b. If 2 years of age and
older, weighing less than 20 kg, dose does not exceed 0.25 mg/kg per day;
c. If 2 years of age and
older, weighing 20 kg or more, dose does not exceed 5 mg per day.
Approval
duration: 6 months
Reauthorization (12
months duration)
1)
Member does not require
tracheostomy or invasive ventilation;
2)
Member is responding
positively to therapy as evidenced by one of the following (a, b, or c):
a.
For age < 2 years, must
meet one of the following (i or ii):
i.
For CHOP-INTEND, must
demonstrate score improvement or maintenance of previous score improvement of ≥
4 points from baseline;
ii.
For HINE motor milestone
score, must demonstrate score improvement or maintenance of previous improvement
in one or more categories AND improvement in more motor milestone categories
than worsening;
b.
For age ≥ 2 years, must
meet one of the following (i, ii, or iii):
i.
If first renewal since
turning 2 years old, must provide submission of baseline HFMSE score, RHS score,
RULM or ULM score, or 6MWT distance AND meet one of the following (1 or 2):
1.
For CHOP-INTEND, must
demonstrate score improvement or maintenance of previous score improvement of ≥
4 points from baseline;
2.
For HINE motor milestone
score, must demonstrate score improvement or maintenance of previous improvement
in one or more categories AND improvement in more motor milestone categories
than worsening;
ii.
If ≤ 2 years at therapy
initiation and request is for subsequent renewal since turning 2, must meet one
of the following (see Appendix D) (1 or 2):
1.
For HFMSE, RHS, ULM or
RULM, must demonstrate score improvement or maintenance of previous score
improvement from baseline score submitted at first renewal since turning 2 years
old;
2.
For 6MWT distance, must
demonstrate improvement or maintenance of baseline distance;
iii.
If > 2 years at therapy
initiation, must meet one of the following (1, 2, 3, or 4):
1.
For HFMSE or RHS, must
demonstrate score improvement or maintenance of previous score improvement of ≥
3 points from baseline;
2.
For ULM, must demonstrate
score improvement or maintenance of previous improvements in ≥ 2 points from
baseline;
3.
For RULM, must
demonstrate score improvement or maintenance of previous improvements in ≥ 4
points from baseline;
4.
For 6MWT distance, must
demonstrate improvement or maintenance of baseline distance;
c.
Member has not had a
decline in motor function test score(s) from baseline AND medical justification
demonstrates and supports that member is responding positively to therapy;
3)
Evrysdi is not prescribed
concurrently with Spinraza and/or Zolgensma;
4)
If request is for a dose
increase, request meets one of the following (a, b, or c):
a.
If 2 months of age to
less than 2 years of age, new dose does not exceed 0.2 mg/kg per day;
b.
If 2 years of age and
older, weighing less than 20 kg, new dose does not exceed 0.25 mg/kg per day;
c.
If 2 years of age and
older, weighing 20 kg or more, new dose does not exceed 5 mg per day