Initial Approval Criteria
A. Spinal Muscular Atrophy
(must meet all):
*Only for initial
treatment dose; subsequent doses will not be covered.
1. Diagnosis of SMA Type
I with onset of symptoms prior to 6 months of age;
2. Genetic testing
confirming 1, 2, or 3 copies of SMN2 gene;
3. 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));
4. Prescribed by or in
consultation with a neurologist;
5. Age < 2 years;
6. Documentation of one
of the following baseline scores (a
or b):
a.
Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorder
(CHOP-INTEND) score;
b.
Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestone
score;
7. Documentation of both
of the following (a and b):
a. Baseline laboratory
tests demonstrating Anti-AAV9 antibody titers ≤ 1:50 as determined by ELISA
binding immunoassay;
b. Baseline liver
function test, platelet counts, and troponin-I;
8. Member does not have
advanced SMA (e.g., complete paralysis of limbs, ventilator dependence for 16 or
more hours per day, tracheostomy, or non-invasive ventilation beyond the use for
sleep);
9. Member has not been
previously treated with Zolgensma;
10. Zolgensma is not
prescribed concurrently with Spinraza® or Evrysdi™;
11. If the member is
currently on Spinraza, must meet the following (a and b):
a. Provider must submit
evidence of clinical deterioration (e.g., sustained decrease in CHOP-INTEND
score over a period of 3 to 6 months) upon completion of all loading doses of
Spinraza;
b. Documentation of
provider attestation of clinical deterioration and Spinraza discontinuation;
12. If the member is
currently on Evrysdi, must meet the following (a and b):
a. Provider must submit
evidence of clinical deterioration (e.g., sustained decrease in CHOP-INTEND
score over a period of 3 to 6 months);
b. Documentation of
provider attestation of clinical deterioration and Evrysdi discontinuation;
13.
Member does not
have an active viral infection;
14. Total dose does not
exceed 1.1 x 1014
vector genomes (vg) per kilogram (kg).
Approval duration: 4 weeks (one time infusion per lifetime)
Continued Therapy
A. Spinal Muscular
Atrophy
1. Re-authorization is
not permitted.
Approval duration: Not applicable
Codes
Used In This BI:
J3399 – injection, onasemnogene abeparvovec-xioi, per treatment, up to 5x10^15
vector genomes