Subject: Evrysdi 4074-A SGM P2023
Policy:
I. INDICATIONS
The indications below including FDA-approved indications and
compendial uses are considered a covered benefit provided that
all the approval criteria are met and the member has no
exclusions to the prescribed therapy.
FDA-Approved Indication
Evrysdi is indicated for the treatment of spinal muscular atrophy
(SMA) in pediatric and adult patients.
All other indications are considered experimental/investigational
and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate
the prior authorization review:
A. Initiation of therapy:
1. Deletion or mutation at the SMN1 allele confirmed by
genetic testing
2. Medical records (e.g., chart notes, laboratory values) of
the baseline assessment for at least one of the following
assessment tools (based on patient age and motor
ability) to establish baseline motor ability:
i. Hammersmith Infant Neurological Exam Part 2
(HINE-2)
ii. Hammersmith Functional Motor Scale Expanded
(HFMSE)
iii. Children’s Hospital of Philadelphia Infant Test of
Neuromuscular Disorders (CHOP-INTEND)
iv. Motor Function Measure 32 (MFM32)
v. Bayley Scales of Infant and Toddler Development –
Third Edition (BSID-III)
B. Continuation of therapy:
Medical records (e.g., chart notes, laboratory values) of the
most recent (less than 1 month prior to continuation
request) assessment by at least one of the following
assessments:
i. HINE-2
ii. HFMSE
iii. CHOP-INTEND
iv. MFM32
v. BSID-III
vi. For members prescribed Evrysdi due to clinical
worsening after receiving gene replacement therapy
(e.g., Zolgensma): documentation of the impact of
Evrysdi therapy (e.g., impact on motor milestones)
III. PRESCRIBER SPECIALTIES
This medication must be prescribed by or in consultation with a
physician who specializes in treatment of spinal muscular
atrophy.
IV. CRITERIA FOR INITIAL APPROVAL
Spinal Muscular Atrophy (SMA)
Authorization of 12 months may be granted for treatment of
SMA when all of the following criteria are met:
A. Member has type 1, type 2, or type 3 SMA
B. There is genetic documentation of 5q SMA homozygous
gene mutation, homozygous gene deletion, or compound
heterozygote
C. The member is 25 years of age or younger at the initiation
of treatment
D. Member is not dependent on either of the following:
1. Invasive ventilation or tracheostomy
2. Use of non-invasive ventilation beyond naps and
nighttime sleep
E. Member meets one of the following criteria:
1. Member has not previously received gene replacement
therapy for SMA (e.g., Zolgensma), or
2. Member has previously received gene replacement
therapy for SMA (e.g., Zolgensma) and has experienced
a worsening in clinical status since receiving gene
replacement therapy as demonstrated by a decline of
minimally clinical important difference from highest
score achieved or baseline on one of the following
exams (based on member age, motor ability, and
specific exam)
i. HINE-2: Decline of at least 2 points on kicking and
1 point on any other milestone (excluding
voluntary grasp)
ii. HFMSE: Decline of at least 3 points
iii. CHOP-INTEND: Decline of at least 4 points
iv. MFM32: Decline from baseline
v. BSID-III: Inability to sit without support for more
than 5 seconds per item 22 of test
F. Member will not use Evrysdi and Spinraza concomitantly
G. Member’s daily dose will not exceed the following:
1. Members less than 2 months of age: 0.15 mg/kg
2. Members 2 months to less than 2 years of age: 0.2
mg/kg
3. Members 2 years of age and older weighing less than 20
kg: 0.25 mg/kg
4. Members 2 years of age and older weighing 20 kg or
more: 5 mg
V. CONTINUATION OF THERAPY
Note: Members who were previously established on Evrysdi and
subsequently administered gene replacement therapy (e.g.,
Zolgensma) must meet all initial criteria prior to re-starting
therapy on Evrysdi.
Authorization of 12 months may be granted for continued
treatment of SMA when all of the following criteria are met:
A. Member has type 1, type 2, or type 3 SMA
B. Member is not dependent on either of the following:
1. Invasive ventilation or tracheostomy
2. Use of non-invasive ventilation beyond naps and
nighttime sleep
C. Submission of medical records (e.g., chart notes, laboratory
values) of the most recent (less than 1 month prior to
continuation request) assessment documenting a positive
clinical response from pretreatment baseline to Evrysdi
therapy, as demonstrated by at least one of the following
assessments:
1. HINE-2
i. One of the following:
a. Member exhibited improvement or maintenance
of previous improvement of at least a 2-point (or
maximal score) increase in ability to kick; or
b. Member exhibited improvement or maintenance
of previous improvement of at least a 1-point (or
maximal score) increase in any other HINE-2
milestone (e.g., head control, rolling, sitting,
crawling, standing, or walking) excluding
voluntary grasp; and
ii. One of the following:
a. Member exhibited improvement or maintenance of
previous improvement in more HINE-2 motor
milestones than worsening (net positive
improvement); or
b. Member achieved and maintained any new motor
milestones when they would otherwise be
unexpected to do so (e.g., sit or stand unassisted,
walk)
2. HFMSE
i. One of the following:
a. Member exhibited improvement or maintenance
of previous improvement of at least a 3-point
increase in score; or
b. Member has achieved and maintained any new
motor milestone from pretreatment baseline
when they would otherwise be unexpected to do
so
3. CHOP-INTEND
i. One of the following:
a. Member exhibited improvement or maintenance
of previous improvement of at least a 4-point
increase in score; or
b. Member has achieved and maintained any new
motor milestone from pretreatment baseline
when they would otherwise be unexpected to do
so
4. MFM32
i. Member has experienced an increase in their MFM32
score from baseline and that increase correlates with
a clinically significant functional improvement
5. BSID-III
i. Member exhibited the ability to sit without support
for at least 5 seconds after 12 months of treatment
6. Member was prescribed Evrysdi due to clinical worsening
after receiving gene replacement therapy (e.g.,
Zolgensma) and there is documentation of stabilization or
improvement in clinical status with Evrysdi therapy (e.g.,
impact on motor milestones).
D. Member will not use Evrysdi and Spinraza concomitantly
E. Member’s daily dose will not exceed the following:
1. Members less than 2 months of age: 0.15 mg/kg
2. Members 2 months to less than 2 years of age: 0.2
mg/kg
3. Members 2 years of age and older weighing less than 20
kg: 0.25 mg/kg
4. Members 2 years of age and older weighing 20 kg or
more: 5 mg
Place of Service:
Outpatient
The above policy is based on the following references:
- Evrysdi [package insert]. South San Francisco, CA: Genentech, Inc; October 2022.
- Arnold WD, Kassar D, Kissel JT, et al. Spinal muscular atrophy: diagnosis and management in a new therapeutic era. Muscle & Nerve. 2015;51(2):157-167.
- Burgunder JM, Schols L, Baets J, et al. EFNS guidelines for the molecular diagnosis of neurogenetic disorders: motoneuron, peripheral nerve and muscle disorders. European J Neurol. 2011;18:207-217.
- Wang CH, Finkel RS, Bertini ES, et al. Consensus statement for standard care in spinal muscular atrophy. J Child Neurol. 2007;22(8):1027-1049.
Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.
October 22, 2023
