Subject: Evrysdi 4074-A SGM P2022a
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; May 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.
July 16, 2022
