Duchenne Muscular
Dystrophy
1)
Diagnosis of DMD with
mutation amenable to exon 53 skipping confirmed by genetic testing;
2)
Prescribed by or in
consultation with a neurologist;
3)
Age ≤ 9 years at therapy
initiation;
4)
4. Member has all of the
following assessed within the last 30 days (a, b, and c):
a.
Ambulatory function
(e.g., ability to walk with or without assistive devices, not wheelchair
dependent) with one of the following (i or ii):
i.
6-minute walk test (6MWT)
distance ≥ 201 m;
ii.
Time-to-stand (TTSTAND) <
10 seconds;
b.
Stable cardiac function
with left ventricular ejection fraction (LVEF) ≥ 40%;
c.
Stable pulmonary function
with predicted forced vital capacity (FVC) ≥ 50%;
5)
Inadequate response (as
evidenced by a significant decline in 6MWT, TTSTAND, LVEF, or FVC) despite
adherent use of an oral corticosteroid (e.g., prednisone, Emflaza™) for ≥ 6
months, unless contraindicated or clinically significant adverse effects are
experienced; *Prior authorization is required for Emflaza
6)
Viltepso is prescribed
concurrently with an oral corticosteroid, unless contraindicated or clinically
significant adverse effects are experienced;
7)
Viltepso is not
prescribed concurrently with other exon-skipping therapies (e.g., Exondys 51®,
Vyondys 53™);
8)
Dose does not exceed 80
mg/kg per week.
Approval duration: 6
months
Reauthorization Criteria
1)
Currently receiving
medication for DMD with mutation amenable to exon 53 skipping or member has
previously met initial approval criteria;
2)
Member is responding
positively to therapy as evidenced by one of the following (a or b):
a.
All of the following
assessed within the last 6 months (i, ii, and iii):
i.
Ambulatory function
(e.g., ability to walk with or without assistive devices, not wheelchair
dependent) with one of the following (1 or 2): 1) 6MWT distance ≥ 201 m; 2)
TTSTAND < 10 seconds;
ii.
Stable cardiac function
with LVEF ≥ 40%;
iii.
Stable pulmonary function
with predicted FVC ≥ 50%;
b.
Member has received this
medication via a healthcare insurer without meeting the requirements above (see
criterion 2a), and medical record shows improved or stable LVEF and FVC,
assessed within the last 6 months;
3)
Member has been assessed
by a neurologist within the last 6 months;
4)
Viltepso is prescribed
concurrently with an oral corticosteroid, unless contraindicated or clinically
significant adverse effects are experienced;
5)
Viltepso is not
prescribed concurrently with other exon-skipping therapies (e.g., Exondys 51,
Vyondys 53);
6)
If request is for a dose
increase, new dose does not exceed 80 mg/kg per week.
Approval duration: 6
months