Emflaza™

Drug - Emflaza™ (deflazacort) [Marathon]

October 2017

Therapeutic area - Duchene Muscular Distrophy (DMD)

Initial approval criteria

Patient must be 5 years of age or older AND
Must be prescribed by a provider specializing in neurology AND
Prescriber’s specialty must be provided at time of request AND
Patient must have documentation of a confirmed diagnosis of Duchenne muscular dystrophy (DMD) AND
Patient retains meaningful voluntary motor function (e.g., patient is able to speak, manipulate objects using upper extremities, ambulate, etc.) AND
Patient should be receiving physical therapy AND
Patient has tried and failed prednisone, having experienced 1 of the following unacceptable adverse reactions directly attributable to previous therapy with prednisone:
- Patient has manifested significant behavioral changes negatively impacting function at school, day care, etc. OR
- Patient has experienced significant weight gain (e.g., crossing 2 percentiles and/or reaching 98th percentile for age and sex)

Patient retains meaningful voluntary motor function (e.g., patient is able to speak, manipulate objects using upper extremities, ambulate, etc.) AND
Patient continues to receive physical therapy AND
Patient has received benefit from therapy, which may include 1 or more of the following:
- Stability or slowing of decline in motor function
- Stability or slowing of decline in respiratory function
- Stability or slowing of decline in sequelae related to diminished strength of stabilizing musculature (e.g., scoliosis, etc.)

Dosing is based on 0.9 mg/kg/day
Patient’s most up to date weight (in kg) must be submitted at time of request
If tablets are requested, round up to the nearest possible dose.
If oral suspension is requested, round up to the nearest tenth of a milliliter (mL)

MHCP Provider Call Center 651-431-2700 or 800-366-5411