Company: Marathon Pharmaceuticals
Approval Status: Approved February 2017
Specific Treatments: Duchenne muscular dystrophy
Emflaza (deflazacort) is a corticosteroid which exerts anti-inflammatory and immunosuppressive effects.
Emflaza is specifically indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older.
Emflaza is supplied as a tablet or oral suspension. The recommended dose is approximately 0.9 mg/kg/day once daily. If tablets are used, round up to the nearest possible dose. Any combination of the four Emflaza tablet strengths (6 mg, 18 mg, 30 mg, and 36 mg) can be used to achieve this dose. If the oral suspension is used, round up to the nearest tenth of a milliliter (mL). Both the tablets and oral suspension can be taken with or without food
The FDA approval of Emflaza was based on a multicenter, randomized, double-blind, placebo-controlled, 52-week study conducted in the United States and Canada. The study population consisted of 196 male pediatric patients 5 to 15 years of age who were ambulatory and non-ambulatory. The patients were randomized to therapy with deflazacort (0.9 or 1.2 mg/kg/day), an active comparator or placebo. After 12 weeks, placebo patients were re-randomized to receive either deflazacort or the active comparator for an additional 40 weeks. A comparison to placebo was made after 12 weeks of treatment. Efficacy was evaluated by assessing the change between Baseline and Week 12 in average strength of 18 muscle groups. The change in average muscle strength score between Baseline and Week 12 was significantly greater for the deflazacort 0.9 mg/kg/day dose group, the recommended dose, than for the placebo group (p=0.017). Increases in muscle strength continued through the 52 week duration of the study demonstrating persistence of the treatment effect observed at Week 12. Results on several timed measures of patient function (i.e., time to stand from supine, time to climb four stairs and time to walk or run 30 feet) also numerically favored deflazacort 0.9 mg/kg/day at Week 12, in comparison with placebo.
Adverse effects associated with the use of Emflaza may include, but are not limited to, the following:
- Cushingoid appearance
- weight increased
- increased appetite
- upper respiratory tract infection
- central obesity
Mechanism of Action
Emflaza (deflazacort) is a corticosteroid prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.
For additional information regarding Emflaza or Duchenne muscular dystrophy, please visit https://emflaza.com/