FDA Approves EMFLAZA™ (deflazacort) for the Treatment of Duchenne Muscular Dystrophy

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FDA Approves EMFLAZA™ (deflazacort) Tablets and Oral Suspension for the
Treatment of Duchenne Muscular Dystrophy in Patients 5 Years and Older

 

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a rare, genetic disease caused by mutations in the dystrophin gene. Without dystrophin, a structural protein, muscles are unable to operate properly and suffer progressive damage.4 Duchenne affects mainly boys and young men, with an incidence of approximately 1 in 5,000 live male births.5 It typically results in the inability to walk by the teen years or earlier, and severe respiratory and cardiac complications that lead to death generally between the late teens and early twenties.6,7,8  The approval of EMFLAZA™ (deflazacort) marks the first time that an FDA-approved treatment is available for patients with all genetic forms of Duchenne

 

About the Research

The effectiveness of EMFLAZA for the treatment of Duchenne was established in a multicenter, randomized, double-blind, placebo-controlled, 52-week study conducted in the United States and Canada.2,3 The study population consisted of 196 male pediatric patients 5 to 15 years of age who were ambulatory and non-ambulatory, one of the largest clinical studies ever conducted in this patient population.

Patients were randomized to therapy with deflazacort (0.9 or 1.2 mg/kg/day), an active comparator or placebo.2 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.

About EMFLAZATM (deflazacort)

EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older.

EMFLAZA is not for patients who are allergic to deflazacort or any of the inactive ingredients in EMFLAZA.

Patients should not stop taking EMFLAZA, or change the amount they are taking, without first checking with their healthcare provider, as there may be a need for gradual dose reduction to decrease the risk of serious side effects.

Corticosteroids, such as EMFLAZA can cause:

· Hyperglycemia, altered glucose metabolism
· Increased risk of infection
· Changes in cardiovascular/kidney function that could lead to increases in blood pressure, salt, and water retention, and decreases in blood levels of potassium and calcium
· Behavioral and mood changes that could lead to potentially severe psychiatric adverse reactions
· Osteoporosis, decrease in bone mineral density
· Serious skin rashes
· May slow growth and development
· Cataracts or glaucoma

Vaccinations:  The administration of live or live attenuated vaccines is not recommended while taking EMFLAZA.  Killed or inactivated vaccines may be administered, but the responses cannot be predicted.  Patients should discuss their vaccine history with their healthcare provider before starting EMFLAZA, and while taking EMFLAZA, patients should check with their healthcare provider before receiving any new vaccines or booster shots.

Common side effects that could occur with EMFLAZA include: Facial puffiness or Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, frequent daytime urination, unwanted hair growth, central obesity, and colds.

 

About Marathon Pharmaceuticals

Marathon Pharmaceuticals, LLC is an American biopharmaceutical company that develops treatments for rare diseases, with a focus on patients who currently have no treatment options. The company’s pipeline of new medicines includes treatments for rare neurological and movement disorders. Marathon is headquartered in Northbrook, Illinois, with offices in Chicago, Cambridge, Mass., New Jersey and Washington D.C. and manufacturing in North Carolina. For more information

 

Reference: http://marathonpharma.com

 

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