Emflaza Receives Expanded Indication
June 7, 2019 – The U.S. FDA has approved an expanded indication for Emflaza® (deflazacort), manufactured by PTC Therapeutics. The drug, which was originally approved in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years of age and older, may now be used in patients as young as two years of age. It is the only FDA-approved treatment for DMD in children as young as two.
DMD is one of nine forms of muscular dystrophy. A severe but rare genetic disorder, it leads to progressive muscle wasting and weakness. Symptom onset typically occurs between the ages of three to five years. Early signs may include delayed development of the ability to sit, stand, or walk, as well as trouble learning how to speak. While the disease most frequently affects male children, it can occur in female children as well. The majority of children who have DMD require a wheelchair full time by 13 years of age, and begin to experience life-threatening heart and respiratory complications by the time they are teenagers.
According to a manufacturer press release, treating children for DMD at a younger age when they have more muscle may provide greater treatment benefits. Emflaza is available in 6mg, 18mg, 30mg, and 36mg tablets (which can be crushed and mixed with applesauce, or taken whole), as well as a 22.75mg/mL oral suspension. The recommended dosing is based on weight.