PTC Therapeutics Shares Spike After FDA Approves Drug For Younger Patients

PTC Therapeutics, Inc. PTCT announced Friday that the Food and Drug Administration approved a label expansion for the drug Emflaza to include younger patients with Duchenne muscular dystrophy who are between 2 and 5 years old. 

PTC Therapeutics shares were trading higher by 2.73 percent to $41.40 at the time of publication Friday. 

Duchenne muscular dystrophy is a rare childhood genetic disorder that causes progressive, irreversible muscle deterioration and weakness.  

Duchenne is a severe type of muscular dystrophy, and symptoms of muscle weakness usually begin around the age of 4 in boys and worsen quickly; females with a single copy of the defective gene may show mild symptoms.

Emflaza was first approved by the FDA in February 2017 for the treatment of Duchenne in patients 5 and older. 

"We are excited to be able to bring Emflaza to younger boys living with Duchenne muscular dystrophy," PTC Therapeutics CEO Stuart Peltz said in a statement. 

"The standard of care is to start Emflaza at the time of diagnosis. We believe that treating patients as young as possible, when they still have a substantial amount of muscle, will have the greatest benefit for patients that are two years and older." 

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