PTC Therapeutics Receives FDA Approval for the Expansion of the Emflaza (deflazacort) Labeling to Include Patients 2-5 Years of Age

PTC Therapeutics, Inc.  announced that the U.S. Food and Drug Administration (FDA) approved the company's supplemental New Drug Application (sNDA) for Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy who are between 2- and 5-years-old. Duchenne is a rare childhood genetic disorder that causes progressive irreversible muscle deterioration and weakness. Emflaza was first approved by the FDA in February 2017 for the treatment of Duchenne in patients 5-years and older.

"We are excited to be able to bring Emflaza to younger boys living with Duchenne muscular dystrophy," said Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "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."

PTC has developed a support program, called PTC Cares™ which is dedicated to helping patients, caregivers, and prescribers understand the prescription process, and financial assistance programs for PTC products. For more information, please visit our website, www.ptccares.com or call 1-844-4PTC-CARES (1-844-478-2227).

About Emflaza

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

About Duchenne Muscular Dystrophy

Primarily affecting males, Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk as early as age ten, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. More information regarding Duchenne is available through the Muscular Dystrophy Association and the Parent Project Muscular Dystrophy.

About PTC Therapeutics, Inc.

PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

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