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PTC Therapeutics Receives FDA Approval for the Expansion of the EMFLAZA® (deflazacort) Labeling to Include Patients 2-5 Years of Age
SOUTH PLAINFIELD, N.J., June 7, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA)
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"SOUTH PLAINFIELD, N.J., June 7, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today 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. \n\"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.\"\nPTC 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).\nAbout EMFLAZA®\nEMFLAZA® is indicated for the treatment of Duchenne muscular dystrophy in patients two years of age and older.\nIMPORTANT SAFETY INFORMATION\nContraindication: Do not use if you are allergic to deflazacort or any of the inactive ingredients in EMFLAZA.\nDo not stop taking EMFLAZA, or change the amount you are taking, without first checking with your healthcare provider, as there may be a need for gradual dose reduction to decrease the risk of adrenal insufficiency and steroid \"withdrawal syndrome\". Acute adrenal insufficiency can occur if corticosteroids are withdrawn abruptly and can be fatal. A steroid \"withdrawal syndrome,\" seemingly unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of corticosteroids. For patients already taking corticosteroids during times of stress, the dosage may need to be increased.\nHyperglycemia: Corticosteroids can increase blood glucose, worsen pre-existing diabetes, predispose those on long-term treatment to diabetes me...