Emflaza Supply Not Affected by COVID-19 Pandemic, PTC Therapeutics Says

Emflaza Supply Not Affected by COVID-19 Pandemic, PTC Therapeutics Says
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The supply of Emflaza (deflazacort), a treatment for people with Duchenne muscular dystrophy (DMD), will not be affected by the COVID-19 pandemic, according to PTC Therapeutics, its developer.

In a statement addressed to patients and health care professionals, released March 16, PTC said it has more than year’s supply of Emflaza in stock and available for delivery.

COVID-19, which stands for COrona VIrus Disease 2019, is an infectious disease caused by a coronavirus. The virus is thought to spread mainly from person-to-person in small droplets of moisture that are produced by sneezing or coughing. Yet, a person also may become infected by touching a surface or object that has the virus and then touching the mouth, nose, or eyes.

The major symptoms of COVID-19 include fever, dry cough, and difficulty breathing, which may appear 2-14 days after exposure to the virus.

PTC said it is carefully monitoring any shipping problems that may occur in the U.S. that would influence treatment delivery. The company added that its main priority is to continue delivery of the treatments, which are key for patients and, in turn, their caregivers. Updates will be provided if any issues should develop.

Should patients have any questions or concerns, they can contact PTC Cares at 1-844-PTC-CARES (844-478-2227).

Originally developed by Marathon Pharmaceuticals, Emflaza contains a corticosteroid precursor that suppresses immune responses and inflammation once metabolized to its active form. The therapy is available as a tablet and as an oral suspension.

Emflaza was originally approved by the U.S. Food and Drug Administration (FDA) in 2017 as a DMD treatment for patients age 5 and older regardless of disease-causing genetic mutation. In 2019, the FDA extended this indication to include children as young as age 2.

Regulatory approval in the U.S. was based on the results of several studies, including a one-year Phase 3 trial with 196 boys, ages 5 to 15. Muscle strength increased throughout treatment in those receiving Emflaza, with assessments of motor function also showing the therapy’s superiority over a placebo.

In addition to its use against DMD, Emflaza is being studied in an international Phase 3 trial (NCT03783923) in people with limb-girdle muscular dystrophy 2I (LGMD2I). This study is currently recruiting around 100 participants to test Emflaza, first in comparison with a placebo for 26 weeks and then in an open-label phase (no placebo) for another 26 weeks. The results are expected in August 2020.

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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