CureDuchenne To Host Webinar With Marathon Pharmaceuticals on November 1 to Update the Duchenne Community on Deflazacort Access and Regulatory Status
NEWPORT BEACH, CA. October 25, 2016 – CureDuchenne, a nonprofit dedicated to funding research and improving patient care for Duchenne muscular dystrophy, will host an informational webinar on November 1, 2016 at 2:00 p.m. ET/11:00 a.m. PT to update the community on Marathon Pharmaceutical’s efforts to secure FDA approval for deflazacort.
If deflazacort is approved by the FDA, it is anticipated it will be the second treatment approved for Duchenne in the United States and the first indicated for all Duchenne patients. This webinar will highlight the regulatory process to date, update the community on Marathon Pharmaceuticals’ Expanded Access Program (EAP) for deflazacort and discuss what a potential FDA approval of deflazacort would mean in terms of access to the drug in the United States. Some of those who currently import versions of deflazacort from abroad may have recently received an update from their distributor with initial details; this will be discussed along with steps current deflazacort patients might choose to take to help ensure access to U.S. supply of deflazacort if FDA approval is granted.
The webinar will be presented by Timothy M. Cunniff, Pharm.D., Executive Vice President of Research & Development, Marathon Pharmaceuticals; Scott Wilhoit, Vice President, Patient Access, Marathon Pharmaceuticals; and moderated by Jak Knowles, MD, VP, Medical and Scientific Affairs, CureDuchenne.
Marathon recently submitted New Drug Applications (NDAs) to the FDA for deflazacort (tablet form and oral suspension). The NDAs were accepted for filing in early August, and FDA expects to complete its review of the applications in February 2017.
To register for the webinar on November 1, click here. The call in number is (844) 337-8088 and the conference ID number is 92835991.
The webinar will include an opportunity for Marathon to address questions from the community. Questions may be submitted to ancia@CureDuchenne.org by October 31.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a fatal genetic disease that causes muscle degeneration and affects 1 in 3,500 boys. Boys with Duchenne are usually diagnosed by age 5, lose their ability to walk by age 12 and most don’t survive their mid-20s.
CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 children and young adults worldwide. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne also brings physical therapy and standard of care to local communities around the country through its CureDuchenne Cares program. For more information, please visit CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.
Deflazacort, an investigational drug, is a glucocorticoid with anti-inflammatory and immunosuppressant properties.1 Deflazacort is currently not approved in the United States for any indication. Versions of deflazacort are available in some countries outside the United States where it is approved for a number of indications, but not for Duchenne. If approved, deflazacort will be among the first commercially available treatments indicated for Duchenne in the United States.
Based on data contained in the NDAs and in published clinical studies, it appears that deflazacort may be an important treatment option for patients with Duchenne, if approved by the FDA.
Side effects that could occur with Deflazacort use include:
Facial puffiness or Cushingoid appearance, skin redness, unwanted hair growth, weight gain, central obesity, headache and increased appetite.
Other important side effects include: a decrease in the density of the bones (osteopenia) or fragility of the bones (osteoporosis) which may lead to fractures, acne, stomach upset or irritation to the stomach lining, cataracts (which can impair vision), increased susceptibility to infection, sugar intolerance and aggravation of diabetes, elevation in blood pressure, behavioral and mood changes, effects on growth and development such as short stature.
Deflazacort use is not recommended for patients who have a systemic fungal infection or are allergic to deflazacort or any of the inactive ingredients in deflazacort, have had recent or ongoing infections or have recently received a vaccine or are scheduled for a vaccination.
About Marathon Pharmaceuticals
Marathon Pharmaceuticals, LLC, is a biopharmaceutical company that develops treatments for rare diseases, with a focus on patients who currently have no treatment options. The company’s pipeline of new medicines includes treatments for rare neurological and movement disorders. Marathon is headquartered in Northbrook, Illinois, with offices in Chicago, New Jersey, Cambridge, MA and Washington D.C.
1 Wong BL, Christopher C. Corticosteroids in Duchenne muscular dystrophy: a reappraisal. Journal of Child Neurol, 2002;17(3):183–9.