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PTC Presents Results from a Real-World Study of Steroid Switching in the Treatment of Patients with Dystrophinopathies
- Desire to slow disease progression and tolerability issues were the primary reasons for switching from prednisone to EMFLAZA® (deflazacort) - - Delayed
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Desire to slow disease progression and tolerability issues were the primary reasons for switching from prednisone to EMFLAZA® (deflazacort) -\n - Delayed disease progression and improved benefit-risk reported during the 6-month average follow up after switching to EMFLAZA -\n\n\nSOUTH PLAINFIELD, N.J., March 15, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to EMFLAZA® (deflazacort) following the FDA approval. The primary reason the majority of patients chose to switch to EMFLAZA was a desire to delay disease progression to improve the benefit and tolerability. During the 6-months average follow-up after switching, physician-reported outcomes were consistent with EMFLAZA addressing the primary reasons for switching. Data were presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference. \n\"We're encouraged by the clinical benefit exhibited by patients taking EMFLAZA in this real-world analysis,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. \"This data supports the results that we saw in patients on corticosteroids in the placebo arms of multiple Duchenne clinical trials and what we have heard from the Duchenne community. We are committed to providing access to clinically differentiated treatments for patients with high unmet need.\" \nThe real-world chart review was conducted between February 2017 and December 2018 by 55 neurologists who contributed data for 92 male patients with DMD or BMD that switched from prednisone to EMFLAZA. Physicians ranked desire to slow disease progression, tolerability and caregiver or patient request as the most common motivations for switching from prednisone to EMFLAZA. Of the 92 patients, 62 individuals had DMD (mean age of 6.2 years at switch) and 30 individuals had BMD (mean age of 20.1 years at switch). To the extent recorded in medical records, Clinical Global Impression (CGI) of Improvement scores were collected at the clinic visit closest to steroid switching during prednisone treatment, and the last available clinic visit during EMFLAZA treatment. The average treatment duration was 3.3 years for prednisone and 6 months for EMFLAZA prior to the c...