Wednesday, March 17, 2021

Real-world study shows steroid switching effective for dystrophinopathies

In a real-world study, individuals with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) switched from being treated with prednisone to deflazacort (Emflaza; PTC Therapeutics, South Plainfield, NJ) to delay disease progression. For those who switched who had a Clinical Global Impression (CGI) scale scores recorded, most had disease progression improve or stabilize during steroid treatment, with a shift toward improvement after switching.

"We're encouraged by the clinical benefit exhibited by patients taking Emflaza in this real-world analysis," said Stuart W. Peltz, PhD, 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."

The real-world chart review was conducted with collected data from 92 male participants with DMD or BMD who switched from prednisone to deflazacort. The most common motivationsfor switching to deflazacort was desire to slow disease progression, tolerability, or caregiver or patient request. Of the 92 participants, 62 had DMD with an average age of 6.2 years during the switch and 30 had BMD with an average age of 20.1 years. The average treatment duration was 3.3 years for prednisone and 6 months for deflazacort prior to the chart extraction. Switching was reported as "very" or "somewhat" effective at addressing primary reasons in 95% of participants with DMD and 90% of participants with BMD.

"Results from the real-world chart review presented today support the potential of Emflaza to alter the natural history of Duchenne muscular dystrophy, demonstrating its capability to slow progression of the disease and improve benefit-risk," said Dr. Susan Apkon, investigator, chief Pediatric Rehabilitation, vice-chair Department of Physical Medicine and Rehabilitation, Fischahs chair in Pediatric Rehabilitation, Children's Hospital of Colorado. "We believe that Emflaza will continue to provide DMD patients a safe and effective treatment option."

The most common adverse events found when treated with prednisone and deflazacort included weight gain, Cushingoid appearance, increased appetite, central obesity, and fluid retention.

These data were presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference.

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