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March 19, 2021
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Emflaza improves, stabilizes progression for two forms of muscular dystrophy

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Emflaza, a corticosteroid, improved or stabilized disease progression in most patients with Duchenne muscular dystrophy or Becker muscular dystrophy who switched from prednisone, according to a press release.

The FDA approved Emflaza (deflazacort, PTC Therapeutics) in February 2017 for the treatment of Duchenne muscular dystrophy in patients aged 5 years and older. The current study analyzed patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) who switched from prednisone to Emflaza following the FDA approval of the agent. Physicians listed the desire to slow disease progression, tolerability, and caregiver or patient request as the most frequent reasons for switching from prednisone to Emflaza.

The real-world chart review involved a group of 55 participating neurologists who provided data on 92 male patients with DMD or BMD from February 2017 through December 2018. Most patients (n = 62) had DMD.

The average length of treatment was 3.3 years for prednisone and 6 months for Emflaza. The researchers reported a mean age of 6.2 years for patients with DMD and 20.1 years for BMD at the time of treatment switch from prednisone to Emflaza.

Over a 6-month average follow-up, 95% of patients with DMD and 90% of patients with BMD considered the drug "very" or "somewhat" effective at addressing their top motivations for trying the new treatment, according to the press release. The researchers reported weight gain, Cushingoid appearance, increased appetite, central obesity and fluid retention as frequent adverse events during treatment with both agents.

“[These data support] 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,” Stuart W. Peltz, PhD, chief executive officer of PTC Therapeutics, said in the release. “We are committed to providing access to clinically differentiated treatments for patients with high unmet need.”

References:

    PTC Therapeutics. PTC presents results from a real-world study of steroid switching in the treatment of patients with dystrophinopathies. Available at: https://ir.ptcbio.com/news-releases/news-release-details/ptc-presents-results-real-world-study-steroid-switching. Accessed March 19, 2021.