Doctors Listened and Duchenne Muscular Dystrophy Patients Benefited

Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019 to treat patients between two and five years of age.

According to a recent report from Biospace, PTC Therapeutics Inc. (PTCI) announced the results of its real-world-study of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) patients who had switched from prednisone to Emflaza after the FDA approved the agent.

In the past, drug developers have relied on randomized clinical trials for approval of their products. In comparison, real-world data comes from sources other than clinical trials and is gaining importance in healthcare decisions.

About DMD and BMD

DMD, a genetic disorder that usually begins in childhood, is the cause of irreversible muscle weakness and deterioration.

BMD is less common than DMD. It exhibits the same symptoms as DMD yet it is not as severe.

DMD and BMD are both X-linked recessive disorders caused by a mutation in genes on the X chromosome.

How Did We Get Here

Physicians listed several motivations that prompted them to recommend switching from prednisone to Emflaza. The primary reason was to slow the progression of the disease, followed by tolerability of the drug, and in general the frequent requests from patients and caregivers.

Results of the Study

The average duration of treatment was three years for prednisone and six months for EMFLAZA. Fifty-five neurologists participated in the real-world chart review from February 2017 to December 2018.

A total of ninety-two patients participated in the study. Of this total, sixty-two patients had DMD and thirty patients had BMD.

Global Impression of Improvement scores were recorded at the point where steroids were switched during treatment with prednisone. Results for treatment with EMFLAZA were collected after the patient’s last clinic visit.

Treatment duration lasted six months for EMFLAZA versus a three-year period for prednisone. During the period of steroid treatment, disease progression generally stabilized or showed some improvement.

During the six-month follow-up, the charts registered “somewhat” or “very” in satisfying the reasons for switching to EMFLAZA.

Adverse Events

The most common adverse events during either EMFLAZA or prednisone treatment were weight gain and fatty tissue deposits in the back and midsection (Cushingoid), and retention of fluid.

Dr. Susan Apkon of Colorado’s Children’s Hospital commented that Real-World Chart results confirm EMFLAZA’s potential to slow the progression of DMD while also improving the benefit-risk.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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