Archives     Advertise     Editorial Calendar     Subscribe     Contact Us    


FDA Approves Drug to Treat Duchenne Muscular Dystrophy


 

On Feb. 9, the U.S. Food and Drug Administration approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD). Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system. Corticosteroids are commonly used to treat DMD across the world. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States.

"This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy," said Billy Dunn, MD, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. "We hope that this treatment option will benefit many patients with DMD."

DMD is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between 3 and 5 years of age and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one of every 3,600 male infants worldwide.

People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

The effectiveness of deflazacort was shown in a clinical study of 196 male patients who were 5 to 15 years old at the beginning of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5. At week 12, patients taking deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared to those taking a placebo. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated patients. In another trial with 29 male patients that lasted 104 weeks, deflazacort demonstrated a numerical advantage over placebo on an assessment of average muscle strength. In addition, although not statistically controlled for multiple comparisons, patients on deflazacort appeared to lose the ability to walk later than those treated with placebo.

The side effects caused by Emflaza are similar to those experienced with other corticosteroids. The most common side effects include facial puffiness (Cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism) and excessive fat around the stomach (central obesity).

Other side effects that are less common include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones and vision problems such as cataracts. Patients receiving immunosuppressive doses of corticosteroids should not be given live or live attenuated vaccines.

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The sponsor is receiving a rare pediatric disease priority review voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive priority review of a subsequent marketing application for a different product. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began.

Emflaza is marketed by Marathon Pharmaceuticals of Northbrook, Illinois.

 
Share:

Related Articles:


Recent Articles

Study Shows ICU Patients with Low-Risk Penicillin Allergies Can Be Tested and Treated

Read More

Cummunity Comes Together Archives

Read More

AHA, AMA & ANA SEND JOINT LETTER TO CONGRESS ON CORONAVIRUS EMERGENCY FUNDING

Read More

Scope of Practice Update

Read More

AHIP Statement on Final CMS and ONC Interoper-ability Rules

Matt Eyles, president and CEO of America's Health Insurance Plans (AHIP), issued this statement following the Centers for Medicare & Medicaid Services' (CMS) final rule on Interoperability and Patient Access, and the Office of the National Coordinator (ONC) for Health Information Technology final rule on Interoperability, Information Blocking, and the ONC Health IT Certification Program:

Read More

Alexander Statement on Final Electronic Health Records Rules

U.S. Senate health committee Chairman Lamar Alexander (R-Tenn.) released the following statement after the Trump Administration released two final electronic health record rules:

Read More

A New Era in Nephrology

New therapies are beginning to come online to change outcomes for kidney disease patients.

Read More

Solving the Rural Health Puzzle

Hospital closures, alternative payment models, workforce issues and an older, sicker population have combined to create huge challenges in delivering rural care.

Read More

The Kidney Project: Pioneering Hope

The Kidney Project is bringing new hope to patients with end stage renal disease.

Read More

Saint Thomas, Nephrology Associates Partner on Transplant

Ascension Saint Thomas and Nephrology Associates recently announced an exclusive partnership to expand kidney transplant care.

Read More

Email Print
 
 

 

 


Tags:
Corticosteroid, Deflazacort, DMD, Duchenne Muscular Dystrophy, Emflaza, FDA, Food & Drug Administration
Powered by Bondware
News Publishing Software

The browser you are using is outdated!

You may not be getting all you can out of your browsing experience
and may be open to security risks!

Consider upgrading to the latest version of your browser or choose on below: