says RS Anand, parent of a child with Duchenne Muscular Dystrophy on the FDA approval of Emflaza (deflazacort).
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), a rare genetic disorder that causes progressive muscle deterioration and weakness. 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. Emflaza is the first steroid to win formal FDA approval to treat DMD. Prednisone, another steroid, is frequently prescribed for the disease though it does not have official FDA approval. Emflaza is thought to have fewer side effects than Prednisone.
RS Anand, father of a child with DMD and founder of Dystrophy Annihilation Research Trust [DART is lab in India focusing on Muscular Dystrophy(MD)] says: "Corticosteroids have been in use for some time now. We have been giving Deflazacort to our son for the last 5 odd years. The benefits are great. Before that was Prednisone. This would seem like a new and improved Deflazacort. Hopefully fewer long term side effects. Great news for the DMD community."
What is DMD - Duchenne Muscular Dystrophy
According to the FDA press release 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.
For details on the clinical studies read the FDA announcement here.
Side effects of Emflaza
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.
Emflaza is marketed by Marathon Pharmaceuticals of Northbrook, Illinois.
For more on reviews of other drugs for DMD read here
Pic: Getty images
