FDA approves Emflaza to treat Duchenne muscular dystrophy

What is Emflaza? 

Emflaza (Deflazacort) is a glucocorticoid, a class of corticosteroids used as an anti-inflammatory and immunosuppressant. Deflazacort works by decreasing inflammation and reducing the activity of the immune system to treat certain conditions such as: acute interstitial nephritis, anaphylaxis, asthma, anemia, Crohn’s disease, dermatomyositis, idiopathic thrombocytopenic purpura, juvenile chronic arthritis, acute and lymphatic leukemia, malignant lymphoma, rheumatoid arthritis, nephrotic syndrome, stemic lupus erythematosus, ulcerative colitis and many others.

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy is a rare genetic condition that weakens and deteriorates the muscles. The symptom of muscle weakness usually begins around 3 to 5 years, specially boys, affecting between 3,500 and 5,000 males at birth worldwide. Duchenne muscular dystrophy occurs due to an absence of dystrophin, a protein that protects muscle cells membranes. Two thirds of cases the patients inherited it from the parents, while one third of cases are due to a new mutation that affects the protein.

Emfalza (Deflazacort) works by improving muscle strength in patients with Duchenne muscular dystrophy, and it comes tablets and oral suspension.

For more information click here.

FDA approves Xyrem (sodium oxybate) generic with a REMS Program

The FDA has approved the first generic version of Xyrem (sodium oxybate) Oral Solution, to treat cataplexy, a primary symptom of narcolepsy where patients suddenly lose muscle tone, including voluntary muscle control, while awake. and excessive daytime sleepiness in patients with narcolepsy. Approximately 70 percent of people with narcolepsy have cataplexy. Sodium oxybate is the only medication approved to treat cataplexy in patients with narcolepsy.

The use of Xyrem has been associated with serious side effects including seizures, trouble breathing, changes in alertness, coma, and death. Additionally, the active ingredient in Xyrem (and in the newly approved generic) is sodium oxybate. Sodium oxybate is the sodium salt of gamma hydroxybutyrate (GHB). GHB has not been approved for any medical use and has the potential for abuse, such as in cases of sexual assault.

Because of the potential risks associated with Xyrem, it is subject to strict safety controls on prescribing and dispensing under a program called a Risk Evaluation and Mitigation Strategy (REMS). FDA’s approval of generic sodium oxybate is subject to a REMS with strict safety controls that are comparable to those currently required for Xyrem.

Specifically, under both the Xyrem REMS and the generic sodium oxybate REMS, sodium oxybate can be prescribed only by a certified prescriber, and dispensed only to an enrolled patient by a certified pharmacy. Only a certified pharmacy that ships directly to patients can dispense sodium oxybate. Sodium oxybate will not be available in retail pharmacies.

In approving this generic version of Xyrem, the FDA is maintaining strict safety requirements for sodium oxybate, while providing patients with access to a generic medication option for narcolepsy.

For more information about the FDA’s press release click here.

To learn if Xyrem goes with your health conditions, match your meds with your conditions.