Deflazacort consists of the active ingredient Deflazacort. It is a synthetic glucocorticoid (type of steroid) medication used to treat various conditions, such as inflammation, allergies, autoimmune disorders, and certain types of cancers. This medicine works by suppressing the immune system and reducing inflammation in the body. Deflazacort was first approved by the United States Food and Drug Administration (FDA) in 2017 for treating Duchenne muscular dystrophy, a genetic disorder that causes progressive muscle weakness and wasting. It is believed to improve muscle strength and delay the progression of the disease in some patients with DMD. It has also been approved in other countries for various indications, including asthma and allergic rhinitis.

In particular, deflazacort is commonly used to treat Duchenne muscular dystrophy (DMD). It is also used to treat a range of inflammatory and autoimmune conditions. These include asthma, allergies, and inflammatory bowel disease (IBD), such as Crohn's disease and ulcerative colitis. It also treats rheumatoid arthritis, lupus (systemic lupus erythematosus), and certain skin conditions.

Deflazacort is usually taken orally in the form of tablets. The tablets should be swallowed whole with water or another liquid and not crushed, chewed, or broken. It is generally recommended to take it on an empty stomach, either one hour before or two hours after meals, to help maximize its absorption and effectiveness. Do not take a double dose to make up for a missed one.

Deflazacort, like all medications, can cause side effects. Common side effects may include nausea, vomiting, indigestion, and changes in appetite. Long-term use can also cause more serious side effects, such as osteoporosis, high blood pressure, and increased risk of infection. Other possible side effects include mood changes, difficulty sleeping, and weight gain. It is important to promptly report any unusual or severe side effects to a healthcare provider.

Deflazacort may increase the risk of infections due to its effects on the immune system. Long-term use of this medication may suppress the function of the adrenal glands, which can cause a condition called adrenal insufficiency. The symptoms include fatigue, weakness, and low blood pressure. The extended use may also increase the risk of developing cataracts, which is a clouding of the lens in the eye, and the risk of osteoporosis (weak and brittle bone). This medication may increase the risk of gastrointestinal bleeding, particularly in individuals with a history of stomach ulcers or bleeding. Long-term use may affect growth in children. This medication may cause fluid retention, leading to swelling of arms, legs, or abdomen. This can also lead to an increase in blood pressure.

It is important to have regular eye exams while taking deflazacort. You must notify your healthcare provider immediately if you experience any signs of infection, such as fever, sore throat, or chills. You should discuss with your healthcare provider the potential risks and benefits of medicine for an extended period. It is important to monitor for signs of fluid retention and to notify your healthcare provider if you experience swelling or weight gain. Monitor the growth of children who are taking this medicine. Tell your doctor immediately if you experience stomach pain, nausea, vomiting, or blood in your stool to avoid further gastrointestinal bleeding complications.

Deflazacort is a medication that should only be used under the supervision of a healthcare provider. It is important to follow the dosage and administration instructions provided by your healthcare provider and promptly report any side effects or concerns. Patients with a history of allergy to this medication or any components should avoid taking it. Pregnant and breastfeeding women should be avoided while taking this medication. Women of reproductive potential should take contraceptives during and after several weeks of treatment. 
Do not receive live or attenuated vaccines during this treatment. Tell your physician before this medication if you have high blood pressure, diabetes, weak bones, or problems in your eyes, heart,  thyroid gland, or blood vessels. Contact your physician immediately if you experience mood disturbances, severe stomach pain, or allergic reactions after taking this tablet.

1. Sanofi, Electronic Medicines Compendium (EMC), [Revised on Nov 2020] [Accessed on 29th April 2023], https://www.medicines.org.uk/emc/files/pil.6287.pdf
2. PTC Therapeutics, Inc., [Revised on Jun 2021] [Accessed on 29th April 2023], https://www.emflaza.com/prescribing-information.pdf
3. Parente, Deflazacort: therapeutic index, relative potency and equivalent doses versus other corticosteroids, BMC Pharmacology and Toxicology, 2017, 18:1, https://bmcpharmacoltoxicol.biomedcentral.com/track/pdf/10.1186/s40360-016-0111-8.pdf

The drug information on this page is not a substitute for medical advice. It is meant for educational purposes only. For further details, consult your doctor about your medical condition to know if you can receive this treatment.