Drugs

FDA Approves Emflaza Duchenne Drug

FDA has approved Marathon Pharmaceuticals’ Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 and older with Duchenne muscular dystrophy (DMD). An agency news release says that Emflaza is a corticosteroid that works by decreasing inflammation and reducing immune system activity. Although 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 U.S.

The drug’s effectiveness was shown in a clinical study of 196 male patients aged 5 to 15 at the beginning of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5. FDA says that 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 deflazacort-treated patients, the release says. In another trial with 28 male patients that lasted 104 weeks, deflazacort demonstrated a numerical advantage over placebo on an assessment of average muscle strength.

Side effects caused by Emflaza are similar to those experienced with other corticosteroids, the agency says. The most common include facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary tract frequency, unwanted hair growth, and excessive fat around the stomach.

Marathon’s application for Emflaza received fast track designation, priority review, and orphan drug designation. The company is receiving a rare pediatric disease priority review voucher under the program intended to encourage development of new drugs and biologics for preventing and treating rare pediatric diseases.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome