FDA Approves Emflaza as Drug for Treating Muscular Dystrophy
The US Food and Drug Administration (FDA) has approved the use of deflazacort, marketed under the brand name Emflaza, for the treatment of Duchenne muscular dystrophy (DMD) in children. The steroid drug is manufactured by Marathon Pharmaceuticals and is the first to be so approved by the FDA to treat people with the condition in the United States.
Muscle dystrophy is a muscle-wasting disease that affects boys aged three and above more than it affects girls. The FDA says it is a rare genetic disease that affects one in 3,600 male infants born worldwide, and it affects only about 15,000 boys in the US. It creates mobility problems in affected male children and confines them to a wheelchair until they pass on at around age 20. Patients also suffer from heart and breathing difficulties and the childhood disease is very life-threatening.
Emflaza will cost $89,000 for one year's treatment supply
Emflaza is produced by Northbrook, Illinois-based Marathon Pharmaceuticals and will cost $89,000 for one year's treatment supply. This is actually on the high side but six-year-old Marathon justifies this costly price with the argument that it took them six years to research, launch and get the neurological drug approved by the FDA; and that only a negligible percentage of the population requires it, while health insurance company will cover most of the costs. It will available for free through a Marathon program only for DMD patients without any insurance coverage.
The truth however remains that rare drugs for rare diseases are often costly, and these are often called "orphan drugs". According to Chicago Tribune, the top 100 orphan drugs in the United States go for an average cost of $111,820 annually per patient as at 2014. Even insured patients with high-deductible plans will still have to pay exorbitant amounts in out of pocket costs to acquire the drug.
FDA gave Emflaza a fast-track review and approval
In a FDA news release, DMD results when the body cannot produce dystrophin, a protein known to keep muscle cells intact and integrated. As the condition progresses, the muscles of the body starts to wear or waste away and affected children soon lose their ability to walk or feed themselves or carry out any other personal duties requiring muscle functions.
Emflaza was issued a priority review and a fast track designation to underscore the eagerness of the FDA to get a promising drug that could treat DMD in the US market. This is to encourage other pharmaceutical companies to fast-track the development of drugs for prevention and treatment of rare pediatric diseases.
"We hope that this treatment option will benefit many patients with DMD," said Dr. Billy Dunn, director of the Division of Neurology Products in FDA's Center for Drug Evaluation and Research. "This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy."
New Study Explains How Body Produces Cholesterols
A recent report titled “Managing Your Cholesterol” and published by the Harvard Special Health Report reveals how the human body produces the famed cholesterols and the kinds of cholesterols that impact the human body and health.
8,000 Opioid Antidote Kits to Be Distributed In West Virginia to Combat Overdose
Health officials West Virginia have concluded plans to distribute over 8,000 opioid antidote kits to emergency medical personnel and other first responders in order to reduce deaths associated with opioid overdose in the state. The drug Naloxone is the opioid antidote of choice.
Donald Trump On Hair Loss Drug, Doctor Reveals
US President Donald Trump’s longtime physician, Dr. Harold Bornstein, has revealed that the president takes hair loss drug among other medications - a revelation that has not gone down well with Trump supporters.
President Trump Nominates Conservative Physician, Scott Gottlieb, To Head the FDA
President Donald Trump has nominated Dr. Scott Gottlieb as potential had of the US Food and Drug Administration (FDA) agency. Gottlieb is regarded as a conservative physician and businessman with extensive experience of how the pharmaceutical industry is run. In fact, many voices in the health industry call for the US Senate to approve his nomination for the job.
Debunking The Duchenne Muscular Dystrophy: Can Exonics’ CRISPR Be The Answer?
What is Duchenne muscular dystrophy and what causes this genetic disorder? Considering the numerous biotech companies venturing in various cure for diseases nowadays, could their CRISPR therapy put an end to this ongoing problem? Will it be the advent of the disease’s cure? Find out what health experts have to say
DMD Gene Editing Therapy Could Cure Affected Boys
Exonics Therapeutics receives $5 million in funding to develop cutting-edge DMD gene editing technology to help cure majority of the disease mutations in young boys. The new CRISPR/Cas9 tech could provide lifelong benefits to Duchenne patients, preventing further decline and improving quality of life.
MORE IN ITECHPOST
The Best Protocol for Low Power IoT Devices
The rise of the Internet of Things (IoT) devices on the global market makes their communication and conductivity a critical topic to think about. IoT devices take advantage of a number of communication protocols that come with different capabilities, communication ranges, power, memory, and data rates. Each of these protocols also offers unique advantages and presents the drawback of which you should be aware.
Scientists Have Simulated The Asteroid That Caused The Extinction Of The Dinosaurs
Using a supercomputer, scientists simulated the asteroid that hit the Earth and made all the dinosaurs go extinct and left behind the Chicxulub crater. Their results are shocking.
A Comprehensive Guide to Choosing the Best Gaming Desktop
The best-performing gaming devices are expensive. So, when you pour your savings into getting a gaming PC, you must make the right choice. Whether you want to play Star Wars or Assassin creed in 4k, you need to know what works well. Here's is are some of the factors you must consider when choosing the right gaming desktop.