This new drug appears to improve muscle function in boys with muscular dystrophy, which is exciting because usually fighting muscular dystrophy means trying to slow the loss of muscle function. Once the decline begins, it usually never improves.
The sudden and totally unprecedented reversal of this pattern in a test group of boys is “incredibly promising” according to Lee Sweeney, chairman of physiology at the University of Pennsylvania’s Perelman School of Medicine. Lee Sweeney wasn’t involved in the study, but he says the results are not normal at all.
Muscular Dystrophy Treatment in America
The most common type of muscular dystrophy is Duchenne muscular dystrophy, which affects about 13,000 boys in the United States. Because the disease is caused by a defect on the X chromosome, girls rarely get Duchenne muscular dystrophy — girls have a second copy of their X chromosome.
The medicine, which is called eteplirsen, is targeted at a specific 13% of boys with Duchenne muscular dystrophy. This 13% has muscular dystrophy because of a specific mutation — one that can be corrected by eteplirsen, which tells the body skip a part of the dystrophin gene called exon 51.
Other drugs are being developed to skip the genes exon 45 and exon 50, which will have the potential to help 9% and 5% of muscular dystrophy patients, respectively.
FDA Approval?
The only uncertainty so far with eteplirsen is how long it will take to make it through FDA approval processes. The initial test group is an extremely small patient population — only 12 subjects in total. Many test groups are several hundreds of patients, and there are often several repeated stages of testing before a drug is approved.
Many patients are hoping for a “super-fast regulatory approval” for eteplirsen, but while the results are convincing, Dr. Sweeney, who worked on the drug, says that a quick approval may be “too much to hope for.”
The unfortunate truth is that the FDA has patient risk liability to think of first and foremost, and the organization will probably not rush a drug through the approval process based on just one study. There’s always the chance, though, as other drugs have been approved with similarly limited experimental evidence, such as Genzyme’s Myozyme drug. This drug, too, was designed to treat an extremely rare disease.
We specialize in providing our over 1,000,000 customers with relevant product and condition information created by our professional editorial staff which includes our team of medical writers, medical practitioners, and health educators. eDrugStore.com Staff on Facebook