WASHINGTON The Food and Drug Administration has granted fast-track status to drug developer AVI Biopharma’s product candidate for Duchenne muscular dystrophy, a fatal genetic disorder in children, the Associated Press reported.
Duchenne muscular dystrophy is an incurable muscle-wasting disease, caused by a mutation in a person's dystrophin gene. The result is membrane leakage and fiber damage, leading to degeneration and death of the muscle fiber. About one in 3,500 boys is born with it, and roughly 15,000 to 20,000 children have DMD in the U.S., according to AVI.
The status means a company can submit data to the FDA as it becomes available and receive agency feedback, rather than having to wait and turn it in all at once. AVI plans to initiate a clinical trial with its drug candidate by mid-2008 to evaluate the safety and effectiveness of the drug in DMD patients who can still walk on their own.