Drug developer AVI Biopharma Inc. said Monday the Food and Drug Administration granted fast-track status to its product candidate for Duchenne muscular dystrophy, a fatal genetic disorder in children.
Fast-track designation is given to product candidates to help them reach the market faster. 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.
The drug candidate, AVI-4658, uses the company's Exon Skipping PreRNA Interference Technology, or ESPRIT. In animal models, ESPRIT technology restored near-normal levels of dystrophin production.
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 DMD, and roughly 15,000 to 20,000 children have DMD in the U.S., according to AVI.
AVI plans to initiate a clinical trial with AVI4658 by mid-2008 to evaluate the safety and effectiveness of the drug in ambulatory DMD patients, or those who can walk on their own.
Shares of Portland, Ore.-based AVI rose 7 cents, or 3 percent, to $2.40 in morning trading.
Terms & Conditions
RSS Headlines
Fool UK