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AVI BioPharma Receives Fast Track Designation for AVI-4658 for Treatment of Duchenne Muscular Dystrophy

PORTLAND, Ore.--(BUSINESS WIRE)--AVI BioPharma, Inc. (Nasdaq:AVII), today announced that the U.S. Food and Drug Administration has granted Fast Track status to its product candidate, AVI-4658, for the treatment of Duchenne muscular dystrophy (DMD).

“We look forward to fruitful and timely discussions with the FDA to agree on an approach that will accomplish these objectives.”

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DMD is the most common fatal genetic disorder to affect children around the world. It is a devastating and incurable muscle-wasting disease associated with specific inborn errors in the gene that expresses dystrophin, a protein that plays an important structural role in muscle fibers. When dystrophin is missing or nonfunctional due to a mutation in the dystrophin gene, as it is in DMD, the result is membrane leakage and fiber damage, ultimately leading to degeneration and death of the muscle fiber. In two-thirds of DMD cases, the genetic mistake is hereditary, but one-third of cases arise spontaneously. There is no cure for DMD. Approximately one in 3,500 boys is born with DMD, and an estimated 15,000 to 20,000 children have DMD in the United States alone.

AVI-4658 uses AVI’s Exon Skipping Pre-RNA Interference Technology (ESPRIT) to potentially benefit DMD patients who have mutations in exon 51 of the dystrophin gene. In animal models, ESPRIT technology has demonstrated the ability to selectively bypass defective exons, thus restoring near-normal levels of dystrophin production. The company believes that by using AVI 4658 to skip exon 51, clinically relevant levels of dystrophin may be produced, limiting further...