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news.AVI BioPharma has said that the FDA's Office of Orphan Products Development has granted the orphan drug designation to AVI-4658, for the treatment of Duchenne muscular dystrophy.
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AVI is planning clinical development of AVI-4658 including a dose-ranging trial using systemic administration of the drug. This trial will be conducted in conjunction with the company’s duchenne muscular dystrophy (DMD) cross-licensing and development partner, Ercole Biotech.
AVI-4658 uses AVI’s ESPRIT technology (exon skipping pre-RNA interference technology) and is designed to benefit patients with certain mutations in the dystrophin gene. By ‘skipping’ exon 51, the proper RNA reading frame can be restored in suitable patients, resulting in the production of functional dystrophin.
Michael Forrest, interim CEO of AVI, said: “The orphan drug designation for AVI-4658 is a significant step in AVI’s development of this drug candidate. DMD is a devastating disease that currently has no viable treatment or cure. Patients deserve the hope of a longer and higher quality of life through innovative therapeutics.”