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AVI BioPharma Receives EU Positive Opinion for Muscular Dystrophy Drug

October 15, 2008

AVI BioPharma has announced that the European Medicines Agency Committee for Orphan Medicinal Products adopted a positive opinion recommending orphan medicinal product designation for AVI-4658 to treat Duchenne muscular dystrophy.

The company believes the positive opinion of the Committee for Orphan Medicinal Products (COMP) will serve as the scientific basis for the European Commission to issue a EU orphan designation pursuant to Regulation (ED) 141/2000.

Additionally, AVI BioPharma received notification from the Gene Therapy Advisory Committee (GTAC) in the UK granting provisional approval for the company’s planned clinical trial for systemic delivery of AVI-4658 to treat Duchenne muscular dystrophy (DMD). The conditions for final GTAC approval include certain wording changes in the patient and parent information documents and completion of normal site specific assessments. AVI expects to comply with the conditions for final approval this quarter.

AVI-4658 is designed to skip exon 51 of the dystrophin gene, thus repairing the mutated reading frame in the mRNA sequence coding for dystrophin, a vital protein which is absent or virtually absent in boys with DMD. By skipping this exon, a truncated, yet functional, form of the dystrophin protein is produced and this could ameliorate the disease process, potentially prolonging and improving the quality of life in these patients, the company said.

Leslie Hudson, president and CEO of AVI BioPharma, said: “The EMEA committee’s positive recommendation on orphan status for AVI-4658 in DMD together with GTAC’s favorable review of our clinical trial protocol for the same drug candidate adds further momentum to the development of AVI’s portfolio of exon skipping drugs to treat this devastating and debilitating disease.

“These positive events are evidence of the company’s continuing commitment to advance potential new treatments for DMD. We expect to see results from our ongoing intramuscular administration trial shortly and to begin the intravascular administration trial once all approvals are finalized. We also look forward to advancing AVI-4658 towards the clinic in the US during 2009.”




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