AVI BioPharma, Inc, a developer of RNA-based drugs, announced that the European Medicines Agency (EMEA) Committee for Orphan Medicinal Products (COMP) adopted a positive opinion recommending orphan medicinal product designation for AVI-4658 to treat Duchenne Muscular Dystrophy (DMD). Additionally, the company 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 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.
The company believes the positive COMP opinion will serve as the scientific basis for the European Commission to issue a European Union orphan designation pursuant to Regulation (ED) 141/2000. Orphan designation in the EU brings several benefits to the sponsor including certain fee waivers, protocol assistance in product development, and marketing exclusivity up to 10 years.
"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," said Dr Leslie Hudson, president and chief executive officer of AVI BioPharma. "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."
In June of 2008, the Medicine & Healthcare Product Regulatory Agency (MHRA) in the UK gave clearance for the Company to move forward with a Company-sponsored systemic clinical trial of AVI-4658 in the United Kingdom. This trial, which will involve an intravascular (IV) administration to sixteen ambulatory boys with DMD, is expected to start in the current quarter once GTAC approval is complete. The Company was granted an orphan drug designation for AVI-4658 by the US Food and Drug Administration (FDA) in November of 2007
AVI BioPharma is currently engaged in a clinical trial at the Imperial College of London where patients with DMD are receiving a single-dose, intramuscular (IM) administration of AVI-4658. This study is being conducted in collaboration with the United Kingdom-based MDEX Consortium. 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.
Leading experts specializing in the fields of genetic disease and/or neuromuscular disorders believe that exon skipping is one of the most exciting and promising approaches to treat a majority of DMD patients. AVI has continued to demonstrate its scientific leadership in the application of exon skipping to DMD through the publication of preclinical results of studies demonstrating the ability of AVI's new class of drug candidates - termed PPMO-B - to induce sustained expression of dystrophin in the mdx mouse model of DMD. Treatment with this new class of AVI compound resulted in the sustained production of functional dystrophin in numerous tissues, including the heart, diaphragm and skeletal muscles. These are key organs for the treatment of the disease.
DMD is the most common fatal genetic disorder to affect children around the world.
AVI BioPharma is focused on the discovery and development of RNA-based drugs using the company's expanded portfolio of proprietary antisense compounds (PMOs).