AVI BioPharma, Inc, a developer of RNA-based drugs, has received an orphan drug designation from the Committee for Orphan Medical Products of the European Medicines Agency (EMEA) for AVI-5038, a drug candidate being developed by AVI for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a genetic muscle wasting disease caused by failure to produce dystrophin. The orphan drug designation potentially may provide AVI up to 10 years of market exclusivity if the drug candidate is approved for marketing in the European Union (EU). AVI-4658, another drug being developed by AVI for DMD, received European orphan drug designation in 2008, and also potentially may receive up to 10 years of marketing exclusivity if approved in the EU.

“The EMEA’s granting of orphan drug designation to AVI-5038 provides important regulatory support for our continuing commitment to develop disease modifying drugs for DMD patients,” stated Dr Leslie Hudson, president and CEO, AVI BioPharma, Inc. “We look forward to the opportunity to report continuing progress in our DMD programme throughout the year, particularly with respect to our lead DMD drug candidate, AVI-4658, which is in an ongoing phase-1b/2 clinical trial.”

Products granted an orphan drug designation by the EMEA are intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU, or are medicines which, for economic reasons, would unlikely be developed without incentives. The aim of the EU orphan medicines designation is to stimulate research and development of medicinal products for rare diseases by providing incentives to the pharmaceutical industry. This initiative helps to give patients suffering from rare diseases access to the same quality of treatment as other patients. Applications for designation of orphan medicines are reviewed by the EMEA through the Committee for Orphan Medicinal Products.

AVI BioPharma is focused on the discovery and development of RNA–based drugs utilizing proprietary derivatives of its antisense chemistry (morpholino-modified phosphorodiamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms of action.