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Dallas – February 25, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced the formation of its EU-based wholly-owned subsidiary, AveXis EU, Ltd. The subsidiary will allow AveXis to move forward with its plans to open clinical trials in the European Union and leverage European Medicines Agency’s programs, such as Orphan Medicinal Product Designation and the Micro-, Small-, and Medium-Sized-Enterprise (SME) program, to advance development.
Similar to the FDA Orphan Drug Designation, the EMA Orphan Designation grants eligible companies several benefits, such as reduced fees for protocol assistance and scientific advice and market exclusivity for 10 years once the drug is granted approval. The EMA, recognizing the need to encourage drug development by small biotech companies, also established the SME program in late 2005 which offers administrative and procedural assistance, among other incentives.
AveXis is currently sponsoring a US-based Phase 1 open-label, single-dose, dose-escalation study, which will enroll nine infants under the age of nine months who have been diagnosed with SMA Type 1. The study is being conducted at The Research Institute at Nationwide Children’s Hospital. The trial is currently open for enrollment and recruiting candidates. For further details on the trial, enrollment criteria, eligibility, and contact information, please visit http://clinicaltrials.gov/ct2/show/NCT02122952.
AveXis has also continued its efforts to grow awareness of the ongoing and upcoming clinical trials with the Contact All Neurologists (C.A.N.) ProgramTM. The C.A.N. ProgramTM has reached over 30 neurologists nationwide, informing them of clinical trials available to infants recently diagnosed with SMA Type 1. For neurologists, pediatricians, or physicians interested in participating in the AveXis C.A.N. ProgramTM, please contact CAN@avexisinc.com.
To learn more about the Gene Transfer Clinical Trial, other available trials for SMA, and SMA patient advocacy group, please visit: www.smastudy.org, a patient-oriented, SMA-focused web resource.
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