Dallas, Texas-based AveXis, Inc., a biotechnology company currently developing an experimental therapy for Spinal Muscular Atrophy (SMA) through the use of innovative gene therapy, recently announced that they have established a European Union (EU) based wholly-owned subsidiary called AveXis EU, Ltd. This subsidiary will be used by AveXis to advance development of its lead product in Europe by opening and managing clinical trials in the EU as well as applying for key European Medicines Agency programs, such as the Orphan Medicinal Product Designation and the Micro, Small, and Medium Sized Enterprise (SME) program.
For AveXis, having an opportunity to utilize the EMA Orphan Designation may offer several major benefits in accelerating the development process of their SMA therapy in Europe, such as reduced fees for assistance with protocols, access to scientific advice from the EMA and market exclusivity for 10 years once the drug is granted approval.
In a press release about the expansion, John A. Carbona, Chief Executive Officer of AveXis, stated “We are excited to begin planning for the expansion of AveXis’ presence in the European Union. Establishing an EU-based subsidiary is our first step in opening communications between the regulatory agencies that govern the conduct of clinical trials in Europe and AveXis.”
When asked about the importance of AveXis’ current work in developing an SMA therapy, Dr. Brian K. Kaspar PhD, Principal Investigator at The Center for Gene Therapy at The Research Institute and AveXis researcher, stated, “We have devoted our scientific discoveries and our tireless pursuits for the patients and families afflicted with SMA. We have every reason to be excited about advancing towards human trials and the potential to make a clinical meaningful impact on this disease. Our team will not rest until we have made a difference.”
This announcement comes just a month after AveXis, released a statement confirming the completion of a $10 million financing agreement to help with funding for their human clinical trial testing. These back-to-back announcements are beginning to give SMA patients, their loved ones and healthcare providers an optimistic outlook for the future, one in which the disease can be both treated and cured.
Currently Open AveXis Trial
In other related SMA news, AveXis is sponsoring a US-based Phase 1 open-label, single-dose, dose-escalation study that 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.
This trial is currently open for enrollment and recruiting candidates. For further details on the trial, enrollment criteria, eligibility, and contact information can be found here.