SMA Drug Developer AveXis Granted SME Status For European Subsidiary

The European Medicines Agency (EMA) has recognized a new European subsidiary of the biotech company AveXis, Inc. by granting it Small and Medium Sized Business Entities (SME) designation. AveXis EU Ltd. will work together with the parent company to improve the quality of life of patients who suffer from genetic and orphan conditions such as spinal muscular atrophy (SMA) by continuing to develop innovative gene therapy for those with the disease.

The status was granted by European authorities in order to support the developmental process of new drugs by the company. The program was established in 2005 and it grants incentives to drug developers in order to accelerate product development. This designation grants the company  administrative assistance, reduced or deferred fees for regulatory processes, scientific advice, and/or inspections.

“Receiving regulatory guidance directly from the EMA will be invaluable as we move forward with the development of our gene therapy, which is considered an innovative and advanced drug product in the pharmaceutical industry,” said  John A. Carbona, CEO of AveXis. Mr. Carbona announced via press release, that the company is going to continue to focus on increasing disease awareness for SMA through the Contact All Neurologists (C.A.N.) Program.

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The C.A.N. Program highlights a series of clinical trials and aims to reach a larger number of neurologists in the country, educating them on the studies available for pediatric patients diagnosed with SMA type 1. The European subsidiary was launched in the beginning of the month with the main purpose of advancing drug 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. In addition to SME, the company also plans to apply for the Orphan Medicinal Product Designation.

AveXis is currently focused on its gene therapy system, called chariSMA, which is currently being evaluated in a phase I clinical trial that includes children with SMA type 1, which is still enrolling. In September 2013, the Research Institute at Nationwide Children’s Hospital received the IND approval and Fast Track designation. In order to accelerate its development, AveXis completed a $10 million financing set, led by Deerfield Management and Roche Venture Fund.