Dallas, Texas-based biotech company AveXis Inc. has announced the formation of a wholly-owned subsidiary based in Europe, AveXis EU, Ltd. The company expects this new subsidiary to facilitate the management of clinical studies in the European Union, as well as handle other procedures involving the European Medicines Agency (EMA) to advance the development of their drugs focused on severe genetic conditions with unmet medical needs such as spinal muscular atrophy (SMA).
The creation of AveXis EU, Ltd. is also expected to enable an expansion of the company’s market in the European Union by applying for the Orphan Medicinal Product Designation and the Micro-, Small-, and Medium-Sized-Enterprise (SME) program from the EMA for their SMA gene therapy candidate.
“We are excited to begin planning for the expansion of AveXis’ presence in the European Union,” said the CEO of AveXis, John A. Carbona, in a press release. “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.”
If approved, the company can benefit from the EMA’s Orphan Designation, which offers reduced fees for assistance in protocols and scientific advice, as well as market exclusivity for 10 years after the approval of the drug. The designation is granted to accelerate the drug development process conducted by biotech companies to address diseases with high unmet medical needs, while the SME program was created in 2005 to provide administrative and procedural support to biotech companies.
AveXis’ current study is evaluating the safety and efficacy of single, escalating doses of ChariSMA in nine infants younger than nine months who suffer from SMA type 1. The Gene Transfer Clinical Trial is currently enrolling and recruiting participants, and is being conducted by the Research Institute at Nationwide Children’s Hospital.
Other projects from AveXis include its Contact All Neurologists (C.A.N.) Program, which aims to raise awareness about ongoing and upcoming studies available to pediatric SMA patients nationwide, which has already reached more than 30 neurologists in the country. In addition, the CEO of the company, John Carbona, attended the 26th Annual Piper Jaffray Healthcare Conference last December, where he presented his company’s novel gene therapy approach to improve quality of life and disease outcome in SMA patients.