Drug Development

AveXis wins the inside track at the FDA with gene therapy ‘breakthrough’

Chicago-based AveXis has lined up the latest breakthrough therapy designation from the FDA, gaining an inside track at the agency for its early-stage gene therapy for lethal cases of spinal muscular atrophy in children. The biotech says it won that mark of distinction after regulators got a preliminary look at the results from their Phase I study.

AveXis was able to score a $95 million IPO last February as it joined the first wave of biotechs to hit the public market this year, a little after the once red-hot gene therapy field had begun to cool somewhat. The offering followed a $65 million crossover round last fall which was joined by Roche.

Like a number of other gene therapy companies, AveXis in-licensed vector tech from RegenX, which banked IP developed by Penn’s James Wilson, a pioneer in gene therapy work. Its study is being done in collaboration with The Research Institute at Nationwide Children’s Hospital — which has a number of gene therapy projects underway — and Ohio State University.

The next step for AveXis: A Type B meeting with regulators to go over their development plans. The FDA set up the BTD effort so it could help accelerate promising drugs through the clinic. And now AveXis should get VIP treatment and an open door when needed at the agency.


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RAPS Regulatory Convergence 2017