Rare Daily Staff

Novartis-owned AveXis said it signed an agreement to purchase an advanced biologics therapy manufacturing campus in Longmont, Colorado, further expanding its production capacity as it prepares to launch Zolgensma, an experimental gene therapy awaiting global regulatory approvals for the treatment of spinal muscular atrophy Type 1.

AveXis currently has a fully-operational state-of-the-art manufacturing facility in Illinois, is building a facility in North Carolina scheduled to be operational in 2020 and is expanding its product development capacity at its San Diego facility.

The addition of the six-building Longmont campus consists of nearly 700,000 square-feet of space for biologic drug manufacturing, offices, laboratories, warehousing, and utilities.

“Our Longmont, Colorado campus, along with our existing manufacturing sites in Illinois, California, and North Carolina, will play a crucial role in helping us achieve the future manufacturing capacity required to meet the global patient need for novel gene therapies,” said Andrew Knudten, senior vice president of global strategic operations. “We are eager to add the talented team in Longmont to AveXis, and we hope that they will choose to join us as we build world-leading manufacturing capabilities in gene therapy.”

Zolgensma is an investigational gene therapy currently in development as a one-time infusion for SMA Type 1. Zolgensma is designed to address the monogenic root cause of SMA and prevent further muscle degeneration. Zolgensma represents the first in a proprietary platform to treat rare, monogenic diseases using gene therapy. In December, the FDA accepted the company’s application for use of Zolgensma with SMA Type 1 patients. The drug previously received Breakthrough Therapy designation and has been granted Priority Review by the FDA, with regulatory action anticipated in May 2019. In addition, the drug is anticipated to receive approval in Japan and the European Union later this year.

SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The most severe form of SMA is Type 1, a lethal genetic disorder characterized by rapid motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support by 24 months of age for more than 90 percent of patients.

Zolgensma is an investigational gene therapy currently in development as a one-time infusion for SMA Type 1. Zolgensma is designed to address the monogenic root cause of SMA and prevent further muscle degeneration. Zolgensma represents the first in a proprietary platform to treat rare, monogenic diseases using gene therapy. In December, the FDA accepted the company’s application for use of Zolgensma with SMA Type 1 patients. The drug previously received Breakthrough Therapy designation and has been granted Priority Review by the FDA, with regulatory action anticipated in May 2019. In addition, the drug is anticipated to receive approval in Japan and the European Union later this year.

Photo: Andrew Knudten, Senior Vice President, Global Strategic Operations

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