Biotechnology company AveXis, which develops treatments for rare neurological genetic disorders, is set by year’s end to launch production of its gene therapy Zolgensma at its Longmont plant, which it purchased in March.
Zolgensma is injected in the blood to treat children younger than 2 with spinal muscular atrophy, said David Lennon, president of AveXis, which is based in Bannockburn, Ill. In May the U.S.Food & Drug Administration approved the therapy.
About 400 to 500 kids in the nation every year are born with spinal muscular atrophy, a genetic disorder that affects their ability to control their muscles, he said. About 60% of kids with the disease die before turning 2, he said.
Zolgensma helps correct the gene defect, Lennon said. The treatment, which is only approved in the United States, costs $2.1 million. The therapy has a high response rate, he said.
The cost of a traditional ongoing treatment for spinal muscular atrophy, or “floppy baby syndrome” is about $300,000 to $400,000 a year, Lennon said.
In March, AveXis purchased the former AstraZeneca site at 4000 Nelson Road for $30 million, according to Boulder County Assessor’s Office, and also hired 150 former AstraZeneca employees.
The company chose to come to Longmont for available talent and nearby research universities, Lennon said.
“Our goal is to make Longmont the world’s largest gene therapy facility,” he said.
Earlier this month the FDA began investigating ” a small portion of the product testing data” contained in the marketing application for Zolgensma after AveXis voluntarily reported “a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals.”
The FDA while assessing the situation remains confident “Zolgensma should remain on the market. …These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program,” according to an FDA statement.
“We are fully confident in the safety, quality and efficacy of Zolgensma and are working cooperatively with FDA on this matter,” Lennon said.
Novartis, which last year acquired AveXis for $8.7 billion, also released an official statement in response to the FDA’s statement, which in part said, “We maintain that the totality of the evidence demonstrating the product’s effectiveness and its safety profile …”
Lennon said said AveXis is focused on nerve and muscle diseases, and is researching possibilities to develop gene therapies for other disorders, including a number of genetic eye diseases; Amyotrophic Lateral Sclerosis, or Lou Gehrig’s Disease, a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord; and Rett syndrome, a rare genetic neurological disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat, and even breathe easily.
Developing gene therapies and making them requires specialized skills, said Sally Dyer, vice president of manufacturing operations and Colorado site head. The company is investing $55 million in the Longmont facility, she said. The number of employees in Longmont will go up from 240 to 400 by the end of 2020, she said.
In addition to Longmont, AveXis has manufacturing facilities in Chicago, and Durham, N.C., and a research and development site in La Jolla, Calif.