- AveXis, a Novartis subsidiary, has purchased another manufacturing facility as it prepares for a fast-approaching approval decision on its most advanced gene therapy.
- The six-building campus in Longmont, Colorado, gives the Swiss biotech nearly 700,000 square feet of additional space for biologic drug manufacturing, laboratories, offices, utilities and warehousing. AveXis said hiring staff and getting the site ready for production, scaling and testing will be some of its first initiatives in Longmont.
- It's expected in the next month or so that the Food and Drug Administration will give a thumbs up or down to Zolgensma, AveXis' treatment for a rare neuromuscular disorder called spinal muscular atrophy. With a potential launch on the horizon, AveXis has been building out its manufacturing capabilities and stockpiling gene therapy product.
Manufacturing is arguably the largest barrier separating experimental gene therapies from coming to market. Spark Therapeutics, for example, has noted how most of the 60,000-page approval application it submitted for Luxturna (voretigene neparvovec) wasn't about clinical trial data, but about chemistry, manufacturing and controls.
Brian Kaspar, chief scientific officer at AveXis, told BioPharma Dive his company has spent years trying to understand the various complexities of gene therapy production. Along that journey, AveXis found it particularly important to have programs and analytics which could verify the stability and consistency of Zolgensma (onasemnogene abeparvovec).
"Batch one should look and act like batch number three, which should look and act like batch number 33 and 303," Kaspar said in a March 29 interview.
AveXis also wanted to make sure it owned that manufacturing process, opting to build or expand facilities rather than contract the work out. With its new campus (which was previously owned by AstraZeneca), the biotech now runs four U.S. production sites scattered across as many states.
Sites in California and North Carolina are undergoing expansions. The latter recently received a $60 million investment and should be operational in 2020. Kaspar noted that AveXis had plans to open a North Carolina facility while the company was a standalone, yet those plans — as well as funding — grew after the acquisition by Novartis.
AveXis expects to create 200 new jobs in science, engineering, operations and other roles in North Carolina and more than 1,000 high-tech biologics manufacturing positions across its entire U.S. footprint by the end of 2019.
The investments in manpower and facilities come as AveXis eyes a Zolgensma launch. Wall Street expects that, if approved, the gene therapy would put competitive pressure on Biogen's Spinraza (nusinersen), which was the first-ever treatment for SMA to clear the FDA.
Though it hasn't disclosed the intended listed price for Zolgensma, Novartis did suggest not too long ago that a price tag of $4 million to $5 million would still offer good clinical value. Spinraza is currently sold at a list price of $750,000 for the first year of treatment and $375,000 each year after.
The Institute for Clinical and Economic Review, a watchdog typically critical of high drug prices, published preliminary evidence in December that showed a one-time gene therapy treatment for SMA set at $2 million could be more cost effective than Spinraza.
Investment bank SVB Leerink pegs Zolgensma's list price at $2 million and its sales in 2024 at $3.1 billion.