AveXis on gene therapies: ‘We’ll get better at this and it will get cheaper’

AveXis has spent about $1 billion in preparing to launch its spinal muscular atrophy (SMA) gene therapy later this year. The Novartis subsidiary is pumping a further $60 million into its North Carolina facility.

In May 2018, Gene therapy firm AveXis announced a $55 million (€49 million) investment to establish a manufacturing center in Durham County, North Carolina. Nine months on, and AveXis is doubling down on the site, revealing plans to invest a further $60 million and create 200 jobs.

This latest expansion in AveXis’s gene therapy network comes ahead of the potential regulatory approval of its lead candidate AVXS-101, known as Zolgensma (onasemnogene abeparvovec). Zolgensma is under regulatory review in the US, Europe and Japan for the treatment of spinal muscular atrophy (SMA). The US Food and Drug Administration (FDA) has set a PDUFA date for May.

AveXis has pledged a further $60m to its County Durham, NC site. Image: iStock/Milenius

“The reality is we will have spent, before launch, about a billion dollars in direct investment, almost half of that into manufacturing,” AveXis president Dave Lennon told delegates during a fireside chat at the Phacilitate conference in Florida last month.

“We’re pretty confident we’ll be ready for launch and we’re continuing to build capacity. This will be an ongoing effort, and this is not just building footprint but it’s also improving the process.”

Lowering processing costs

The current costs of cell and gene therapies is a major talking point. Spark Therapeutics’ Luxturna (voretigene neparvovec), the first gene therapy for a genetic disease approved in the US, had a list price of $850,000, or $425,000 per eye. Lennon has said Zolgensma, if it reaches the market, could have a price in the range of $4 million to $5 million, something that is likely to be problematic for payors and healthcare systems.

For a therapy like Zolgensma, the product is the process in this case, and so reducing the cost of manufacturing will immediately reduce the price.

It is early days in the gene therapy space, and the cost of innovation remains high. “But we will get better at this, and it will get cheaper,” Lennon told delegates.

“Avexis has always been structured around finding the best solutions wherever they are, and I think we will continue that approach,” he said. “There are so many steps to optimize here. We’re going to continue to look at partners and [technology] advances; we’ll explore of course doing that ourselves but at the same time we’re very happy to be partnering.”

A biotech within a Big Pharma

Lennon said the constant investments at AveXis’ plants in Illinois and North Carolina, have helped to ensure a “really rigorous production for commercial production,” adding this was a big driver for why Novartis paid $8.7 billion for the firm last May.

“We at AveXis believe we can be the world’s leading gene therapy firm,” he told delegates. “We have the know-how, the position, and we’re building up a portfolio with the support of a large pharma company.”

He added despite the questions surrounding these next-generation therapeutics, Novartis’ acquisition replicates the model Big Pharma has undertaken in the biotech space for decades.

“The reality is very few biotechs have been able to go global in a true sense without the support of a Big Pharma partner so we’re really encouraged with the attitude Novartis is taking, keeping Avexis whole within the company – a biotech within a Big Pharma.”