With over one million square-feet of manufacturing space, Novartis says it is prepared for the imminent approval of AveXis’ SMA gene therapy Zolgensma.
Speaking during its Q1 2019 results, Novartis said it is set for the imminent arrival of gene therapy Zolgensma (onasemnogene abeparvovec), added to the firm’s pipeline through the acquisition of AveXis. The one-time therapy targeting spinal muscular atrophy (SMA) Type 1, will be reviewed by the US Food and Drug Administration in May, despite the recent news of a second trial death currently under investigation.
“We have established readiness ahead of our US approval, expected now in May for our PDUFA date,” Novartis CEO Vasant Narasimhan told investors. “As you can see, from an institutional standpoint, we have delivery infrastructure set up. We’re set up for rapid product delivery. We’ve already reached the 60 top centers. And we’re ready to cover 80% of infants with SMA.”
If Zolgensma reaches the market, AveXis has said its price could be in the range of $4 million to $5 million per patient.
The firm also continues to build up its manufacturing capabilities in preparation for launch and future scale-up.
“We have over one million square feet now [in] manufacturing space and [we are] preparing that space to continue to ramp up production,” Narasimhan continued. “We’ve engaged over 70 payers in discussions, covering 80% of the SMA infant population and expect to have contracts in place at launch to cover 30% of commercial lives.”
Novartis has manufacturing capacity in Illinois and is expanding product development at a facility in San Diego, California. Meanwhile, two investments totaling $115 million have established a gene therapy manufacturing center in Durham, North Carolina, and earlier this month the firm acquired a 700,000 square-foot production plant in Longmont, Colorado from AstraZeneca.
Paul Hudson, CEO of Novartis Pharmaceuticals, added more color to the Longmont acquisition:
“We were already set up before we took on the Colorado facility to match what we think will be the demand over the next year or so. We took the opportunity to take on a high-tech facility, a biologics GMP-approved facility to make sure that as we go further into the prevalent population, if there’s an unprecedented level in the [Phase] 2s and 3s, we can cover that, too. So, we have more than enough capacity to deal with what we think could be an unprecedented demand.”
In the first quarter 2019, Novartis’s chimeric antigen receptor (CAR) T-cell therapy Kymriah (tisagenlecleucel) pulled in $45 million (€40 million).
While still relatively low, Kymriah sales are increasing each quarter driven by new treatment sites in the EU, progress with reimbursement in the US, and the gradual approval of additional indications in various geographic markets, the firm said.
“I think when you look over the last six months, we made quite good progress in terms of building our manufacturing footprint,” Susanne Schaffert, CEO of Novartis Oncology said on the call. “We recently got FDA approval to increase manufacturing capacity. We got in addition approval from health authorities outside the US, namely EU, Switzerland, Australia, Japan and Canada to widen the commercial specification in line with the clinical product.
“FDA has not yet approved our prior approval supplement application citing insufficient information to widen Kymriah’s commercial specification. So, we are obviously disappointed by this decision and we are working closely with FDA to find a way forward. We also plan to collect additional data and we update you as soon as we have more clarity.”