- AveXis, now part of Swiss pharma Novartis, is expanding its manufacturing center in North Carolina's Research Triangle Park, announcing plans to invest an additional $60 million and add 200 more jobs.
- The new roles will include engineers, manufacturing and quality control personnel and supply chain specialists, bringing an additional annual payroll of $20 million to a state known for its biotech and research prowess.
- The state's Economic Investment Committee supported the expansion, granting the biotech $2.2 million in investment monies spread over 12 years.
AveXis established its manufacturing center in the Research Triangle area last May with $55 million in investment to produce therapies to address rare neurological genetic diseases.
Yet the biotech, which Novartis acquired last year, appears to have already outgrown those plans.
"Continued investment in our infrastructure in North Carolina will allow us to manufacture multiple gene therapies simultaneously, helping us reach more patients, faster," said Andy Stober, chief technology officer of AveXis, in a statement.
Gene therapy presents unique manufacturing challenges and requires production of the viral vectors required to deliver the treatments. AveXis utilizes an adeno-associated vector 9 platform for delivery.
Novartis picked up AveXis in a deal worth $8.7 billion, aiming to build a gene therapy franchise. AveXis is developing DNA-based treatments for rare neurological genetic diseases, such as spinal muscular atrophy (SMA), genetic amyotrophic lateral sclerosis and Rett syndrome.
AveXis' lead gene therapy, Zolgensma (onasemnogene abeparvovec), is currently under review by U.S. regulators for the treatment of SMA Type 1, a severe form of the inherited disease that typically leads to death before age 2. A regulatory decision is expected in May and would mark another milestone in the hastening emergence of gene therapy.
Yet, even if approval is won, Zolgensma will likely spur fresh worries over pricing and reimbursement. Novartis has argued that the treatment would be cost effective at a price of $4 million to $5 million per patient.
While the Institute for Clinical and Economic Review has calculated a one-time gene therapy for SMA could be more cost-effective than Biogen's marketed drug Spinraza (nusinersen) in some scenarios, it also concluded prices that high would exceed the group's typical cost-effectiveness thresholds.