Novartis today announced the U.S. Food and Drug Administration (FDA) has granted commercial licensure approval for its Durham, N.C. site, a multi-product gene therapy manufacturing facility. This approval allows the state-of-the-art, 170,000 square-foot facility to make, test and release commercial Zolgensma, as well as produce gene therapy product for current and future clinical trials.
The clearance of this milestone brings online the second commercially-licensed manufacturing facility for Novartis Gene Therapies, joining the Libertyville, Ill. site, which was approved for Zolgensma® (onasemnogene abeparvovec) manufacturing and distribution in 2019. The Durham site will build on Novartis Gene Therapies’ manufacturing capacity to form a two-site network, adding extensive segregated production suites for multi-product manufacturing.
“Bringing the North Carolina facility fully online reinforces the supply of Zolgensma and signifies the Novartis commitment to this critical advanced therapy platform,” said Christine Fox, President of Novartis Gene Therapies. “Not only will this facility support the Novartis pipeline through the manufacture of both clinical trial and commercial products, it ultimately allows us to help more patients and families living with rare, genetic diseases.”
The North Carolina facility is located in Research Triangle Park. Through close collaboration with the government and life sciences community, Novartis is contributing to this hub of innovation.
North Carolina Governor Roy Cooper said, "The investment by Novartis Gene Therapies in Durham illustrates North Carolina's leadership in the life sciences industry, our talented workforce and extensive resources to support major companies. This commercial licensure approval places our state at the forefront of cutting-edge gene therapies and we're looking forward to continued collaboration with Novartis."
The North Carolina site’s primary initial responsibility will be manufacturing Zolgensma®, an essential one-time treatment for spinal muscular atrophy (SMA). The facility will begin producing and shipping the gene therapy across the U.S. immediately.
If left untreated in its most severe forms, SMA leads to death or the need for permanent ventilation by the age of two in more than 90 percent of cases.1,2 Approved in more than 40 countries, to date, more than 1,800 patients have been treated with Zolgensma globally, including in clinical trials, managed access programs and in the commercial setting.
“Powered by our multidisciplinary teams and our pioneering technology, this milestone represents our ability to effectively manufacture and supply complex gene therapies at quality and scale,” added Steffen Lang, Global Head of Novartis Technical Operations.