Novartis expands Zolgensma® manufacturing capacity
with approval of multi-product North Carolina facility
The FDA has granted commercial licensure approval for Novartis’ 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. 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.
Zolgensma® is the only approved gene therapy for the treatment of spinal muscular atrophy and the only SMA treatment designed to directly address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. 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.
Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children’s Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self- complementary DNA technology for the treatment of SMA.