Novartis’ North Carolina gene therapy manufacturing site gets FDA approval

By Rachel Arthur

- Last updated on GMT


Related tags Novartis Gene therapy Spinal muscular atrophy Us

The US Food and Drug Administration (FDA) has granted commercial licensure approval for Novartis’ Durham, N.C. site, a multi-product gene therapy manufacturing facility, which will start producing Zolgensma immediately.

This approval allows the state-of-the-art, 170,000 square-foot facility to make, test and release commercial Zolgensma (onasemnogene abeparvovec), a treatment for spinal muscular atrophy (SMA)and the initial focus for the site, as well as produce gene therapy product for current and future clinical trials.

The Durham site becomes the second commercially-licensed manufacturing facility for Novartis Gene Therapies, joining the Libertyville, Illinois site, which was approved for Zolgensma manufacturing and distribution in 2019. The second site adds 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.

Novartis gained Zolgensma as part of its $8.7bn acquisition of AveXis Inc in 2018; gaining FDA approval the following year. Known as the world’s most expensive drug, the one-dose treatment costs $2m.

Sales for Zolgensma reached $1.4bn in 2021, up 47% on the year before. Steady US sales were joined by expanded access in Europe and other markets.

Last year, Novartis decided to shut down an additional manufacturing site for Zolgensma in Longmont, Colorado: deciding it only needed two sites instead of three for the drug. The site has been acquired by AGC Biologics.


Approved in more than 40 countries, more than 1,800 patients have been treated with Zolgensma globally to date.

It is the only approved gene therapy for the treatment of spinal muscular atrophy (SMA) 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.

Novartis Gene Therapies continues to evaluate Zolgensma across a robust clinical development program, as well as the investigational intrathecal administration of OAV101 in patients with later-onset forms of SMA.

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.

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