- The Food and Drug Administration has cleared a Novartis manufacturing plant in North Carolina to produce commercial-grade gene therapies, giving the Swiss pharmaceutical giant a second agency-approved facility to make the complex medicines.
- The 170,000-square-foot plant located at Research Triangle Park officially opened in February 2020, but couldn't be properly used until Novartis obtained a commercial license. With the FDA's sign-off, the site can now fulfill its primary function: producing Zolgensma, a treatment the FDA approved in 2019 to treat young children with a rare neuromuscular disorder.
- Along with manufacturing commercial-grade Zolgensma, Novartis said the site will supply gene therapies for current and future clinical trials. The company's other licensed gene therapy plant in Libertyville, Illinois, can turn out roughly 700 to 800 doses of Zolgensma a year, which is enough to treat the infants in the U.S. who are currently eligible to receive the therapy.
Like several of its peers, Novartis has made significant bets on gene therapy and the technology's potential to essentially cure a wide array of diseases. In 2018, for instance, it spent almost $9 billion to acquire the biotech company responsible for developing Zolgensma. More recently, Novartis dropped another $800 million to purchase a small drugmaker working on genetic medicines for the eye.
While investments continue to pour in, gene therapy research still presents many unresolved challenges. A series of safety issues cropped up in clinical trials over the past couple of years, leading to more critical oversight from the FDA. Gene therapies have also proven exceptionally complex to make. Before Tuesday, Novartis had just the one site approved for commercial-grade Zolgensma manufacturing, which raised concerns about supply.
"We feel comfortable where we are, but we'd love to have these other sites onboard to make sure we're really robust and don't face any risks of shutdowns or anything that could impact supply," David Lennon, the former president of Novartis' gene therapy unit, told BioPharma Dive in February 2020, when the North Carolina plant was just coming on line.
In a statement, Christine Fox, the current president of the gene therapy division, said the clearance of the North Carolina plant "reinforces the supply of Zolgensma and signifies the Novartis commitment to this critical advanced therapy platform."
Initially, Novartis had aimed to win the FDA's sign-off last year, but filed for licensure "slightly later than first anticipated" so as to better prepare the site for inspection, a spokesperson said.
Novartis also had plans for an additional, nearly 700,000-square-foot gene therapy plant in Longmont, Colorado. But the company chose to shut the facility down a little more than a year after its opening in early 2020. Lennon said around that time that the decision was based on multiple factors, including delays in certain research projects and incorrect assumptions about how many Zolgensma doses would be needed to meet commercial and clinical demands.
Zolgensma received FDA approval as a treatment for children under the age of two with spinal muscular atrophy. Novartis is now testing the therapy in older children, research that could lead to an expanded label and a larger number of eligible patients.
Priced at more than $2 million, Zolgensma has become one of Novartis' top-selling drugs. The company recorded nearly $1.4 billion in net Zolgensma sales last year, up 47% from 2020.