FDA Approves Catalent Facility to Aid in Zolgensma’s Production

The U.S. Food and Drug Administration (FDA) has given Catalent Biologics approval to aid in manufacturing products for use with AveXis’ spinal muscular atrophy (SMA) gene therapy, Zolgensma, at its Maryland facility.

The FDA inspected the company’s commercial-scale gene therapy manufacturing center in June.

Catalent and AveXis, a Novartis company, announced a long-term partnership giving AveXis greater production capacity for Zolgensma and further technical expertise in July 2019. Catalent has dedicated space at its Harmans facility for the commercial manufacture of the adeno-associated virus (AAV) used in Zolgensma.

“This is a significant milestone for Catalent and the gene therapy industry as a whole. Catalent is proud to be the first contract development and manufacturing organization to be approved for commercial gene therapy production,” Manja Boerman, president of Catalent, said in a press release.

“This approval allows us to leverage our now-licensed, state-of-the-art GMP commercial manufacturing facility, and our deep AAV expertise, to support AveXis as it delivers a life-changing treatment for patients.”

The facility is set up to support Phase 3 testing through commercial manufacturing of advanced therapeutic products, Catalent reported. These include AAV vectors like those used with Zolgensma, next-generation vaccines, and cancer immunotherapies. It houses over 200,000 square feet of late-stage clinical and commercial-stage gene therapy production.

The Harmans site is one of Catalent’s five gene therapy facilities in Maryland, and includes multiple manufacturing suites that have FDA current good manufacturing practices (CGMP) certifications, the company reported.

“Given the complexity and length of time required to make gene therapies, manufacturing is critically important,” said Dannielle Appelhans, chief technical officer for AveXis. “This approval further complements our internal manufacturing capacity and, over time, will allow us to increase supply to meet growing patient needs.”

Zolgensma is approved as an intravenous infusion therapy in children up to age 2 with any type of SMA in the U.S. and Japan, and for those with almost all types who weigh up to 21 kilograms (about 46 pounds) in Europe.

The therapy, intended to be a one-time treatment, provides patients with a healthy copy of the SMN1 gene, which is mutated in SMA. These mutations result in low or no levels of the SMN protein, which is critical to the proper function of motor neurons, being produced.

Zolgensma delivers the healthy gene to these neurons via a viral vector called AAV9, modified to be harmless.