AveXis, a Novartis Company, will use Catalent-owned Paragon Gene Therapy’s new commercial manufacturing center near Baltimore-Washington International Airport as an additional manufacturing site for its spinal muscular atrophy (SMA) gene therapy Zolgensma® (onasemnogene abeparvovec-xioi), Catalent Biologics said today.
Catalent and AveXis have signed a long-term strategic agreement whose value was not disclosed. AveXis will use dedicated manufacturing space within Paragon’s Harmans, MD, facility, a 151,000-square-foot former warehouse that Paragon converted into a contract developing and manufacturing site which opened in April.
“We are pleased to add additional manufacturing capacity and technical expertise through this collaboration as we rapidly scale up product production,” Andy Stober, senior vp of technical operations for AveXis, said in a statement. “We look forward to working with Catalent in continued service of the SMA community.”
Catalent said the additional capacity, along with access to its adeno-associated virus (AAV) gene therapy development, manufacturing, and process characterization expertise, will enable AveXis to ensure a stable and consistent supply of Zolgensma as AveXis prepares to launch the gene therapy.
“This collaboration allows us to leverage our new state-of-art GMP commercial manufacturing facility and AAV development and scale-up expertise to support AveXis from the early development stage to this critical launch,” added Pete Buzy, Catalent’s president, gene therapy.
Paragon Gene Therapy will also provide process development for clinical supply of additional viral therapies in AveXis pipeline, Catalent added.
Zolgensma won the FDA’s first approval for an SMA gene therapy on May 24. While most public attention on Zolgensma has focused on its list price of $2.1 million—Novartis has said it is developing discounted patient-access programs with insurers—AveXis has sought to ensure it can manufacture the gene therapy.
Beyond in-house manufacturing
The collaboration with Paragon reflects AveXis’ interest in looking beyond in-house capacity to meet Zolgensma-related manufacturing needs. Until now, AveXis had addressed manufacturing of the gene therapy by lining up four U.S. production sites, including AstraZeneca’s former advanced biologics therapy manufacturing campus in Longmont, CO, which AveXis agreed to acquire in April. AveXis paid $30 million for the Longmont site in a sale recorded April 2, according to data from the Boulder County Assessor’s Office.
AveXis’ other three sites include a manufacturing facility in Bannockburn, IL, where the company is headquartered; a Durham, NC, facility that the company began building in 2018 and said in February it would expand, creating 400 jobs by the time it becomes operational in 2020; and a San Diego facility whose product development capacity is being expanded.
Zolgensma is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The vector is designed to deliver a fully functional copy of human SMN gene into the target motor neuron cells.
A one-time intravenous administration of Zolgensma is intended to result in expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and survival of a child with SMA. Dosing is determined based on the weight of the patient.
The FDA based its approval on positive clinical data showing that Zolgensma provided unprecedented rates of survival never seen in the natural history of the disease; rapid motor function improvement, often within one month of dosing; and durable milestone achievement, including the ability to sit without support, a milestone never achieved in untreated patients.