Catalent, AveXis Enter Long-term Strategic Mfg. Pact

Catalent Biologics entered a long-term strategic agreement for the development and manufacture of Zolgensma (onasemnogene abeparvovec-xioi), an AveXis gene therapy treatment for spinal muscular atrophy (SMA).

AveXis, a Novartis company, will have dedicated manufacturing space at the new commercial manufacturing center near Baltimore-Washington International Airport established by Paragon Gene Therapy, a unit of Catalent Biologics. This additional capacity, along with access to Catalent’s adeno-associated virus (AAV) gene therapy development, manufacturing, and process characterization services, will allow for secure market supply of the newly approved treatment. Paragon Gene Therapy will also provide process development for clinical supply of additional viral therapies in the AveXis pipeline.

SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis, and when left untreated in its most severe form, permanent ventilation or death for most patients by age 2. Zolgensma is designed to address the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression through sustained SMN protein expression with a single one-time intravenous infusion. Zolgensma is the first and only gene therapy approved by the U.S. FDA for the treatment of SMA, including in patients who are pre-symptomatic at diagnosis.

"We are pleased to add additional manufacturing capacity and technical expertise through this collaboration as we rapidly scale up product production," commented Andy Stober, Senior Vice President of Technical Operations for AveXis. "We look forward to working with Catalent in continued service of the SMA community."

"We are honored to be a partner in this transformational journey," added Pete Buzy, Catalent’s President, 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. We work tirelessly to support a patient-first culture, and we are fully committed to be a premier collaborator with AveXis as they deliver this incredible new treatment for patients."

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