Novartis and Catalent enter agreement to manufacture SMA gene therapy

18-Jul-2019 - Last updated on 18-Jul-2019 at 14:22 GMT

Catalent Biologics works with Novartis to develop and manufacture its SMA gene therapy treatment and provide manufacturing capacity, as the treatment enters the market.

Paragon, Catalent Biologics’s newly purchased subsidiary, entered an agreement with AveXis to develop and manufacture its spinal muscular atrophy (SMA) treatment, Zolgensma (onasemnogene abeparvovec-xioi), and provide greater capacity for its oncology pipeline.

Through the long-term agreement, the gene therapy treatment from AveXis, which was acquired by Novartis in April 2018 for $8.7bn (€7.75bn), will have dedicated production space at a commercial manufacturing center in Baltimore, Maryland, established by Paragon Gene Therapy.

This additional capacity will provide space to enable a secure market supply for Zolgensma, which was recently approved in the US by the Food and Drug Administration (FDA).

Andy Stober, CTO of AveXis, told us that it has the most gene therapy manufacturing capacity in the industry, but it is continuing to expand capacity to meet the needs of patients.

AveXis has invested in facilities across the US in Illinois, North Carolina, and Colorado, and Stober explained that, after the acquisition by Novartis, it has been able to scale quickly and expand its pipeline, which requires additional manufacturing capacity.

Paragon Gene Therapy will give AveXis access to Catalent’s adeno-associated virus (AAV) gene therapy development, manufacturing, and process characterization capabilities. Paragon will provide process development for clinical supply of additional viral therapies in AveXis’ pipeline.

Stober told us that Paragon has been an established partner of the company over the last few years.

“We have aligned, patient-focused cultures and [Paragon] have been able to work on our expedited timelines, sharing the same sense of urgency to deliver product for devastating, rare genetic diseases. They’re also able to expand and contract rapidly, and that flexibility is important to our overall manufacturing strategy,” he explained.

Pricey but promising

Zolgensma treats SMA as it works on the SMN1 gene to prevent disease progression through sustained protein expression after a single one-time intravenous infusion. Novartis stated that the treatment price, the most expensive drug on the market, is broken into five annual payments of $425,000m, for a total cost of $2.1m per patient.

The treatment is the first and only gene therapy approved by the FDA for the treatment of SMA. Without treatment the rare genetic disease leads to progressive muscle weakness, paralysis and can led to permanent ventilation or death by age two.

A spokesperson for Catalent Biologics told us that the SMA Foundation estimates that SMA affects as many as 10,000 to 25,000 children and adults in the US, making it one of the most common rare diseases in the country. They also said that one in 6,000 to one in 10,000 children are born with the disease.

Novartis recently entered an agreement with SAVSU Technologies, which has been acquired by BioLife Solutions, to use the companies cold chain shipment technology, after the technology had been previously adopted by AveXis.