StemCell Therapy

Supplies of Zolgensma, the gene therapy approved last year for spinal muscular atrophy, are tight.

Novartis, which sells the one-time treatment, can currently make about 700 to 800 doses a year at its manufacturing plant in Libertyville, Illinois. That's enough to cover the infants in the U.S. currently eligible to receive Zolgensma but leaves little room for treating a wider group of patients, which the Swiss drugmaker aims to do.

On Thursday, executives from AveXis, the Novartis unit that developed Zolgensma, opened a new facility in Durham, North Carolina, that the company views as a critical cog in its plans to expand supply of the gene therapy.

Initial production will begin this spring. But until the Food and Drug Administration licenses the plant, Novartis won't be able to use product made there for commercial sale. The company expects to gain approval next year.

Between now and then, Novartis also hopes to secure regulatory OKs for manufacturing Zolgensma at a site in Longmont, Colorado, bought last year, and through the contract manufacturer Catalent.

"There is a short-term challenge over the next six to nine months to make sure that we can manage the supplies that are out there," said David Lennon, president of Novartis' AveXis unit, in an interview.

"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."

Limited supply has also kept Novartis from widening a program set up to make the gene therapy available free of charge to patients in countries where it's not yet approved. The "expanded access" scheme, which was launched in January, randomly allocates doses of Zolgensma for participating patients under the age of two with genetically confirmed spinal muscular atrophy.

This year, Novartis plans to distribute 100 doses through the lottery, which has been criticized as putting a child's life to chance.

"We obviously know that not everyone is happy with the program," said Lennon. "We're still considering what we might do, but we're open to making changes if it makes sense for the community and to meet the goals of the program."

Lennon said he hoped to expand the program as more manufacturing capacity for Zolgensma becomes available.

Novartis has committed upwards of $200 million to building out the site in Durham, which will employ about 400 staff by the end of the year.

Spanning 170,000 square feet, the facility will be used for both commercial Zolgensma manufacturing as well as to support clinical production of other gene therapies that Novartis is developing.

"This is as much an investment in the short term in building out our supply for Zolgensma, as it is for the long term to have the flexibility to deliver on a robust pipeline," said Lennon.

Novartis currently expects to treat about 100 infants every three months in the U.S. under Zolgensma's current label. But it's also working to expand the therapy's approval to treat older children over two using a spinal injection rather than an infusion.

That patient population is significantly larger and will test Novartis' ability to produce a steady supply of the drug, although the FDA has placed a partial "hold" on the study testing the new dosing.

Novartis' launch of Zolgensma is under significant scrutiny, both because of the $2.1 million price tag the drugmaker put on the therapy and due to a data manipulation scandal that engulfed the company last year.

Despite the high cost, insurers have largely covered treatment, leading to strong sales of Zolgensma in its first three quarters on the market.

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With supply tight, Novartis readies gene therapy plant for production - BioPharma Dive

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