Longmont-made gene therapy significantly extends infant patient survival rate, new data says

BASEL, Switzerland and LONGMONT — Novartis AG (NYSE: NVS) said its treatment for spinal muscular atrophy, which is partially made in Longmont, produces long-term benefits for its infant patients in an ongoing clinical study.

In a statement, the Swiss pharmaceutical company said a group of 29 infants with spinal muscular atrophy all survived months past the injection date without the need for a ventilator. Among those patients, who ranged from just more than three to just more than 21 months of age, 19 were able to sit on their own power for at least 30 seconds, and 11 of them were able to stand without aid.

Zolgensma is a one-time intravenous injection that combats the effects of SMA, a genetic disorder that prevents newborns from developing the strength in their backs needed to move body parts unassisted. Most patients die before they reach the age of 2.

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“This stands in sharp contrast to the natural progression of SMA Type 1, which would otherwise render them helpless within the first year of life,” said Dr. Kevin Strauss, the medical director of the Clinic for Special Children in Pennsylvania and a collaborator on the study.

One of the key facilities for producing the drug is the former AstraZeneca PLC (NYSE: AZN) complex in Longmont, which Novartis reopened in January 2020 with the goal of setting it up as a hub for producing hundreds of doses of Zolgensma and other gene therapies that the company is researching. The facility had employed 347 people as of its opening.

The drug is controversial for being the most expensive in the world, hitting the U.S. market in 2019 at a price of $2.1 million for the lone dose. Federal regulators also publicly chided the company for manipulation of pre-clinical trial data of the drug in mice, leading to the firing of two of the company’s top scientists. Regulators found no evidence of manipulation of human trial data and allowed the drug to remain for sale.

In its latest annual report, Novartis reported selling $920 million worth of the drug, split roughly in half between the U.S. market and other countries where the drug is available for sale.

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