One-time gene therapy Zolgensma from Novartis shows lasting benefits for SMA patients

[Zolgensma image from Novartis]

A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations and demonstrated a positive overall benefit-risk profile. LT-001 is an ongoing 15-year long-term follow-up study of patients who completed the phase 1  START study. More than 3,000 children have been treated with Zolgensma in clinical trials, managed access programs and commercial settings. 

Novartis expects Zolgensma to eventually generate $1.5–2 billion, according to BioPharma Dive. NVS shares were up 1 percent to $83.01 today after announcing the new data. 

FDA approved Zolgensma (onasemnogene abeparvovec-xioi) in 2019 for chi…

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Novartis to shutter Zolgensma plant, resulting in 275 layoffs 

Basel, Switzerland–based Novartis has decided to consolidate the manufacturing for its gene therapy Zolgensma (onasemnogene abeparvovec-xioi). Plants dedicated to producing Zolgensma were based in Libertyville, Illinois and Durham, North Carolina. The company has announced that it will close the former plant, ultimately eliminating 275 jobs. 

The company anticipates that the Libertyville site will remain operational until the end of 2023. 

Novartis also will provide 90-day notifications to employees at the Libertyville site and offer severance packages and other benefits. 

Novartis first warned that it planned to close the plant in late October. 

Earlier this year, Novartis also announced its plan to spin off its Sandoz generic and biosimilar division.  

Sales of the gene therapy Zolgensma declined in the third quarter of this year, with its use mostly constrained to infants with spinal muscular atrophy. According to Novartis, sales of gene…

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